Stem Cell Clinic

‘Heart-on-a-Chip’ For Safer Cancer Treatment – Newswise

By Stem Cell Medicine

Newswise LOS ANGELES (Feb. 1, 2024) --Chemotherapy can be toxic to heart cells. To help protect the hearts of cancer patients, Cedars-Sinai investigators have created a three-dimensional heart-on-a-chip to evaluate drug safety. In a study published in the peer-reviewed journalLab on a Chip,they show that the heart-on-a-chip, created using stem cells, accurately predicts the effects of drugs on human heart cells.

The investigators worked with induced pluripotent stem cells, which are blood cells that have been reprogrammed into stem cells and can be turned into any cell type in the body. They used the stem cells to create two types of heart cells, but instead of placing them all together in an unstructured cell culture dish, as is usually done in heart toxicity testing, the investigators introduced the cells into specialized chips.

The 3D chips feature two channels that are arranged to cross each other, keeping each cell type separate but allowing them to interact. The chips also allow for movement and the introduction of fluids.

We grew heart muscle cells and blood vessel cells, saidArun Sharma, PhD, a research scientist in theBoard of Governors Regenerative Medicine Institute,Smidt Heart Institute,Cedars-Sinai Cancer, and theDepartment of Biomedical Sciencesat Cedars-Sinai and senior author of the study. The chip allows us to stretch the cells back and forth to mimic a heartbeat, and to introduce fluid to mimic the flow of blood through the heart. Its like giving the cells a workout that strengthens the muscle cells and allows the vessel cells to form mini blood vessel-like structures.

These matured cells provide a better test platform for drug toxicity studies than cells that havent undergone this maturation process because they more closely resemble the way adult heart cells function, Sharma said.

To demonstrate the proficiency of heart-on-a-chip as a drug-testing platform, the research team, including lead author and postdoctoral fellowMaedeh Mozneb, PhD, subjected the heart chip to a chemotherapy drug called a VEGFR/PDGFR-inhibiting tyrosine kinase inhibitor, which is known to have adverse effects on heart muscle and blood vessel cells. Damage was observed to both cell types in the heart chips.

If future studies continue to show good results, heart-on-a-chip technology could significantly reduce drug development costs and improve the rate at which new therapies become available.

Another future possibility for these heart chips is the creation of patient-specific chips to personalize cancer treatment.

If a patient with cancer might receive a treatment that could have adverse effects on their heart, we can create induced pluripotent stem cells from a small sample of their blood, Sharma said. We can turn those stem cells into heart muscle and blood vessel cells and put them on a chip that will serve as a personalized avatar for how that persons heart might react to the treatment. This is one of the most exciting applications of this technology, truly advancing personalized medicine at Cedars-Sinai.

Also a possibility: Taking these chips beyond a single organ.

We eventually hope to bring our various organ models together, saidClive Svendsen, PhD, executive director of the Board of Governors Regenerative Medicine Institute and co-author of the study. My lab has beenfine-tuning this technologyto help us develop models of neurological diseases, and other labs in our institute have been working on chips for the liver and the gut microbiome. Bringing all of these chips together to create a patient-on-a-chip model is part of our long-term vision for precision medicine.

Funding: This project was supported by American Heart Association Career Development Award 856987; National Institutes of Health grant numbers T32 HL116273, 1UG3TR003148, and 5UG3NS105703; Allen Distinguished Investigator Award number 12879; the Cedars-Sinai Board of Governors Regenerative Medicine Institute; and The ALS Association.

Read More from Discoveries:Regenerative MedicineA New Path for ALS Treatment

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'Heart-on-a-Chip' For Safer Cancer Treatment - Newswise

Rebirth Clinics Launches State-of-the-Art Stem Cell Centers in Abu Dhabi and Dubai – PR Web

By Stem Cell Medicine

Leading Regenerative Medicine, Rebirth Clinics Brings Innovative Stem Cell and Anti-Aging Therapies to the UAE

HONG KONG, Feb. 1, 2024 /PRNewswire-PRWeb/ -- Rebirth Clinics, a global leader in regenerative medicine products and services, today announced the launch of two of its "Rebirth Clinics" in Abu Dhabi and Dubai. The launch enables them to meet increasing market demand for stem cell treatments and Anti-Aging Therapies.

In addition to offering the highest-grade stem cell therapies available on the marketFour days old xeno-free stem cells sourced from fresh umbilical cord tissueRebirth Clinics also provide regenerative therapies including NAD+, Brain Peptides, Multi-Vitamins, and more.

"We are living in a historic time, where we are witnessing the emergence of stem cell therapy as a rapidly growing modality that will soon become a standard of care in modern medicine," said Ronny Shany, Founder of Rebirth Clinics, "Science has given us an amazing gift with stem cells; they are providing us with answers to medical challenges that have remained beyond our grasp for too long. At Rebirth, we fully embrace this gift, as there are so many diseases that can now be cured, and so many people suffering who can now be helped, it's good business and it's good medicine."

"We are thrilled to announce the expansion of Rebirth Clinics into Abu Dhabi," said Dr. Nashwa Al Ruwaini, CEO of Pyramedia Group, "This venture not only aligns with our commitment to healthcare innovation but also marks a significant step in making advanced regenerative medicine accessible in the region. Our collaboration with Rebirth Clinics LTD. is a testament to our dedication to bringing the latest in stem cell therapies and anti-aging treatments to our community. We envision a future where cutting-edge treatments like umbilical cord stem cell therapies become a cornerstone in modern healthcare. However, it's important to acknowledge that these forward-looking statements are based on current market trends and scientific advancements, and as the medical landscape evolves, so too may our strategies. We are committed to adapting and growing in this dynamic field".

"We are proud to be at the forefront of introducing revolutionary stem cell therapies to our patients", added Dr. Mohamed Al Ruwaini, Director of Delma Medical Center, "This collaboration with Rebirth Clinics in Abu Dhabi signifies a major leap in our mission to offer the most advanced medical treatments available. By integrating Rebirth Clinics' expertise in stem cell therapy, including their groundbreaking umbilical cord stem cell treatments, we are set to redefine healthcare standards in the region. Our commitment to excellence remains steadfast, even as we adapt to new developments and challenges in this pioneering area of healthcare".

Rebirth Clinics is a groundbreaking medical institute dedicated to transforming the landscape of advanced medical rejuvenation therapies using fresh Umbilical cord stem cells and IV treatments. As a leader in the regenerative medicine and the cellular therapy industry, Rebirth specializes in physician training certification, stem cell products, and IV therapies, with its lead product, fresh Umbilical cord stem cells. In addition, the organization is involved with the creation and management of stem cell clinics around the world.

"The opportunity to be a part of history at a time when we are witnessing an incredible paradigm shift in the use of stem cell therapy is monumental," said Derek Halpern, Chief Marketing Officer of Rebirth Clinics. "All advancements in science require time. By diversifying our regenerative medicine offerings, we are moving through this very important time in medicine while simultaneously making sure our customers continue to have a variety of safe and effective options for their care that will empower them to choose the product and/or service they feel is right for them."

For more information about Rebirth Cinics, visit their website at http://www.Rebirthglobe.com.

Media Contact

Derek Halpern, CMO, Rebirth Clinics, +1-5162346564, derek@svnetworx.com,http://Www.Rebirthglobe.com

SOURCE Rebirth Clinics

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Rebirth Clinics Launches State-of-the-Art Stem Cell Centers in Abu Dhabi and Dubai - PR Web

Houston regenerative medicine company to IPO, move toward more human trials – InnovationMap

By Stem Cell Medicine

Want a piece of one of Houstons most promising biotech companies? On January 31, FibroBiologics will begin the trading of its common stock on the Nasdaq stock exchange.

While most labs in the realm of regenerative medicine are focused on stem cells, FibroBiologics has bet on fibroblasts as the secret to treating myriad ailments. Fibroblasts, the most common type of cell in the body, are the primary cells that compose connective tissue.

Interested investors can find a prospectus to peruse before taking the leap. FibroBiologics filed with the U.S. Securities & Exchange Commission (SEC) on November 7, 2023. In September, FibroBiologics CEO Pete OHeeron told InnovationMap, I think what we're going to see is that fibroblasts are going to end up winning... They're just a better overall cell than the stem cells.

OHeeron was first exposed to the possibilities of fibroblasts as a means of regrowing discs in the spine. Since starting the company in 2008 as SpinalCyte, OHeeron and FibroBiologics have organically written and filed more than 320 patents. Potential treatments go far beyond spinal surgery to include wound care, cancer, and multiple sclerosis.

According to OHeeron, the goal in going public is to raise capital for human trials.

Weve had really fantastic results with animals and now were ready for humans, he explained in September. We've done small human trials, but we haven't done the large ones that are going to get the commercialization approval from the FDA.

FibroBiologics is growing with impressive speed. OHeeron told us that he is hiring as quickly as he is able to find qualified scientists with the expertise to do the one-of-a-kind work required. The company opened a new lab last fall at the UH Technology Bridge, Newlin-Linscomb Lab for Cell Therapies. With its new status as a publicly traded company, FibroBiologics is primed to break even more ground.

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Houston regenerative medicine company to IPO, move toward more human trials - InnovationMap

It’s been a struggle getting out of bed – Liam Harrison on the sheer difficulty of living through injuries before stem cell … – Sportskeeda

By Stem Cell Medicine

Combat sports is often a cruel mistress, and Liam Harrison knows that price all too well.

Behind his world titles, accolades, and overall praise, Harrison also had to deal with the physical toll of achieving such greatness.

In an interview with the South China Morning Post, the British Muay Thai legend discussed how his daily life became a constant struggle after he put his body through immense damage throughout his entire career.

Harrison practically lived on one leg after he suffered a nasty knee injury during his world title fight against Thai legend Nong-O Hama at ONE on Prime Video 1.

Apart from a shattered left knee, Liam Harrison also dealt with the lingering pain that he went through before he challenged for the ONE bantamweight Muay Thai world title in August 2022.

He said:

Things only got better for Harrison when he underwent stem cell treatment in Costa Rica earlier this year, and it was a lofty price to keep his body from breaking down even before he reached 40 years old.

Harrison has since returned to training and is looking forward to what could be his final fight in ONE Championship.

Watch Harrison's entire interview below:

Liam Harrison has no apprehensions about his impending retirement, and he already named the fighter he wants to share the ring with for the final time in his career.

In the same interview, Harrison expressed his desire to take on Seksan Or Kwanmuang for his retirement bout:

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It's been a struggle getting out of bed - Liam Harrison on the sheer difficulty of living through injuries before stem cell ... - Sportskeeda

Engineered cartilage and osteoarthritis – Boston Children’s Answers – Boston Children’s Discoveries

By Induced Pluripotent Stem Cells

About one in seven adults live with degenerative joint disease, also known as osteoarthritis (OA). In recent years, as anterior cruciate ligament (ACL) injury and other joint injuries have become more common among adolescent athletes, a growing number of 20- and 30-somethings have joined the ranks of aging baby boomers living with chronic OA pain.

Key takeaways

Treatments for degenerative joint disease are limited, largely because the cartilage that protects the joints doesnt regenerate after birth. Without a way to stimulate regrowth of damaged cartilage, most treatments focus on managing symptoms. And with few curative treatment options, OA remains one of the leading causes of pain and disability in the United States.

Boston Childrens researcher April Craft, PhD, and her team want to change that. Their approach: grow cartilage in the lab that could be used to replace damaged articular tissues in patients joints.

The team first set out to understand how cartilage and joint tissues develop naturally and how stem cells differentiate into cartilage cells, or chondrocytes. The next step was to replicate that process in the lab, putting cells through the same stages of development.

In a study published this year in BMJ, members of the Craft Lab described their approach for generating cartilage from induced pluripotent stem cells (iPSC). Derived from patients own cells, iPSCs can give rise to virtually any type of cell in the body, including chondrocytes. The team generated cartilage-like tissues from two patients with progressive pseudorheumatoid arthropathy of childhood (PPAC), a genetic condition that causes severe premature joint degeneration.

We chose to study PPAC because joint degeneration in this condition progresses rapidly toward a state that is indistinguishable from end-stage OA, says Craft. Our iPSC model of PPAC cartilage will help us learn about this devastating disease. Their findings may possibly apply more broadly to OA from acute injuries or chronic overuse, as well as provide the basis for future therapeutics development.

Using cartilage engineered in the Craft Lab, the team has successfully repaired damaged joint tissues in rats and is preparing to test the procedure in large animals.

Because joint-lining cartilage is avascular and the implanted chondrocytes will be encased by the cartilage tissue itself, there is a reduced likelihood of implant rejection. Because of this, Craft believes that someday off-the-shelf cartilage for human patients could be created using one cell line. If so, live cartilage tissues could be produced, stored, and delivered to surgical teams as needed to replace damaged cartilage.

In some ways, the procedure resembles the most advanced cell therapy for cartilage: autologous chondrocyte implantation. In this two-procedure process, chondrocytes are harvested from one area of the body, expanded in number, and then implanted into the damaged area.

Off-the-shelf cartilage implants would allow patients to undergo just one surgical procedure rather than two. Replacing damaged cartilage with a piece of new cartilage that was generated ahead of time would omit the delay in manufacturing associated with autologous cartilage harvesting, reduce the rehabilitation time, and allow patients to return to their normal activities sooner after surgery.

The first humans to receive this novel implant would likely be patients who have pain and joint damage but havent yet progressed to severe degeneration. And eventually, it could be tried in others, such as athletes with joint damage.

This could have a profound impact on people as they age as well as athletes experiencing joint pain, says Craft.

Learn more about the Craft Lab and the Orthopedic Department.

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Engineered cartilage and osteoarthritis - Boston Children's Answers - Boston Children's Discoveries

BlueRock takes up option on iPSC cell therapy candidate OpCT-001 – The Pharma Letter

By Induced Pluripotent Stem Cells

German pharma major Bayers (BAYN: DE) independently operated company BlueRock Therapeutics today revealed it has exercised its option to exclusively license OpCT-001 under a 2021 deal with FUJIFILM Cellular Dynamics and Opsis Therapeutics.

OpCT-001 is an induced pluripotent stem cell (iPSC) derived cell therapy candidate for the treatment of primary photoreceptor diseases and is the lead cell therapy candidate being developed under the strategic

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BlueRock takes up option on iPSC cell therapy candidate OpCT-001 - The Pharma Letter

The Enormous Potential of Induced Pluripotent Stem Cells (iPSCs) in Biomedical Research and Health Care – Medriva

By Induced Pluripotent Stem Cells

In the realm of biomedical research and health care, one of the most promising advancements in recent years involves induced pluripotent stem cells (iPSCs). These cells, which can be reprogrammed to behave like embryonic stem cells, have vast potential for understanding and treating a broad range of diseases, including diabetes, cancer, and neurological disorders. Theyre also being used to develop new drugs and could pave the way for personalized medicine.

iPSCs are adult cells that have been genetically reprogrammed to an embryonic stem cell-like state. This means they can potentially transform into any cell type in the body, making them a valuable resource for regenerative medicine and disease modeling. For example, they can be used to create patient-specific cell lines, which can then be used to study the mechanisms of disease at a cellular level, or to test potential treatments.

One of the significant advantages of iPSCs is their use in studying genetic diseases. By creating iPSCs from the cells of patients with specific genetic conditions, researchers can observe how these diseases develop and progress at a cellular level. This can provide invaluable insights into the underlying mechanisms of these conditions and could lead to the development of new, more effective treatments.

Moreover, iPSCs are playing a crucial role in drug discovery. They offer a more accurate and efficient way to test potential new drugs. Traditionally, new drugs are tested in animal models before being trialed in humans. But iPSCs provide a way to test these drugs on human cells, potentially speeding up the process and reducing reliance on animal testing.

Beyond disease study and drug development, iPSCs hold immense promise in the realm of regenerative medicine. They offer the potential to grow patient-specific tissues and organs for transplantation. This could revolutionize treatment for a variety of conditions, including heart disease, diabetes, and neurological disorders.

Furthermore, iPSCs have the potential to usher in a new era of personalized medicine. By creating patient-specific cell lines, treatments can be tailored to the individual, increasing their effectiveness and reducing the risk of adverse effects.

Despite their enormous potential, the use of iPSCs is not without challenges and ethical considerations. Issues such as the risk of tumorigenesis, the efficiency of reprogramming, and the possibility of immune rejection must be addressed. Moreover, the ethical implications surrounding the use of human cells in research and clinical applications must also be carefully considered.

Nonetheless, as our understanding and techniques improve, iPSCs are set to play an increasingly significant role in biomedical research and health care. With their potential to revolutionize disease study, drug development, regenerative medicine, and personalized healthcare, they represent one of the most exciting areas of modern medicine.

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The Enormous Potential of Induced Pluripotent Stem Cells (iPSCs) in Biomedical Research and Health Care - Medriva

Stem cells used to successfully treat arthritis in gorilla at Budapest zoo – University of Sheffield News

By Stem Cell Treatment

Stem cell therapy has been used to treat osteoarthritis in a gorilla for the first time, by scientists at the University of Sheffield.

Scientists at the University of Sheffield have used mesenchymal stem cells to treat arthritis in a gorilla at Budapest Zoo

The use of stem cells for the treatment of arthritis and regeneration of the damaged cartilage has been successfully piloted in several animal species, such as dogs and horses, in recent years

Liesel the gorilla is believed to be the first primate in the world to benefit from this joint work

Stem cell therapy has been used to treat osteoarthritis in a gorilla for the first time, by scientists at the University of Sheffield.

Liesel, the elderly matriarch at the Budapest Zoo has been finding it difficult to walk on her left leg for some time now, suggesting that she may be suffering from arthritis.

An international team, led by Endre Ss, Chief Vet and Acting Director General at the Budapest Zoo and Professor Mark Wilkinson, an Orthopaedic Surgeon and leading international expert in the treatment of human arthritis from the University of Sheffield, carried out a comprehensive assessment of Liesels major joints and used mesenchymal stem cells to treat alterations in her left hip and knee joints.

Osteoarthritis is a progressive degenerative process of the joint. Once the cartilage is worn and damaged, the process is irreversible and current treatments focus on symptomatic control but not to treat the disease itself.

The use of stem cells for the treatment of arthritis and regeneration of the damaged cartilage has been successfully piloted in several animal species in recent years, such as dogs and horses and small-scale clinical trials in humans have also proven to be a promising treatment for this condition. Liesel is thought to be the first primate in the world to receive the treatment and successfully benefited from the work of the research team.

Following previous successful research trials on arthritis-affected dogs, Stem CellX - a company made up of ateam of international scientists working in the field of stem cells, regenerative medicine, and genetics - was established to develop new technologies for the formulation of stem cell-based products for arthritis treatment in animals.

Stem CellX founder and Professor of Cell Signalling at the University of Sheffield, Endre Kiss-Tth has collaborated with Professor Mark Wilkinson for a number of years to explore novel treatment options for human arthritis. They now jointly lead a preclinical programme to test Stem CellX technologies for the development of a similar stem cell treatment in human patients.

The company recently partnered with Budapest Zoo to provide this treatment for animals in need, as well as supplying zoos globally.

The mesenchymal stem cells used for the procedure on Liesel were isolated from a piece of fat tissue donated by N'yaounda, a young female gorilla who underwent a planned minor operation in 2022. A specialist team at Stem CellX then isolated, purified and cultured these cells at their R&D base in Hungary to formulate a cell suspension that could be kept deep-frozen until the treatment.

Professor Kiss-Tth and Professor Wilkinson are now jointly leading a preclinical programme to test Stem CellX technologies for the development of a similar stem cell treatment in human patients.

Professor Endre Kiss-Toth, from the University of Sheffield and Founder of Stem CellX said:It has been a great privilege to be part of this word-first collaboration and bring together Stem CellX expertise in stem cell technologies, with the internationally leading clinical skills and knowledge in osteoarthritis pathogenesis of the University of Sheffield to provide a novel treatment option for Liesel to improve her quality of life in her golden years.

We are now following her recovery closely, in the hope to see marked improvement in her movements and in the use of her osteoarthritis affected leg.

Professor Mark Wilkinson, from the University of Sheffield and Leader of Clinical Orthopaedic Team, said:I was delighted to be part of the team doing this ground-breaking work and having the opportunity to treat Liesels arthritis. We are currently developing a similar treatment for humans. This work is in its very early stages but hopefully will lead to a real solution for patients to the pain and suffering that arthritis causes.

Honorary Associate Professor, Endre Ss, Leader of the Zoo Team said:The advanced husbandry and veterinary practices in modern zoos result inincreased longevity in many species, including apes. Our task is to providethe best medical care and best quality of life for these animals, despite theirage-related conditions. Stem-cell therapy hopefully brings in a new era in thisfield as well.

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Stem cells used to successfully treat arthritis in gorilla at Budapest zoo - University of Sheffield News

Could Treatments for HIV and Sickle Cell Open the Gene Therapy Floodgates? – BioSpace

By Stem Cell Treatment

Pictured: Illustration of gloved hands using tools to manipulate a DNA double helix/iStock, MicrovOne

In the early 1990s, Mike McCune and his colleagues thought theyd found a path to a one-time HIV treatment for babies: genetically modifying hematopoietic stem cells to suppress the virus and then transplanting them into the bone marrow. But the treatment never made it to clinical trials.

It was not considered to be a good business plan, he explained, because companies would make more money by selling these patients antiretroviral therapies for a lifetime than to deliver a one-time treatment.

Vertex Pharmaceuticals and CRISPR Therapeutics Casgevy, which involves a process much like McCunes HIV treatmentex vivo gene editing of blood stem cells for the treatment of sickle cell disease (SCD) followed by transplantation back into the patientdid make it to market, having been recently approved by the FDA as the first CRISPR-based therapy. But Casgevy is pricy: it costs a whopping $2.2 million dollars. Its competitor Lyfgenia, another ex vivo gene therapy for SCD approved at the same time, carries a sticker price of $3.1 million. The high cost may limit access even for patients in the U.S., and will likely be a far more formidable barrier in sub-Saharan Africa, where 79% of babies with SCD are born.

Now McCune and others are looking to bring gene therapy to the masses by developing treatments that involve treating cells in vivo, which could be far less costly than the ex vivo variety. Requiring only a single injection, the in vivo approach would use vectors to deliver DNA-changing machinery into cells. In the case of SCD, the target would be blood-making stem cells and the tweaks would induce them to make healthy hemoglobin, McCune explained; for HIV, viral DNA could be snipped inside infected cells to prevent viral replication and spread, as well as reinfection.

While an existing in vivo gene therapies for rare genetic diseases also have eye-popping sticker prices, McCune argued that over time, costs will fall.

McCune, now head of the Gates Foundations HIV Frontiers program, is helping to forge agreements between the philanthropy and multiple private companies with the aim of making gene therapy an accessible reality as a treatment for both HIV and SCD. And at least one biotech, Excision BioTherapeutics, is pursuing a similar approach to HIV treatment independently of Gates.

As McCune sees it, there will be both a market and payers for a one-shot HIV treatment, both in the U.S. and in sub-Saharan Africa. He points out that the healthcare system is currently paying tens of thousands of dollars annually for each HIV patient in the U.S., money that could be redirected to paying for a cure. And in sub-Saharan Africa, he added, the U.S. is spending about $7 billion each year on antiretroviral treatment for people with HIV under a program called PEPFARfunding that could likely cover the cost of a one-shot treatment instead.

Theres no comparable payer for an SCD treatment, he said. We have to work on that. And I think thats going to become really important because the inequities of healthcare, so poignantly highlighted during COVID, are going to become even more glaringly obvious now that there are multimillion-dollar treatments that are likely to remain inaccessible to many people with SCD.

Gates has partnered with companies including Guide Therapeutics, bluebird bio, GreenLight Biosciences, Intellia Therapeutics, CRISPR Therapeutics, Immunocore, BioNTech, Vir, Ensoma, Emmune, Addition and Novartis, as well as nonprofits and academic labs, to work on aspects of in vivo gene therapy. The foundation has also partnered with the National Institutes of Health, which committed to kicking in $100 million toward the effort.

Agreements with partners, McCune said, include a non-exclusive right for the Gates Foundation to make sure that the technologies are available to people living with HIV and SCD in low- and middle-income countries. Why would they give us those rights? he asked. Part of the answer is that the applications of this platform are diverse, outside of the realm of HIV and sickle [cell disease] and into the realm of the more remunerative diseases that companies seem to focus on in the context of gene therapy, including cancer. They would see this as a pathway for return on investment, which [could] be huge, McCune said.

Intellia, a former Gates grantee, is in the preclinical stages of developing an in vivo CRISPR treatment for SCD, company spokesperson Ian Karp wrote in an email to BioSpace. The benefit of a one-time treatment certainly has applicability to patients across the globe, he said. Additionally, our technology platform is modular, such that we do hope to leverage it across multiple indications / diseases. Oftentimes the only change we need to make from one investigational product to the next (particularly when targeting the same cell type and edit type) is in the targeting region of the guide RNA which serves to direct the CRISPR machinery to the gene of interest.

Similarly, Christine Silverstein, chief financial officer at Excision BioTherapeutics (which has not received Gates funding), said that the technology behind the companys candidate CRISPR HIV treatment, the Fast Tracked EBT-101, may be applied to other chronic viral infectious diseases including herpes and hepatitis B. In fact, our work in HIV is setting the foundation for advancements of Excisions pipeline which unites next-generation CRISPR nucleases with a novel, multiplexed gene editing approach to develop potentially curative therapies, she said in a statement.

But success is by no means assured. Despite the current excitement over CRISPR, hurdles to its therapeutic use remain, including not-insignificant safety concerns, and safety issues have also cropped up with other gene therapies. The Gates Foundation itself is hedging its bets, working in parallel to drive the development of a therapeutic vaccine for HIV.

Still, McCune is dreaming big. Even for conditions where effective treatments exist, these sorts of in vivo treatments might be something that takes pills off the shelves and sets the course for a different kind of medicine.

Shawna Williams is a senior editor at BioSpace. She can be reached at shawna.williams@biospace.com or on LinkedIn.

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Could Treatments for HIV and Sickle Cell Open the Gene Therapy Floodgates? - BioSpace

Embryo Patrol. Artificial Embryos Are Not Human Babies | by Karen Marie Shelton | ILLUMINATION-Curated | Jan, 2024 – Medium

By Embryonic Stem Cells

Artificial Embryos Are Not Human Babies Didactic Model of Human Embryonic Development Wagner Souza Esilva Wikimedia

Artificial embryos are not human. Theyre simply a cluster of cells. To be legally human, they must meet the definition of an in vitro fertilized human ovum.

As of the end of 2023, artificial embryos couldnt be successfully implanted into mammals or humans. They couldnt lead to pregnancies, and there is no plan for that to happen in the future.

Synthetic embryos utilize stem cells groundbreakingly, sidestepping the need for sperm or eggs. Ongoing breakthroughs might eventually aid research into genetic disorders and improve babies health, including reducing the risk of problem pregnancies and miscarriages.

Artificial embryos are not related to in vitro fertilization (IVF), which can lead to a human pregnancy.

The term is misleading. These structures arent really synthetic, nor are they exactly embryos. But theyre similar. They are tiny balls of cells arising from a sperm fertilizing an egg but created from stem cells grown in the lab.

Synthetic human embryos, or SHEEFs (synthetic human entities with embryo-like features), are created from very early (actually pre-embryonic) zygotic cells called stem cells.

The stem cells are called pluripotent because they have the potential to develop into almost every cell of the body.

The lab-created embryos are not connected to a beating heart or a brain. They do include cells that would typically go on to form a version of a placenta, yolk sac, and embryo itself.

The model embryos, which resemble human versions, recreate the earliest stages of human development. They could provide a crucial window into genetic disorders and the underlying biological causes of recurrent miscarriage.

Robin Lovell-Badge, headent head of stem cell biology and developmental genetics at Francis Crick Institute in London, reported project advancements. She explained weve cultivated embryos to a specific stage just beyond what is equivalent to 14 days of development for a natural embryo.

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Embryo Patrol. Artificial Embryos Are Not Human Babies | by Karen Marie Shelton | ILLUMINATION-Curated | Jan, 2024 - Medium

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