Breakthrough in Gene Editing Comes as Scientists Correct Disease-Causing Mutation in Human Embryo – TrendinTech

Its an exciting time for gene editing currently as scientists achieve the first ever safe repair of a single-gene mutation in a human embryo. Using the CRISPR-Cas9 system, medical professionals used the technique to correct the mutation for a heart condition. The procedure was carried out early enough in the embryonic development so that the defect wouldnt be passed down to further generations.

The study is an important breakthrough and could pave the way for improved in vitro fertilization (IVF) outcomes further down the line as well as finding cures for even a few of the thousands of diseases caused by single gene mutations. Juan Carlos Izpisua Belmonte, a professor in Salks Gene Expression Laboratory and corresponding author of the paper commented that, Gene editing is still in its infancy so even though this preliminary effort was found to be safe and effective, it is crucial that we continue to proceed with the utmost caution, paying the highest attention to ethical considerations. Although gene editing has become relatively easy to carry out for scientists these days, they do still proceed with much caution. This is partly to avoid introducing any mutations into the germ line.

*Hypertrophic cardiomyopathy (HCM) affects around 1 in every 500 people and is the most common cause of sudden death in otherwise healthy, young athletes. A dominant mutation in the MYBPC3 gene is the cause of it and theres a 50 percent chance of a carrier passing it to their offspring. If the mutation in the embryo was corrected it would prevent the disease in children as well as their descendants. During the study, researchers produced induced pluripotent stem cells from a sample taken from a male with HCM. They also developed a CRISPR-Cas9 gene editing strategy that was able to target specifically the mutated copy of the MYBPC3 gene for repair. The MYBPC3 gene was cut by the Cas9 enzyme allowing the mutation to be fixed in the next round of cell division. Researchers then used IVF techniques to inject top gene-editing components into healthy donor eggs which had been newly fertilized. They were then able to monitor just how well the mutation was repaired.

This method proved to be both extremely safe and efficient, much to the surprise of the researchers. No strange mutations were induced and a high number of embryonic cells were repaired. They also developed a way in which they could ensure the repair happened consistently in all of the embryonic cells which is a bonus as spotty repairs have been known to cause mutations. Even though the success rate in patient cells cultured in a dish was low, we saw that the gene correction seems to be very robust in embryos of which one copy of the MYBPC3 gene is mutated, states Salk staff scientists and one of the papers first authors, Jun Wu. Our technology successfully repairs the disease-causing gene mutation by taking advantage of a DNA repair response unique to early embryos. However, it is still early days and both Izpisua Belmonte and Wu agree further research is required to ensure no unintended effects occur.

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Fertile offspring produced from sterile mice using iPS cells – Kyodo News Plus

Researchers at Kyoto University have succeeded in producing fertile offspring from sterile mice with chromosome abnormality by using iPS cells, the university said Friday.

The research outcome by the international team was published online by the U.S. magazine Science with the headline "Fertile offspring from sterile sex chromosome trisomic mice."

The team intentionally produced sterile trisomic mice and showed that fibroblasts from the abnormal mice lose the extra sex chromosome during reprogramming to induced pluripotent stem cells, or iPS cells.

The team termed the phenomenon trisomy-biased chromosome loss.

It is vital to have the correct number of chromosomes for normal development and health.

The researchers have successfully produced fertile offspring with a usual pair of sex chromosome by injecting functional sperm originated from the euploid iPS cells into eggs.

"(The finding) could lead to the development of treatment for infertility caused by chromosome or other genetic abnormalities," said Michinori Saito, a professor of the Graduate School of Medicine at Kyoto University and a research team member.

The team also involves James Turner of the Francis Crick Institute in Britain.

Sex chromosome trisomy, associated with infertility, affects 0.1 percent of the human population, according to the research team.

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Fertile offspring produced from sterile mice using iPS cells - Kyodo News Plus

Brain Spheroids Hatch Mature Astrocytes | ALZFORUM – Alzforum

18 Aug 2017

Astrocytes are more than bystanders in neurotransmissionthey take an active role in synaptic activity. However, their functions are hard to study because the cells are difficult to grow in vitro and its hard to coax them to mature from progenitors. Now, researchers from the labs of Sergiu Paca and Ben Barres, both at Stanford University School of Medicine, California, report that astrocytes come of age in spherical balls of human brain cells cultured in a dish for almost two years. As reported in the August 16 Neuron, these astrocytes develop much like those from real brains, undergoing similar transcriptomic, morphologic, and functional changes. Studying the processes involved in this astrocyte maturation will help researchers understand neurodevelopmental disorders such as autism and schizophrenia, researchers say, and might even shed light on problems in adultbrains.

That these 3D cultures can be maintained for such a long time allows us to capture an interesting transition in astrocytes, said Paca. We are starting to appreciate aspects of human brain development to which we would not otherwise haveaccess.

The breakthrough is that they can develop human astrocytes very close to maturity in their 3D culture models, said Doo Yeon Kim, Massachusetts General Hospital, Charlestown, who uses 3D culture models to study pathological process that occur in Alzheimers disease. Some researchers are using 3D cultures to model other neurodegenerative disorders, such as ALS, and still others are planning to use cultured astrocytes for cell therapy. If astrocytes are not mature enough in culture, patterns [we see] may not be the same as in the diseased brain, saidKim.

This developing human astrocyte (red), which comes from a 350-day-old cortical spheroid, is taking shape as a mature cell. [Image courtesy of Sloan et al.Neuron]

A few years back, Pacas group developed a method for differentiating human induced pluripotent stem cells (hiPSCs) into a 3D culture of brain cells. They used special dishes that the cells could not easily attach to, coaxing them to stick to each other instead. Under these conditions the iPSCs balled up into neural spheroids that grew to about 4 mm in diameter. A cocktail of growth factors early on encouraged them to form excitatory pyramidal cells like those in the cortex, and the cells spontaneously organized into layers. These cortical spheroids survived a year or more and spontaneously grew astrocytes in addition to neurons (Paca et al., 2015). Not long after, the Barres lab reported that astrocytes in the adult human brain look different from those isolated from fetuses. They called the latter astrocyte progenitor cells (APCs). Each had their own transcriptional patterns and functions (Jan 2016 news). Together, Barres and Paca wondered if it was possible to see the APCs morph into mature astrocytes in these long-lived corticalspheroids.

To find out, first author Steven Sloan and colleagues examined spheroids generated from iPSCs derived from healthy human fibroblasts. Sloan grew the spheroids for about 20 months. Along the way, he took samples, isolated the astrocytes, and compared them to those isolated from fetal and postnatal humanbrain.

At about 100 days in culture, astrocytes began to sprout spontaneously from within the mostly neuronal milieu of the cortical spheroids. At first, these cells were simple, adorned by few branches and expressing genes akin to those active in APCs. But as the spheroids reached about 250 days, the astrocytes therein looked more mature, having numerous processes. After this point, APC gene expression tapered off and the astrocytes started producing proteins typical of matureastrocytes.

Astrocytes also underwent functional changes as they matured. Early versions divided in fast and furious fashion, much like their counterparts from the fetal tissue. That division slowed as the spheroids aged. Dividing APCs dropped from 35 percent of all astrocytes at day 167 to 3 percent at day 590. Taken from the spheroids at day 150 and cultured in a 2D layer, immature astrocytes also harbored a voracious appetite for added synaptosomes, much like immature astrocytes recently characterized in mice (see image below; Dec 2013 conference news on Chung et al., 2013). However, that hunger waned as astrocytes approached the 590-daymark.

At the older end of the spectrum, mature astrocytes seemed to take on a supportive role, strengthening calcium signaling in nearbyneurons.

Studying the neurons and astrocytes in these cortical spheroids could be useful for addressing certain unanswered questions about human biology, said other researchers. This could be a very strong opportunity to understand what goes wrong in human genetic disorders that affect astrocyte function, said M. Kerry OBanion, University of Rochester Medical Center, New York. Its also possible that such cultures could reveal as yet unknown facets of familial mutations that cause Alzheimers disease, he suggested. However, given that these cultures take a long time to grow and develop, they are unlikely to completely supplant other types of cultures or faster-maturing animal models, hesaid.

Kim agreed, saying, The results are very exciting, but not practical yet for disease modeling." However, Kim hopes that researchers will make progress on accelerating the maturationprocess.

The Barres and Paca labs are trying just that with the spheroid. They will also analyze what they secrete to support neuronal signaling. In addition, they are exploring how to make the astrocytes reactive, as they often are in neurodegenerative diseases, such as Alzheimers. Doing so might reveal how such astrocytes interact withneurons.

An immature astrocyte taken from a 150-day-old spheroid gobbles up added synaptosomes (red). [Neuron, Sloan et al.2017]

To Pacas knowledge, these cortical spheroids are some of the longest human cell cultures ever reported. His group has continued to cultivate these clumps, with the oldest still going strong at day 850. Granted, these systems are missing many cell types: endothelial cells, oligodendrocytes, and microglia to name a few, he said. However, his lab has introduced new ways to add in other cells. Earlier this year, he reported 3D cultures of cortical glutamatergic neurons and GABAergic interneurons that fused together when they were placed side-by-side (Birey et al., 2017).

Clive Svendsen, Cedars-Sinai Medical Center in Los Angeles, California, saw clinical implications for this paper. It shows iPSC derived astrocytes can mature to an adult phenotype, he said. This further supports their use in clinical transplantation, as we are planning to do. His group has begun a Phase 1 clinical trial that implants human fetal astrocytes into the spinal cords of ALS patients.Gwyneth DickeyZakaib

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Brain Spheroids Hatch Mature Astrocytes | ALZFORUM - Alzforum

How Do We Get Pluripotent Stem Cells? | Boston Children’s …

Pluripotent stem cells can be created in several ways, depending on the type.

Genetic reprogramming (induced pluripotent cells): Several labs, including that of George Q. Daley, MD, PhD, Director of Stem Cell Transplantation Program, have shown that it requires only a handful of genes to reprogram an ordinary cell from the body, such as a skin cell, into whats known as an induced pluripotent cell (iPS cell). Currently, these genes (Oct4, Sox2, Myc, and Klf4) are most commonly brought into the cell using viruses, but there are newer methods that do not use viruses.

Although skin cells are probably the number-one source of iPS cells currently, lines are also being created from blood cells and mesenchymal stem cells (a type of multipotent adult stem cell that gives rise to a variety of connective tissues). Laboratories in the Stem Cell Program at Childrens Hospital Boston are exploring whether iPS lines made from different kinds of patient cells are easier to work with, or can more readily form the particular kind of cell a patient might need for treatment.

Childrens researchers are also continuing to experiment with more efficient programming techniques, so they can get a higher yield of true pluripotent stem cells.

IVF donations of unused/discarded embryos (ES cells): Another major source of pluripotent stem cells for research purposes is unused embryo donated by couples undergoing in vitro fertilization (IVF). Some of these may be poor-quality embryos that would otherwise be discarded. The resulting cells are considered to be true embryonic stem cells (ES cells).

The donated embryos are placed in a media preparation in special dishes and allowed to develop for a few days. At about the fifth day the embryo reaches the blastocyst stage and forms a ball of 100-200 cells. At this stage, ES cells are derived from the blastocysts inner cell mass. In some cases, the ES cells can be isolated even before the blastocyst stage.

To date, Childrens has created more than a dozen new ES cell lines using this approach, which we are now making available to other scientists. These ES cells are not genetically matched to a particular patient, but instead are used to advance our knowledge of how stem cells behave and differentiate.

Some people question the ethics of using discarded IVF embryos for research. For more discussion, see Policy and Ethics.

Somatic cell nuclear transfer: The process called nuclear transfer involves combining a donated human egg with a cell from the body (typically a skin cell) to create a type of embryonic stem cell, sometimes called an ntES cell. Nuclear transfer requires an egg donor.

First, an incredibly thin microscopic needle is used to remove the eggs nucleus, which contains all the eggs genetic material, and replace it with the nucleus from the body cell. The process of transferring the nucleus into the egg reprograms it, reactivating the full set of genes for making all the tissues of the body. How this happens isnt well understood yet, and researchers in the Stem Cell Program at Boston Childrens Hospital are trying to understand it better.

Next, the resulting reprogrammed cell is encouraged to develop and divide in the lab, and by about day five, it forms a blastocyst, a ball of 100-200 cells. The inner cells of the blastocyst are then isolated to create ntES cells.

Of all the techniques for making pluripotent cells, nuclear transfer is the most technically demanding and therefore the least efficient. To date, it has only been successful in lower animals, not in humans. But because the stem cells created would be an exact genetic match to the patient, nuclear transfer may eliminate the tissue matching and tissue rejection problems that are currently a serious obstacle to successful tissue transplantation. For this reason, nuclear transfer is an important area of research at Childrens.

Because ntES cells created from human patients would match them genetically, nuclear transfer is sometimes called therapeutic cloningnot to be confused with the concept of reproductive cloning.

Parthenogenesis (unfertilized eggs): Using a series of chemical treatments, its possible to trick an egg into developing into an embryo without being fertilized by sperm. This process, called parthenogenesis, sometimes happens in nature, allowing many plants and some animals to reproduce without the contribution of a male.

By inducing parthenogenesis artificially, researchers have been able to create parthenogenetic embryonic stem cells, or pES cells, in mice. The embryos created, known as parthenotes, are grown for about five days until they reach the blastocyst stage. Development is then stopped and pES cells were derived from the blastocysts inner core of cells.

Parthenogenesis hasnt been accomplished in human eggs yet, at least not by choice (a Korean team is thought to have created human pES cells accidentally in 2007). But researchers at Childrens are trying to do so, since pES cells, if carefully typed genetically, could potentially be used to create master banks of pluripotent stem cells. Doctors could then choose a cell line thats genetically compatible with the patients immune system. (For details, see How do pluripotent stem cells get turned into treatments?).

Of more immediate concern is the possibility that parthenogenesis could be used to make pES cells for the egg donor herself or a sibling. However, before using these cells in patients, researchers need to know more about the safety of this approach.

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How Do We Get Pluripotent Stem Cells? | Boston Children's ...

Induced Pluripotent Stem Cells in Global Effort to …

Since the discovery of induced pluripotent stem cell (iPSC) technology more than a decade ago, it has become an important part of the life sciences industry. In the past five years, the global market for iPSCs has grown rapidly. This pace is projected to continue over the next several years to reach $3.6 billion, according to a new forecast by BCC Research. By 2021, the major contributor will be the drug development and toxicity testing segment, which will account for approximately 61% of the total market. This will be followed by the academic research (24%) and regenerative medicine (15%) sectors.

GLOBAL MARKET SHARES FOR INDUCED PLURIPOTENT STEM CELL PRODUCTS BY APPLICATION, 2021

Drug development and toxicity testing are currently the major source of revenue. However, the market for regenerative medicine is the fastest growing. The segment of iPSC applications in translational medicine research is also expanding quickly. iPSC technology has improved the drug discovery process and, in particular, has been instrumental in promoting precision medicine and the development of personalized drugs and diagnostic tests. The iPSC technology platform is being exploited for research applications in order to supply products and services to the academic, public research, and life science industry sectors.

HOW IT WORKS

In 2006, researchers at Kyoto University in Japan identified conditions that would allow specialized adult cells to be genetically reprogrammed to assume a stem celllike state. These adult cells, called induced pluripotent stem cells (iPSCs), were reprogrammed to an embryonic stem celllike state by introducing genes important for maintaining the essential properties of embryonic stem cells. Researchers have rapidly improved the techniques to generate iPSCs, creating a powerful new way to de-differentiate cells for which developmental fates had been previously assumed to be determined. NIH Stem Cell Information Home Page

The clinical market for iPSCs is still nascent. BCC Research predicts a hefty 11.6% five-year compound annual growth rate (CAGR) for this market as it comes of age, with developments such as the establishment of the start-up BlueRock Therapeutics by Bayer AG and Versant Ventures in December 2016. In 2014, the first human iPSC clinical trial began, which yielded encouraging results published earlier this year about the first patient with macular degeneration to be treated with sheets of retinal pigmented epithelial cells made from iPSCs [The New England Journal of Medicine, 3/16]. The iPSC clinical research and services market is expected to maintain its rapid growth over the next few years.

In its recent report, Induced Pluripotent Stem Cells: Global Markets, BCC Research made several key observations. The pharmaceutical industry needs better cell sources, such as iPSC-derived functional cells, for drug toxicity testing and screening. In the United States, the Food and Drug Administration (FDA) has been authorized to provide orphan drug designations for many of the therapies being developed for rare diseases, such as Parkinsons and Huntingtons, using stem cells. Also, iPSC technology is developing into a platform for precision medicine, which is experiencing rapid growth globally.

In Australia, scientists created brain-like tissue in the lab using a 3D printer and special bio-ink made from iPSCs. After the bio-ink was printed into a 3D scaffold, the stem cells turned into nerve cells found in the brain. The research moves toward being able to make replacement brain tissue derived from a patients own skin or blood cells to help treat conditions such as brain injury, Parkinsons disease, epilepsy, and schizophrenia. Jeremy Crook, from the University of Wollongong and ARC Centre of Excellence for Electromaterials Science, said the ability to customize brain tissue from a persons own body tissue circumvents issues of immune rejection, which is common in organ transplantation. It's personalized medicine.

The research team, whose work was published in the journal Advanced Healthcare Materials, used 3D printing to make neurons involved in producing GABA and serotonin, as well as support cells called neuroglia. In the future, they plan to print neurons that produce dopamine, deficiencies of which are linked to Parkinsons disease. We might want to make a tissue that specifically generates that neurotransmitter for grafting into the brain of a Parkinsons patient, said Dr. Crook. [ABC Science, 7/26]

Rapid growth in medical tourism and contract research outsourcing are among factors driving the stem cell market in the Asia-Pacific region. BCC Research forecasts this to be the fastest-growing geographic segment, with a five-year CAGR of 13.7%. Corporate Japan has lagged its Western rivals in stem cell commercialization efforts. Now, Japanese companies are joining the search for ways to commercialize iPSCs, spurred in part by recent legislation designed to fast track such forms of regenerative medicine.

Daiichi Sankyo announced that it will seek to commercialize sheets of heart muscle tissue derived from iPSCs as a treatment for heart disease. The company is investing an undisclosed amount in the Osaka University spin-off Cuorips, which developed the sheets of myocardial cells. The idea is to grow the sheets and graft them onto the heart to help it beat properly. This would give patients with severe heart failure an alternative to a transplant or an artificial heart. Daiichi Sankyo will conduct clinical trials in cooperation with doctors at Osaka University and work to develop a way to mass produce the sheets of myocardial tissue.

The same day, Megakaryon announced that it has developed a way to mass produce blood platelets derived from iPSCs. It has been working on this project in collaboration with 15 companies, including Otsuka Holdings and Sysmex, as a way to address the shortage of blood for transfusions. The goal is to gain regulatory approval in 2020. [Nikkei Asian Review, August 8]

The first iPSC clinical products will most likely enter the market in the next few years, says BCC Research analyst Mike Fan. Regarding therapeutic solutions for diseases without ethical issues, a series of technical breakthroughs have been made in recent years for improving cellular reprogramming, differentiation, and large-scale production of Good Manufacturing Practice (GMP)grade iPSCs and their derivatives for clinical use.

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Induced Pluripotent Stem Cells: Global Markets Report 2017-2021

DUBLIN, July 6, 2017 /PRNewswire/ --

Research and Markets has announced the addition of the "Induced Pluripotent Stem Cells: Global Markets" report to their offering.

The Global Market for Induced Pluripotent Stem Cells (iPSCs) should reach $3.6 Billion in 2021, Increasing at a CAGR of 11.6% from 2016 through 2021

This study is focused on the market side of iPSCs rather than its technical side. Different market segments for this emerging market are covered.

For example, application-based market segments include academic research, drug development and toxicity testing, and regenerative medicine; product function-based market segments include molecular and cellular engineering, cellular reprogramming, cell culture, cell differentiation and cell analysis; iPSC-derived cell-type-based market segments include cardiomyocytes, hepatocytes, neurons, endothelia cells and other cell types; geography-based market segments include the U.S., Europe, Asia-Pacific and Rest of World. Research and market trends are also analyzed by studying the funding, patent publications and research publications in the field.

Key Topics Covered:

1: Introduction

2: Summary and Highlights

3: Overview

4: Induced Pluripotent Stem Cell Applications

5: Induced Pluripotent Stem Cell Market Segmentation and Forecast

6: Induced Pluripotent Stem Cell Research Application Market

7: Drug Discovery and Development Market

8: Induced Pluripotent Stem Cell Contract Service Market

9: Induced Pluripotent Stem Cell Clinical Application Market

10: Research Market Trend Analysis

11: Clinical Application Market Trend Analysis

12: Company Profiles

13: Conclusions

For more information about this report visit https://www.researchandmarkets.com/research/3ns6k3/induced

Media Contact:

Laura Wood, Senior Manager press@researchandmarkets.com

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716

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Induced Pluripotent Stem Cells: Global Markets Report 2017-2021

Global Induced Pluripotent Stem Cells Market: HTF Market

Table of Contents

Global Induced Pluripotent Stem Cells Market Size, Status and Forecast 2022

1 Industry Overview of Induced Pluripotent Stem Cells

1.1 Induced Pluripotent Stem Cells Market Overview

1.1.1 Induced Pluripotent Stem Cells Product Scope

1.1.2 Market Status and Outlook

1.2 Global Induced Pluripotent Stem Cells Market Size and Analysis by Regions

1.2.1 United States

1.2.2 EU

1.2.3 Japan

1.2.4 China

1.2.5 India

1.2.6 Southeast Asia

1.3 Induced Pluripotent Stem Cells Market by Type

1.3.1 Hepatocytes

1.3.2 Fibroblasts

1.3.3 Keratinocytes

1.3.4 Amniotic Cells

1.4 Induced Pluripotent Stem Cells Market by End Users/Application

1.4.1 Academic Research

1.4.2 Toxicity Screening

1.4.3 Regenerative Medicine

1.4.4 Drug Development and Discovery

2 Global Induced Pluripotent Stem Cells Competition Analysis by Players

2.1 Induced Pluripotent Stem Cells Market Size (Value) by Players (2016 and 2017)

2.2 Competitive Status and Trend

2.2.1 Market Concentration Rate

2.2.2 Product/Service Differences

2.2.3 New Entrants

2.2.4 The Technology Trends in Future

3 Company (Top Players) Profiles

3.1 Bristol-Myers Squibb Company

3.1.1 Company Profile

3.1.2 Main Business/Business Overview

3.1.3 Products, Services and Solutions

3.1.4 Induced Pluripotent Stem Cells Revenue (Value) (2012-2017)

3.1.5 Recent Developments

3.2 Fujifilm Holding Corporation

3.2.1 Company Profile

3.2.2 Main Business/Business Overview

3.2.3 Products, Services and Solutions

3.2.4 Induced Pluripotent Stem Cells Revenue (Value) (2012-2017)

3.2.5 Recent Developments

3.3 Astellas Pharma Inc.

3.3.1 Company Profile

3.3.2 Main Business/Business Overview

3.3.3 Products, Services and Solutions

3.3.4 Induced Pluripotent Stem Cells Revenue (Value) (2012-2017)

3.3.5 Recent Developments

3.4 Fate Therapeutics, Inc.

3.4.1 Company Profile

3.4.2 Main Business/Business Overview

3.4.3 Products, Services and Solutions

3.4.4 Induced Pluripotent Stem Cells Revenue (Value) (2012-2017)

3.4.5 Recent Developments

3.5 Aastrom Biosciences, Inc.

3.5.1 Company Profile

3.5.2 Main Business/Business Overview

3.5.3 Products, Services and Solutions

3.5.4 Induced Pluripotent Stem Cells Revenue (Value) (2012-2017)

3.5.5 Recent Developments

3.6 ViaCyte, Inc.

3.6.1 Company Profile

3.6.2 Main Business/Business Overview

3.6.3 Products, Services and Solutions

3.6.4 Induced Pluripotent Stem Cells Revenue (Value) (2012-2017)

3.6.5 Recent Developments

3.7 Celgene Corporation

3.7.1 Company Profile

3.7.2 Main Business/Business Overview

3.7.3 Products, Services and Solutions

3.7.4 Induced Pluripotent Stem Cells Revenue (Value) (2012-2017)

3.7.5 Recent Developments

3.8 Japan Tissue Engineering Co., Ltd.

3.8.1 Company Profile

3.8.2 Main Business/Business Overview

3.8.3 Products, Services and Solutions

3.8.4 Induced Pluripotent Stem Cells Revenue (Value) (2012-2017)

3.8.5 Recent Developments

3.9 Organogenesis Inc.

3.9.1 Company Profile

3.9.2 Main Business/Business Overview

3.9.3 Products, Services and Solutions

3.9.4 Induced Pluripotent Stem Cells Revenue (Value) (2012-2017)

3.9.5 Recent Developments

3.10 Acelity Holdings, Inc.

3.10.1 Company Profile

3.10.2 Main Business/Business Overview

3.10.3 Products, Services and Solutions

3.10.4 Induced Pluripotent Stem Cells Revenue (Value) (2012-2017)

3.10.5 Recent Developments

3.11 StemCells, Inc.

4 Global Induced Pluripotent Stem Cells Market Size by Type and Application (2012-2017)

4.1 Global Induced Pluripotent Stem Cells Market Size by Type (2012-2017)

4.2 Global Induced Pluripotent Stem Cells Market Size by Application (2012-2017)

4.3 Potential Application of Induced Pluripotent Stem Cells in Future

4.4 Top Consumer/End Users of Induced Pluripotent Stem Cells

5 United States Induced Pluripotent Stem Cells Development Status and Outlook

5.1 United States Induced Pluripotent Stem Cells Market Size (2012-2017)

5.2 United States Induced Pluripotent Stem Cells Market Size and Market Share by Players (2016 and 2017)

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Global Induced Pluripotent Stem Cells Market: HTF Market

MESO-BRAIN initiative receives 3.3million to replicate brain’s neural networks through 3D nanoprinting – Cordis News

The MESO-BRAIN consortium has received a prestigious award of 3.3million in funding from the European Commission as part of its Future and Emerging Technology (FET) scheme. The project aims to develop three-dimensional (3D) human neural networks with specific biological architecture, and the inherent ability to interrogate the networks brain-like activity both electrophysiologically and optically.

The MESO-BRAIN projects cornerstone will use human induced pluripotent stem cells (iPSCs) that have been differentiated into neurons upon a defined and reproducible 3D scaffold to support the development of human neural networks that emulate brain activity. The structure will be based on a brain cortical module and will be unique in that it will be designed and produced using nanoscale 3D-laser-printed structures incorporating nano-electrodes to enable downstream electrophysiological analysis of neural network function. Optical analysis will be conducted using cutting-edge light sheet-based, fast volumetric imaging technology to enable cellular resolution throughout the 3D network. The MESO-BRAIN project will allow for a comprehensive and detailed investigation of neural network development in health and disease.

The MESO-BRAIN project will launch in September 2016 and research will be conducted over three years.

The MESO-BRAIN initiative targets a transformative progress in photonics, neuroscience and medicine. The project aims to develop human induced pluripotent stem cell (iPSC)-derived neural networks upon a defined and reproducible 3D scaffold to emulate brain activity and improve our understanding and treatment of conditions such as Parkinsons disease, dementia and trauma. This research, led by Aston University, is a collaboration between Axol Bioscience Ltd. (UK), Laser Zentrum Hannover (Germany), University of Barcelona (Spain), Institute of Photonic Sciences (Spain) and KITE Innovation (UK). The project is funded by the European Commission through its Future and Emerging Technology (Open) programme.

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MESO-BRAIN initiative receives 3.3million to replicate brain's neural networks through 3D nanoprinting - Cordis News

induced pluripotent stem cell (iPS cell) | biology …

Alternative Title: iPS cell

Induced pluripotent stem cell (iPS cell), immature cell that is generated from an adult (mature) cell and that has regained the capacity to differentiate into any type of cell in the body. Induced pluripotent stem cells (iPS cells) differ from embryonic stem cells (ES cells), which form the inner cell mass of an embryo but also are pluripotent, eventually giving rise to all the cell types that make up the body. Induced pluripotent cells were first described in 2006 by Japanese physician and researcher Shinya Yamanaka and colleagues. The first experiments were performed by using mouse cells. The following year, however, Yamanaka successfully derived iPS cells from human adult fibroblast cells. Until that time, human stem cells could be obtained only by isolating them from early human embryos. Hence, an important feature of iPS cells is that their generation does not require an embryo, the use of which is fraught with ethical issues.

The generation of iPS cells from somatic cells (fully differentiated adult cells, excluding germ cells) was based on the idea that any cell in the body can be reprogrammed to a more primitive (stemlike) state. Among the first to discover that possibility was British developmental biologist John B. Gurdon, who in the late 1950s had shown in frogs that egg cells are able to reprogram differentiated cell nuclei. Gurdon used a technique known as somatic cell nuclear transfer (SCNT), in which the nucleus of a somatic cell is transferred into the cytoplasm of an enucleated egg (an egg that has had its nucleus removed). In 1996 British developmental biologist Ian Wilmut and colleagues used SCNT to create Dolly the sheep, the first clone of an adult mammal. The experiments with SCNT were crucial to the eventual production of iPS cells. Indeed, by the time of Dollys creation, it was widely accepted that factors in the egg cytoplasm were responsible for reprogramming differentiated cell nuclei. The factors controlling the process were unknown, however, until Yamanaka published his first report describing iPS cell generation. (Yamanaka and Gurdon shared the 2012 Nobel Prize for Physiology or Medicine for their discoveries.)

Several proteins have been identified that are capable of inducing or enhancing pluripotency in nonpluripotent (i.e., adult) cells. Of key importance are the transcription factors Oct-4 (octamer 4) and Sox-2 (sex-determining region Y box 2), which maintain stem cells in a primitive state. Other proteins that may be used to enhance pluripotency include Klf-4 (Kruppel-like factor 4), Nanog, and Glis1 (Glis family zinc finger 1).

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stem cell: Induced pluripotent stem cells

Due to the ethical and moral issues surrounding the use of embryonic stem cells, scientists have searched for ways to reprogram adult somatic cells. Studies of cell fusion, in which differentiated adult somatic cells grown in culture with embryonic stem cells fuse with the stem cells and acquire embryonic stem-cell-like properties, led to the idea that specific genes could reprogram...

Pluripotency factors can be introduced into nonpluripotent cells in different ways, such as by plasmids or delivery as proteins or modified RNAs. Among the most effective and widely used methods, however, is delivery via a retroviral vector. Retroviral vectors can readily enter cells, making the genes they carry accessible to the cell; other retroviral activities are silenced. However, because retroviruses integrate into the nuclear genome, their use raises the risk of virus-induced tumour formation. Nonetheless, retroviral delivery remains highly effective, and technical advances to prevent the integration of retroviral material into the nuclear genome have allowed for the generation of iPS cells via ectopic expression (in the cytoplasm) of retrovirus-delivered transcription factors. Ectopic expression also has been achieved with the use of recombinant adeno-associated virus.

Since the initial development of iPS cells, researchers have been working to improve the techniques and to learn what drives pluripotent stem cells to differentiate in particular ways. They also have been investigating the use of iPS cells in the treatment of certain diseases. Of significance is the potential to create patient-specific iPS cells (using a patients own adult cells), which could allow for the generation of perfectly matched cells and tissues for transplantation therapies. Such therapies could help overcome the risk of immune rejection, which is a major challenge in regenerative medicine.

an undifferentiated cell that can divide to produce some offspring cells that continue as stem cells and some cells that are destined to differentiate (become specialized). Stem cells are an ongoing source of the differentiated cells that make up the tissues and organs of animals and plants. There...

...in animals. This is primarily because of the technical challenges and ethical controversy arising from the procuring of human eggs solely for research purposes. In addition, the development of induced pluripotent stem cells, which are derived from somatic cells that have been reprogrammed to an embryonic state through the introduction of specific genetic factors into the cell nuclei, has...

...into pluripotent stem cells. Examples of these factors include Oct-4 (octamer 4), Sox-2 (sex-determining region Y box 2), Klf-4 (Kruppel-like factor 4), and Nanog. Reprogrammed adult cells, known as induced pluripotent stem (iPS) cells, are potential autogeneic sources for cell transplantation and bioartificial tissue construction. Such cells have since been created from the skin cells of...

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induced pluripotent stem cell (iPS cell) | biology ...

Artificial Blood Vessels Mimic Rare Accelerated Aging Disease – Duke Today

Biomedical engineers have grown miniature human blood vessels that exhibit many of the symptoms and drug reactions associated with Hutchinson-Gilford Progeria Syndromean extremely rare genetic disease that causes symptoms resembling accelerated aging in children.

The technology will help doctors and researchers screen potential therapeutics for the disease more rapidly, with the goal of eventually creating a platform for personalized screening. The technique also offers a new way to study other rare diseases and could provide insights into treating heart disease in the elderly.

The study was published online on August 15 in the journal Scientific Reports.

"One of the drugs currently prescribed for this disease extends patients' lives by three months, and that's been considered a major feat," said Leigh Atchison, a doctoral candidate in biomedical engineering at Duke University and first author of the study. "They're looking for anything that will extend lifespan by even a few months. It's that devastating."

Hutchinson-Gilford Progeria Syndromeor simply progeria for shortis a non-hereditary genetic disease caused by a single-point mutation in the genome. It is so rare and so deadly that there are currently only about 250 known cases worldwide.

Progeria is triggered by a defective protein called progerin that accumulates outside of a cell's nucleus rather than becoming part of its structural support system. This causes the nucleus to take on an abnormal shape and inhibits its ability to divide. The resulting symptoms look much like accelerated aging, and affected patients usually die of heart disease brought on by weakened blood vessels before the age of 14.

"Progeria isn't considered hereditary, because nobody lives long enough to pass it on," said George Truskey, the R. Eugene and Susie E. Goodson Professor of Biomedical Engineering at Duke. "There are currently 75 children in clinical trials, which is amazing given the rarity of the disease. But with 15 compounds under consideration for trials, the math just ultimately won't work out. You can't try all of these drugs or various combinations of them in humans, so we're hoping our platform will provide an alternative way to test them."

Blood vessels are difficult to simulate because their walls have multiple layers of cells, including the endothelium and the media. The endothelium is the innermost lining of all blood vessels that interacts with circulating blood. The media is made mostly of smooth muscle cells that help control the flow and pressure of the blood within.

Researchers believe that it is the deterioration of these smooth muscle cells that ultimately leads to the heart disease and failure that so often kills patients with progeria. But because there are so few people with progeria, it is extremely difficult to study in the patients themselves.

"Because it's such a hard disease to study, we wanted to see if we could create a platform using human cells that more accurately represents the disease and then use it for drug testing," said Atchison. "So we tried to grow miniature artificial blood vessels using induced pluripotent stem cells derived from cells taken from patients with progeria."

The plan worked. In just four weeks of growth, the engineered blood vessels exhibit many of the symptoms seen in people with the diseasesymptoms that simple cell cultures have not been able to recreate. The blood vessels also respond similarly to pharmaceuticals, revealing nuances into how current therapies are working.

While the blood vessels showed improved function after a week of being dosed with an analogue of rapamycin, a drug known as everolimus, calcification and other symptoms of cardiovascular disease remained. This implies that the drug is helping the smooth muscle cells work better, but not remedying the underlying symptoms.

"That's why our system could be so useful," said Atchison. "It could tell us exactly what the drug is doing in a quicker, more high-throughput manner, and whether we need a second treatment to address other aspects of the disease."

The success may aid the study of other rare diseases, too.

"The major thing we're happy with is that this serves as a proof of principle for creating a vascular model of a rare disease in the laboratory to better understand it and hopefully develop a therapy," said Truskey.

The research may also provide insight into why some elderly people become especially prone to heart disease. Many heart patients have shown the same buildup of the progerin protein, so researchers believe there may be a link between the two conditions.

There are, of course, limitations to the new artificial blood vessels. They are not connected to any outside organs, nor are they embedded in the complicated biology of a living human being.

"We only created smooth muscle cells from progeria patients in this study, but their endothelial cells might play a major role as well," said Atchison. "If we can incorporate endothelial cells derived from the patients' own cells into the model as well, then we can create a more personalized testing platform for these patients."

This research was supported by the National Institutes of Health (UH3TR000505, R01HL126784), the National Science Foundation (GRFP Grant #1106401), the Maryland Stem Cell Research Fund, and the Progeria Research Foundation.

CITATION: "A Tissue Engineered Blood Vessel Model of Hutchinson-Gilford Progeria Syndrome Using Human iPSC-derived Smooth Muscle Cells," Leigh Atchison, Haoyue Zhang, Kan Cao, George A. Truskey. Scientific Reports, Aug. 15, 2017. DOI: 10.1038/s41598-017-08632-4

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Artificial Blood Vessels Mimic Rare Accelerated Aging Disease - Duke Today