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Investigating the Human Intestinal Mucus Barrier Up-close and Personal – Technology Networks

We have a mutualistic but complicated relationship with the collection of microbes in our gut, known as the intestinal microbiome. This complex community of bacteria breaks down different food components, and releases nutrients such as vitamins and a plethora of other factors that control functions in tissues way beyond the intestinal tract. However, the sheer numbers of microbes also present a threat as they can trigger inflammation, which is thought to be at the root of many intestinal diseases, including inflammatory bowel disease, radiation-induced intestinal injury, and some cancers.

To allow the uptake of beneficial substances from the gut lumen, and at the same time prevent gut microbes from contacting the intestinal epithelial tissue surface, specialized cells called goblet cells continuously produce mucus, the slimy goo-like substance that coats the entire intestinal surface. Mucus thus far has been notoriously difficult to study: its structure quickly disintegrates in surgically removed sections of the gut, the system most often used to study mucus, and no in vitro culture system has been able to reconstitute an in vivo-like mucus layer with the natural structure seen in living intestine outside the human body. Adding to these difficulties, mucus also differs between humans and other species, different sections of the intestinal tract, and even different individuals.

Now, focusing on the large intestine or colon which houses the greatest number of commensal microbes and has the thickest mucus layer, a team of tissue engineers at Harvards Wyss Institute for Biologically Inspired Engineering has developed a colon-on-a-chip (Colon Chip) microfluidic culture device lined by patient-derived colon cells that spontaneously accumulates a mucus layer with the thickness, bi-layered structure, and barrier functions typically found in normal human colon. The mucosal surface in their model also responds to the inflammatory mediator prostaglandin E2 (PGE2) by mounting a rapid swelling response. Their findings are published in Cellular and Molecular Gastroenterology and Hepatology.

Our approach provides researchers with the opportunity to find answers to questions about normal and disease-associated mucus biology, such as its contributions to intestinal inflammatory diseases and cancers, and complex host-microbiome interactions, said Founding Director Donald Ingber, M.D., Ph.D., who is the senior investigator on the study. Importantly, we use patient-derived cells to line these devices and so this represents an entirely new approach for personalized medicine where it can be possible to study how mucus functions or dysfunctions in a particular patient, and to tailor therapy accordingly.

Ingber is also the Judah Folkman Professor of Vascular Biology at Harvard Medical School and the Vascular Biology Program at Boston Childrens Hospital, as well as Professor of Bioengineering at Harvards John A. Paulson School of Engineering and Applied Sciences. His team is part of a multi-institutional collaboration supported by a Cancer Research UK Grand Challenge grant in which his Wyss team investigates how inflammation-related changes contribute to formation of cancers, including colon cancers. The Grand Challenge is an ambitious international cancer research initiative, supporting world-leading teams of scientists to take on some of the toughest challenges in cancer, and giving them the freedom to try novel approaches at scale.

The teams approach starts out with patient-derived colon cells from colon resections and endoscopic biopsies that are first grown as organoids, tiny organized balls of colon tissue that contain mainly epithelial stem cells. After fragmenting the organoids, their cells are used to populate the upper of a two parallel channels of a microfluidic chip that are separated by a porous membrane. Simply by perfusing the channels continuously with nutrient medium, the colon stem cells grow into a continuous sheet and form highly functional goblet cells that secrete mucus.

Growing the cells on-chip under flow results in about 15% of epithelial cells spontaneously differentiating into goblet cells. Distributed throughout the epithelium, these produce an in vivo-like mucus layer, said first-author Alexandra Sontheimer-Phelps, a graduate student from the University of Freiburg, Germany, working in Ingbers group. At the same time, other epithelial cells that keep dividing also replenish the goblet cell population just like in living colon, which means that the chip can be maintained in steady-state conditions for more than two weeks, which makes it highly useful for longer-term studies.

The Wyss team showed that the colon epithelium in the chip is fully polarized with distinct markers restricted to its lumen-exposed, mucus-secreting side and its opposite membrane-binding side. Its goblet cells secrete the major mucus protein mucin 2 (MUC2), which when linked to complex chains of sugar molecules, assembles into multi-molecular network or gel that takes up water. Our approach actually produces the bi-layered structure of normal colon mucus with an inner dense layer that we show is impenetrable to bacteria-mimicking particles flowed through the intestinal channel, and a more loose outer layer that allows particles to enter. This has never been accomplished before in vitro, said Sontheimer-Phelps.

To investigate the functionality of the mucus, she and her co-workers exposed the chip to the inflammatory mediator PGE2. The mucus underwent rapid swelling within minutes and independent of any new mucus secretion, and this process of mucus accumulation can be visualized in living cultures by viewing the chips from the side with dark field illumination. This dynamic response could be blocked by inhibiting one particular ion channel, which pumps ions into the colon epithelium and passively allow water molecules to follow and apparently, this drives mucus swelling when stimulated by signals such as PGE2.

Mucus has long been thought to be a passive, host barrier, but it is becoming increasingly clear that microbial species affect its structure and function in addition to feeding on its carbohydrates as an energy source. Our in vitro system brings us one step closer to figuring out how individual bacterial species and more complex microbial communities can affect mucus and vice versa, as well as how this complex interplay impacts development of intestinal diseases. We also now have a testbed to discover new therapeutic drug and probiotic strategies that might prevent or reverse these diseases said Ingber.

Reference:Sontheimer-Phelps, A., Chou, D. B., Tovaglieri, A., Ferrante, T. C., Duckworth, T., Fadel, C., Ingber, D. E. (2019). Human colon-on-a-chip enables continuous in vitro analysis of colon mucus layer accumulation and physiology. Cellular and Molecular Gastroenterology and Hepatology. https://doi.org/10.1016/j.jcmgh.2019.11.008

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Investigating the Human Intestinal Mucus Barrier Up-close and Personal - Technology Networks

Stem cells may trigger immune repair to mend hearts – BioNews

2 December 2019

Stem cell therapies may become redundant in repairing cardiac function after a heart attack, suggests a new study in mice.

It showed how stem cell treatments can heal hearts by triggering an immune response which can be achieved by using a chemical instead.

'This work is paradigm-shifting because it demonstrates a mechanism to explain a perplexing phenomenon that has intrigued cardiologists as a result of decades of cardiac stem cell trials,' Dr Jonathan Epstein at the University of Pennsylvania's Perelman School of Medicine in Philadelphia told The Scientist.

Stem cell therapies to repair damaged heart tissue are currently being tested in human clinical trials. In these treatments, human stem cells are injected into the heart and this leads to an improvement in heart function. However, how this works is not fully understood.

One possibility is that the injected stem cells are incorporated into the heart tissue and repair the damage. However, the latest study, published in the journal Nature, suggests that this may not be the case. Instead, the study indicated that the repair is actually a result of triggering the innate immune response.

Researchers injected different types of stem cell or a chemical inducer (zymosan) of the innate immune response into an experimental mouse model of heart disease. They saw improvement in heart function that was similar in all cases, and showed that this repair occurs via activation of macrophage cells of the innate immune system.

'The innate immune response acutely altered cellular activity around the injured area of the heart so that it healed with a more optimised scar and improved contractile properties,' said Dr Jeffery Molkentin at the University of Cincinnati and Cincinnati Children's Hospital Medical Centre, Ohio, who led the study. 'The implications of our study are very straightforward and present important new evidence about an unsettled debate in the field of cardiovascular medicine.'

The work could open up new possibilities for optimising the treatments currently in development, as well as alternative new therapies.

'If there is a chemical off-the-shelf, it would be a much more feasible therapy [than stem cell transplants],'Dr Kory Lavine at Washington University in St Louis, Missouri, told Nature News.

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Stem cells may trigger immune repair to mend hearts - BioNews

Omeros Reports Positive Data Across Primary and Secondary Endpoints in Pivotal Trial of Hematopoietic Stem Cell Transplant-Associated Thrombotic…

SEATTLE--(BUSINESS WIRE)--Omeros Corporation (Nasdaq: OMER) today announced positive data from its pivotal clinical trial of the companys novel investigational complement inhibitor narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), a frequently lethal complication of HSCT. These preliminary data were recently provided to FDA as part of the companys ongoing interactions with the Agency on the narsoplimab Biologics License Application (BLA). All safety and efficacy endpoints including the composite primary endpoint and the secondary endpoints are agreed with FDA. The reported data support a strongly positive benefit-risk balance.

The primary efficacy endpoint in this single-arm open-label trial of HSCT-TMA patients is the proportion of patients who achieve a highly rigorous set of response criteria that requires both improvement in HSCT-TMA laboratory markers and improvement in clinical status (organ function and transfusions). Patients who did not fully meet these criteria were considered non-responders. The secondary endpoints include survival rates and change from baseline in HSCT-TMA laboratory markers. Consistent with the pre-specified statistical analysis plan for the trial, the primary and secondary endpoints are assessed for (1) all patients who received at least one dose of narsoplimab and (2) patients who received at least 4 weeks of narsoplimab dosing. Patients enrolled in this trial had a high expected mortality rate. In severe cases of HSCT-TMA, mortality can exceed 90 percent.

Primary Efficacy Endpoint:

Secondary Endpoints:

Safety:

The HSCT-TMA patient population enrolled in this trial had multiple high-risk features that portend a poor outcome. These include persistence of HSCT-TMA despite modification of immunosuppression (which was a criterion for entry into the trial), graft-versus-host disease, significant infections, non-infectious pulmonary complications and neurological findings. Patients in the trial had a high expected death rate, with 93 percent of them having multiple risk factors.

Patient enrollment in the pivotal trial has been completed. The details of the endpoints, including the response criteria agreed with FDA, and the number of patients in the trial remain confidential for competitive business reasons.

Last year the company reported data on 19 HSCT-TMA patients treated with narsoplimab on which FDA granted breakthrough therapy designation. The results reported today are even stronger. The response rate remains equally high at 56 percent, while the 100-day survival has improved from 53 percent to 65 percent.

The response rate in this high-risk population would be expected to be 10 to 15 percent with a 100-day survival rate of less than 20 percent. The response rate and 100-day survival achieved with narsoplimab in this trial demonstrate an unprecedented effect in this condition, said Rafael Duarte M.D., Ph.D., F.R.C.P., Associate Professor, Head of Hematology Department and Hematopoietic Transplantation Program, University Hospital Puerta de Hierro Majadahonda, Madrid, Spain, and Secretary of the European Society for Blood and Marrow Transplantation. The other secondary endpoints are equally impressive. The data are consistent with my personal experience with narsoplimab. Patients with severe forms of HSCT-TMA have a dismal prognosis with no treatment currently available. I expect a treatment with this profile would be widely adopted for use in these patients and even lead to increased physician recognition of the disorder.

Omeros reported the initiation of its rolling BLA in October. Narsoplimab, also referred to as OMS721, is Omeros lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2) and has breakthrough therapy designation from FDA for this indication.

The striking results seen in our pivotal trial are tremendously gratifying, said Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros. Our rolling BLA is underway the nonclinical sections have been submitted and the data from this trial form the efficacy basis of the application. We continue to compile the remaining sections of the BLA and look forward to continued partnership with regulators to make narsoplimab widely available for the treatment of this devastating condition.

Data from this pivotal trial will also support the narsoplimab marketing authorization application for HSCT-TMA in Europe. The data are planned for publication and for presentation at international congresses in the first part of 2020.

In addition to breakthrough therapy designation from FDA, narsoplimab has orphan drug designation in both the U.S. and Europe for HSCT-TMA. Narsoplimab also has been awarded breakthrough therapy designation for immunoglobulin A nephropathy (IgAN), and Omeros has Phase 3 programs for narsoplimab ongoing in IgAN and in atypical hemolytic uremic syndrome (aHUS).

Conference Call and Webcast Details

Omeros management will host a webcast and conference call to present data from its pivotal trial of narsoplimab in HSCT-TMA. The call will be held today at 8:30 a.m. Eastern Time; 5:30 a.m. Pacific Time. To access the live conference call via phone, please dial (844) 831-4029 from the United States and Canada or (920) 663-6278 internationally. The participant passcode is 3774209. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available for one week following the call and may be accessed by dialing (855) 859-2056 from the United States and Canada or (404) 537-3406 internationally. The replay passcode is 3774209.

To access the live or subsequently archived webcast and presentation materials on the internet, go to the companys website at http://www.omeros.com and select Events under the Investors section of the website. To access the live webcast, please connect to the website at least 15 minutes prior to the call to allow for any software download that may be necessary.

About Omeros Corporation

Omeros is an innovative biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting complement-mediated diseases, disorders of the central nervous system and immune-related diseases, including cancers. In addition to its commercial product OMIDRIA (phenylephrine and ketorolac intraocular solution) 1%/0.3%, Omeros has multiple Phase 3 and Phase 2 clinical-stage development programs focused on complement-mediated disorders and substance abuse. In addition, the company has a diverse group of preclinical programs including GPR174, a novel target in immuno-oncology that modulates a new cancer immunity axis recently discovered by Omeros. Small-molecule inhibitors of GPR174 are part of Omeros proprietary G protein-coupled receptor (GPCR) platform through which it controls 54 new GPCR drug targets and their corresponding compounds. The company also exclusively possesses a novel antibody-generating platform.

About HSCT-TMA

Hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) is a significant and often lethal complication of stem cell transplants. This condition is a systemic, multifactorial disorder caused by endothelial cell damage induced by conditioning regimens, immunosuppressant therapies, infection, GvHD, and other factors associated with stem cell transplantation. Endothelial damage, which activates the lectin pathway of complement, plays a central role in the development of HSCT-TMA. The condition occurs in both autologous and allogeneic transplants but is more common in the allogeneic population. In the United States and Europe, approximately 25,000 to 30,000 allogeneic transplants are performed annually. Recent reports in both adult and pediatric allogeneic stem cell transplant populations have found an HSCT-TMA incidence of approximately 40 percent, and high-risk features may be present in up to 80 percent of these patients. In severe cases of HSCT-TMA, mortality can exceed 90 percent and, even in those who survive, long-term renal sequalae are common. There is no approved therapy or standard of care for HSCT-TMA.

About Narsoplimab

Narsoplimab, also known as OMS721, is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), a novel pro-inflammatory protein target and the effector enzyme of the lectin pathway of complement. Importantly, inhibition of MASP-2 does not appear to interfere with the antibody-dependent classical complement activation pathway, which is a critical component of the acquired immune response to infection. Omeros controls the worldwide rights to MASP-2 and all therapeutics targeting MASP-2.

Phase 3 clinical programs are in progress for narsoplimab in hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), in immunoglobulin A (IgA) nephropathy, and in atypical hemolytic uremic syndrome (aHUS). The FDA has granted narsoplimab breakthrough therapy designations for HSCT-TMA and for IgA nephropathy; orphan drug status for the prevention (inhibition) of complement-mediated thrombotic microangiopathies, for the treatment of HSCT-TMA and for the treatment of IgA nephropathy; and fast track designation for the treatment of patients with aHUS. The European Medicines Agency has granted orphan drug designation to narsoplimab for treatment in HSCT and for treatment of primary IgA nephropathy.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, which are subject to the safe harbor created by those sections for such statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, likely, look forward to, may, on track, plan, potential, predict, project, prospects, scheduled, should, slated, targeting, will, would and similar expressions and variations thereof. Forward-looking statements, including statements regarding anticipated regulatory submissions, expectations regarding regulatory exclusivities, the timing and results of ongoing or anticipated clinical trials, and the therapeutic application of Omeros investigational product, are based on managements beliefs and assumptions and on information available to management only as of the date of this press release. Omeros actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, availability and timing of data from clinical trials and the results of such trials, unproven preclinical and clinical development activities, regulatory oversight, intellectual property claims, competitive developments, litigation, and the risks, uncertainties and other factors described under the heading Risk Factors in the companys Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 1, 2019. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the company assumes no obligation to update these forward-looking statements, whether as a result of any new information, future events or otherwise, except as required by applicable law.

Source: Omeros Corporation

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Omeros Reports Positive Data Across Primary and Secondary Endpoints in Pivotal Trial of Hematopoietic Stem Cell Transplant-Associated Thrombotic...

Hunter Syndrome Treatment Market Size, Share & Trend Analysis By Treatment, By Region And Segment Forecasts, 2019 – 2026 – P&T Community

NEW YORK, Dec. 4, 2019 /PRNewswire/ --

Hunter Syndrome Treatment Market Size, Share & Trend Analysis By Treatment (Enzyme Replacement Therapy, Hematopoietic Stem Cell Transplant), By Region, And Segment Forecasts, 2019 - 2026

Read the full report: https://www.reportlinker.com/p05830601/?utm_source=PRN

The global hunter syndrome treatment market size is expected to reach a value of USD 1.52 billion by 2026, expanding at a CAGR of 7.1%. High unmet needs, robust pipeline, increasing awareness about this rare disease and growing R&D activities for the development of novel therapies are expected to drive market growth over the forecast period.

Hunter syndrome, also referred as mucopolysaccharidosis type II (MPS II), is a rare genetic disorder caused by the missing or malfunctioning iduronate-2-sulfatase enzyme. According to the data published by the National Institute of Neurological Disorders and Stroke, MPS II syndrome occurs in around 1 in every 100,000 to 150,000 male births.

Presently, there are no approved curative therapies for the treatment of Hunter syndrome.The available treatment options such as enzyme replacement therapy (ERT) and hematopoietic stem cell transplant (HSCT) are focused on providing symptomatic relief and management of complications associated with disease progression.

Shire plc's Elaprase (idursulfase) is the only key drug available for the treatment of Hunter syndrome worldwide, with GC Pharma's Hunterase (idursulfase beta) being approved only in South Korea.

Key players are focused on extensive R&D activities for product development and gaining approval as novel therapies.Launch of such novel therapies in the near future is expected to significantly fuel the Hunter syndrome treatment market growth.

For instance, in May 2018, REGENXBIO Inc. received the U.S. FDA's Fast Track designation for its novel drug candidate RGX-121, indicated for the disease treatment.

Further Key Findings from the Study Suggest: The enzyme replacement therapy segment has acquired the largest share in 2018, owing to the increased adoption of Elaprase and the potential approval of Hunterase worldwide The absence of curative therapies for MPS II creates lucrative opportunities for the key players in the market for developing new therapies The launch of late-stage pipeline therapies is expected to drive the market growth during the forecast period Currently, Shire Plc. is one of the leading players, supported by strong sales of their marketed drug ELAPRASE for the disease treatment Major players in the market are adopting inorganic growth strategies such as partnerships and collaborations for the development and commercialization of novel therapies In 2018, North America held a dominant position in the global market, owing to favorable reimbursement scenario, high awareness regarding rare disorders, and presence of major players Some of the key companies in hunter syndrome treatment market include GC Pharma, Sangamo Therapeutics, Inc.; JCR Pharmaceuticals Co Ltd.; RegenxBio Inc.; and Shire Plc. (Takeda Pharmaceutical Company)

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Hunter Syndrome Treatment Market Size, Share & Trend Analysis By Treatment, By Region And Segment Forecasts, 2019 - 2026 - P&T Community

Platelet Rich Plasma and Stem Cell Alopecia Treatment Market is on Course to Expand at a CAGR of XX% Over the Forecast Period 2018 – 2026 – Weekly Spy

Latest Study on the Global Platelet Rich Plasma and Stem Cell Alopecia Treatment Market

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As per the report, the global Platelet Rich Plasma and Stem Cell Alopecia Treatment market is projected to grow at a CAGR of ~XX% and exceed the value of ~US$ towards the end of 2029. Moreover, an in-depth analysis of the micro and macro-economic factors that are anticipated to influence the trajectory of the Platelet Rich Plasma and Stem Cell Alopecia Treatment market during the forecast period (2019-2029) is included in the report.

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competitive landscape of the industrial workwear market, thereby positioning all the major players according to their geographic presence and recent key developments. The comprehensive industrial workwear market estimates are the result of our in-depth secondary research, primary interviews, and in-house expert panel reviews. These market estimates have been analyzed by taking into account the impact of different political, social, economic, technological, and legal factors along with the current market dynamics affecting the growth of the industrial workwear market. Market share analysis has been included under the competitive landscape to understand the top players contribution to the market. Each company is studied on the basis of basic information, financial highlights, revenue highlights of regional contribution and segment contribution, and product portfolio. Additionally, the company strategy and recent developments if any are also incorporated under each company profile section.

Some of the key players in the industrial workwear market include 3M, VF Corporation, Ansell Ltd., and Honeywell International Ltd., E. I. du Pont de Nemours and Company, Williamson-Dickie Manufacturing Co., Hultafors Group, Lakeland Inc., Aramark, and Fristads Kansas Group. The key players operating in the industrial workwear market adopt numerous strategies to sustain in the market. Product development and acquisition and partnership are some of the main strategies adopted by key players to gain a competitive edge.

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Industrial Workwear Market

By Product Type

By End-use Industry

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By Geography

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Platelet Rich Plasma and Stem Cell Alopecia Treatment Market is on Course to Expand at a CAGR of XX% Over the Forecast Period 2018 - 2026 - Weekly Spy

miR-194 Inhibits the Proliferation of SW620 Colon Cancer Stem Cells Th | CMAR – Dove Medical Press

Bo Sun,1,* Yan-Tian Fang,1,* Dan-Juan Jin,2 Zong-You Chen,3 Zhen-Yang Li,3 Xiao-Dong Gu,3 Jian-Bin Xiang3

1Department of Gastric Cancer, Fudan University Shanghai Cancer Center, Shanghai, Peoples Republic of China; 2Department of General Surgery, Songjiang District Central Hospital, Shanghai, Peoples Republic of China; 3Department of General Surgery, Huashan Hospital, Fudan University, Shanghai, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Xiao-Dong Gu; Jian-Bin XiangDepartment of General Surgery, Huashan Hospital, Fudan University, 12 Wulumuqi Middle Road, Shanghai 200040, Peoples Republic of ChinaTel +86-21-5288 7044; +86-21-5288 7040Fax +86-21-62495490Email gxdgxd737@163.com; xjbzhw131@sohu.com

Purpose: Colorectal cancer (CRC) stem cells are tumorigenic, capable of self-renewal, and resistant to therapy. Although the expression pattern and functions of micro RNA (miR)-194 in CRC cells have been widely investigated, little is known about its role in CRC stem cells. Therefore, the aim of this study was to investigate the potential role of miR-194 in CRC stem cells.Materials and methods: CRC stem cells were isolated from the SW620 colon cancer cell line using microbeads. The expression levels of miR-194 and slingshot 2 (SSH2) in CRC stem cells were detected by RT-PCR and Western blot. A luciferase reporter assay was performed to confirm that miR-194 directly targets SSH2. Proliferation of CRC stem cells was examined by colony formation and MTT assays. Apoptosis in CRC stem cells was detected by cell cycle and apoptosis assays. The role of miR-194 in tumor growth was determined in vivo.Results: Cells positive for CD44 and CD133 accounted for approximately 88.7% of the isolated population after microbead isolation. We reveal for the first time that miR-194 expression is decreased in CRC stem cells. Specifically, miR-194 is involved in inhibiting the proliferation of CRC stem cells and promoting CRC stem cell apoptosis by directly targeting SSH2. Furthermore, overexpression of miR-194 resulted in blocking the G1/S transition, the induction of cellular apoptotic process, thereby suppressing the malignant behaviors of CRC stem cells.Conclusion: This study represents a novel characterization of miR-194 function in CRC stem cells, which may aid in the development of promising therapeutic strategies targeting CRC.

Keywords: colorectal cancer, miR-194, SSH2, proliferation, stem cell

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Stem Cell Therapy Market Growth opportunities, Trends, Industry Analysis, and Forecast to 2020 – Kentucky Reports

Persistence Market Research(PMR), in its recent market report, suggests that the Stem Cell Therapy Market report is set to exceed US$ xx Mn/Bn. The report finds that the Stem Cell Therapy Market registered ~US$ xx Mn/Bn in 2018 and is spectated to grow at a healthy CAGR over the foreseeable period 2020.

The Stem Cell Therapy Market research focuses on the market structure and various factors (positive and negative) affecting the growth of the market. The study encloses a precise evaluation of the Stem Cell Therapy Market, including growth rate, current scenario, and volume inflation prospects, on the basis of DROT and Porters Five Forces analyses. In addition, the Stem Cell Therapy Market study provides reliable and authentic projections regarding the technical jargon.

ThisPress Release will help you to understand the Volume, growth with Impacting Trends. Click To get SAMPLE PDF (Including Full TOC, Table & Figures) athttps://www.persistencemarketresearch.co/samples/3253

The Stem Cell Therapy Market study answers critical questions including:

The content of the Stem Cell Therapy Market report includes the following insights:

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All the players running in the global Stem Cell Therapy Market are elaborated thoroughly in the Stem Cell Therapy Market report on the basis of R&D developments, distribution channels, industrial penetration, manufacturing processes, and revenue. In addition, the report examines, legal policies, and comparative analysis between the leading and emerging Stem Cell Therapy Market players.

Some of the major companies operating in the global stem cell therapy market are Mesoblast Ltd., Celgene Corporation, Aastrom Biosciences, Inc. and StemCells, Inc.

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About us:

Persistence Market Research (PMR) is a third-platform research firm. Our research model is a unique collaboration of data analytics and market research methodology to help businesses achieve optimal performance.

To support companies in overcoming complex business challenges, we follow a multi-disciplinary approach. At PMR, we unite various data streams from multi-dimensional sources. By deploying real-time data collection, big data, and customer experience analytics, we deliver business intelligence for organizations of all sizes.

Contact us:Persistence Market Research305 Broadway, 7th FloorNew York City, NY 10007United StatesPh.no. +1-646-568-7751

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Stem Cell Therapy Market Growth opportunities, Trends, Industry Analysis, and Forecast to 2020 - Kentucky Reports

As per new Study on Stem Cell Therapy Market 2019 Future Strategy, Analysis and Prediction by Leading Manufacturers, its Application and Types…

A new business intelligence report released by Garner Insights with title Global Stem Cell Therapy Market Research Report 2019 that targets and provides comprehensive market analysis with prospects to 2024. The analysts of the study have garnered extensive research methodologies and data sources (i.e. Secondary & Primary Sources) in order to generate collective and useful information that delivers latest market undercurrents and industry trends.

Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use.

Get a Sample PDF Report: https://www.garnerinsights.com/Global-Stem-Cell-Therapy-Market-2019-by-Company-Regions-Type-and-Application-Forecast-to-2024#request-sample

Some of key competitors or manufacturers included in the study are: Osiris Therapeutics, NuVasive, Chiesi Pharmaceuticals, JCRPharmaceutical, Pharmicell, Medi-post, Anterogen, Molmed, Takeda (TiGenix),

If you are involved in the Global Stem Cell Therapy industry or intend to be, then this study will provide you comprehensive outlook. Its vital you keep your market knowledge up to date segmented by major players. If you have a different set of players/manufacturers according to geography or needs regional or country segmented reports, we can provide customization according to your requirement.

Market Segment by Type, covers: Autologous, Allogeneic

Market Segment by Applications, can be divided into: Musculoskeletal Disorder, Wounds & Injuries, Cornea, Cardiovascular Diseases, Others,

Market segment by Regions/Countries, this report coversNorth AmericaEuropeChinaRest of Asia PacificCentral & South AmericaMiddle East & Africa

What are the affecting elements that are made reference to in the report?

Market Scenario:The report further highlights the development trends in the global Stem Cell Therapy market. Factors that are driving the market growth and fueling its segments are also analyzed in the report. The report also highlights on its applications, types, deployments, components, developments of this market.

Key Market Highlights:The Stem Cell Therapy report gives a top to bottom examination on a portion of the key elements, involving income, cost, limit, limit usage rate, creation, generation rate, utilization, import/send out, supply/request, net, piece of the pie, CAGR, and gross edge. Furthermore, the report shows a far reaching investigation of the market development factors and their most recent patterns, alongside important market fragments and sub-portions.

Analytical Tools:The Global Stem Cell Therapy Market report incorporates the decisively examined and assessed information of the significant market members and their market scope utilizing various investigative devices. The diagnostic apparatuses incorporate Porters five powers examination, SWOT investigation, achievability study, and venture return investigation, which have been utilized to consider the development of the key players working in the market.

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Some of the Points cover in Global Stem Cell Therapy Market Research Report is:

Chapter 1: Overview of Global Stem Cell Therapy Market (2019-2024) Definition Specifications Classification Applications Regions

Chapter 2: Market Competition by Players/Suppliers 2019 and 2024 Manufacturing Cost Structure Raw Material and Suppliers Manufacturing Process Industry Chain Structure. Continued

The main points which are answered and covered in this Report are-

What will be the total Stem Cell Therapy Market in the coming years till 2024?What will be the key factors which will be overall affecting the industry?What are the various challenges addressed?Which are the major companies included?

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As per new Study on Stem Cell Therapy Market 2019 Future Strategy, Analysis and Prediction by Leading Manufacturers, its Application and Types...

Stem Cell Therapy Market Size, Share, Trends, and Opportunity Analysis by 2017 – 2025 – The Market Expedition

Global Stem Cell Therapy Market Analysis

The recent report published by TMRR on the global Stem Cell Therapy market is an in-depth analysis of the overall prospects of the Stem Cell Therapy market in the upcoming years. The data collected from credible primary and secondary sources is accurately represented in the report backed up by relevant figures, graphs, and tables. The report includes a quantitative and qualitative analysis of the various aspects of the market by collecting data from the key participants in the Stem Cell Therapy market value chain.

The report reveals that the global Stem Cell Therapy market is set to grow at a CAGR of ~XX% over the forecast period (2019-2029) and surpass the value of ~US$XX by the end of 2029. The presented study also includes a thorough analysis of the micro and macroeconomic factors, regulatory framework, and current trends that are expected to influence the growth of the Stem Cell Therapy market during the assessment period.

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Vital Information Enclosed in the Stem Cell Therapy Market Report:

Important Queries Addressed in the Report

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Stem Cell Therapy Market Segmentation

The market study put forward by TMRR segments the global Stem Cell Therapy market to offer a microscopic understanding of the various aspects of the Stem Cell Therapy market. The Stem Cell Therapy market is segmented on the basis of region, product type, end-user, and more.

The study offers a Y-o-Y growth projection of each market segment and sub-segment over the stipulated timeframe of the study.

Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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Stem Cell Therapy Market Size, Share, Trends, and Opportunity Analysis by 2017 - 2025 - The Market Expedition

Newly approved sickle cell drug ushers in an era of therapeutic breakthroughs – Worcester Telegram

Terri Booker's worst sickle cell crisis almost killed her.

Her sticky, stiff, crescent-shaped red blood cells formed a blockage in her bloodstream, starving her of oxygen, triggering unbearable pain, and sending her, at 20 years old, into kidney and lung failure.

Now 36, the Philadelphia lawyer is thrilled by the U.S. Food and Drug Administration's recent approval of Novartis' sickle cell drug Adakveo, a monthly intravenous infusion for patients over age 15 that has been shown to dramatically reduce vessel-obstruction crises and hospitalizations.

The new drug, which targets a specific protein involved in cell stickiness, is part of a wave of novel therapies that could transform the outlook for patients with the devastating illness, the most common inherited blood disorder. In February, the FDA is expected to approve Global Blood Therapeutics' daily pill Voxelotor, which treats the underlying cause by helping to prevent red blood cells from deforming.

More than 30 other therapies are in development, including some that may offer a cure by repairing the genetic mutation that hinders hemoglobin's ability to carry oxygen the defect that causes red blood cells to sickle. Just last month, Vertex Pharmaceuticals and CRISPR Therapeutics announced that the first treatment made with genetically edited cells is producing a crucial protein in the inaugural sickle cell patient, four months after her infusion.

"I'm actually excited for the future of sickle cell treatment," Booker said. "Not only treatments but cures are in the pipeline."

That optimism was echoed by Alexis A. Thompson, head of hematology at Lurie Children's Hospital of Chicago.

"For such a challenging disease, we have had so few tools to treat it," said Thompson, past president of the American Society of Hematology. Adakveo "is terribly exciting. It represents not just an additional tool, but one that was designed specifically for sickle cell disease. These new agents moving toward approval address the disease process in a rational way."

An estimated 100,000 Americans, most of them African American, have sickle cell disease. Worldwide, millions are affected.

For decades, doctors could give them nothing more than symptom relief using blood transfusions, painkilling opioids, and hydroxyurea, a drug that increases the hemoglobin in red blood cells. Developed as a cancer drug, hydroxyurea was approved for sickle cell disease in 1998. Almost two decades passed before a second drug, Endari, an amino acid that reduces oxidative stress on deformed cells, was approved in 2016.

Despite limited treatments, improvements in medical care have lengthened patients' lifespan to about 50 years.

"This used to be a pediatric disease because patients didn't survive into adulthood," said David J. Axelrod, the physician who co-directs the sickle cell program at Jefferson University Hospitals. "Now it's more of a chronic disease, so patients are dying from end-stage organ disease."

Living with the disorder is a constant struggle. Zemoria Brandon sees it every day as an administrator and social worker with the Philadelphia-Delaware chapter of the Sickle Cell Disease Association of America, cofounded by her husband, who died in 1998.

"We have children on morphine to relieve their pain," Brandon said. "We've had children who have had strokes."

Like others, Brandon is happy about Adakveo, which cut the rate of obstructed-vessel crises by nearly half to 1.63 episodes a year, compared with 2.98 a year for patients on a placebo.

"But," Brandon added, "we're hoping that a universal cure is on the horizon."

The only proven cure for children with the disease is a stem-cell transplant from a tissue-matched sibling. The risky procedure which destroys the patient's own immune system, then restores it using the donor's cells is an option for only about 10% of children because matched siblings are so rare.

Recently, however, researchers have begun to expand the pool of eligible patients by using less perfectly matched donor stem cells, along with techniques that enrich the transplanted cells and reduce the chances of infection and rejection. Even adults have been successfully treated.

"The toxicity of the transplant used to be too much for adults. There was 100% mortality," said Thompson at Lurie Children's Hospital. "We've identified ways to minimize the risks."

Another promising approach involves gene therapy using an inactivated virus to ferry a corrective gene into the patient's cells. Bluebird bio's experimental gene therapy LentiGlobin adds a gene that can enable patients to make healthy red blood cells, with the goal of replacing sickled cells as they die off. Early results from a small group of patients are encouraging.

Cost, as always, is a looming issue. Novartis said Adakveo will be priced at $2,357 per vial, so a year's treatment would cost between about $84,000 and $113,000.

Booker has personal experience with the cost barrier. She wanted to try Endari, but couldn't afford the $300 monthly co-pay. She applied to Emmaus Medical Inc.'s financial assistance program and recently began taking the company's drug.

"I do keep abreast of research. I need to because sometimes even doctors don't know what's going on," Booker said. "I worry that these new drugs will be too expensive for patients to afford or, worse, they won't know about them."

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Newly approved sickle cell drug ushers in an era of therapeutic breakthroughs - Worcester Telegram