Category REGENERATIVE MEDICINE

Researchers uncover mechanism for how common gene therapy vectors enter cells

Identification of GPR108 as ‘lock’ for adeno-associated virus vector (AAV) is crucial to this emerging novel class of genetic medicine and may provide framework for developing more targeted gene therapies Researchers led by a team at Massachusetts Eye and Ear have identified a novel cellular entry factor for adeno-associated virus vector (AAV) types–the most commonly

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Researchers regrow damaged nerves with polymer and protein

University of Pittsburgh School of Medicine researchers have created a biodegradable nerve guide—a polymer tube—filled with growth-promoting protein that can regenerate long sections of damaged nerves, without the need for transplanting stem cells or a donor nerve. Source UPMC So far, the technology has been tested in monkeys, and the results of those experiments appeared

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Exploding cancer cells can cause serious side effects in CAR-T cell therapies

Blocking a protein makes cells shrink instead, causing fewer problems Techniques to genetically modify patient immune cells have revolutionized the fight against hard-to-treat cancers. But they can come with dangerous side effects. Now, researchers have found one reason why. A particularly messy form of cell death sparks severe inflammation in patients receiving CAR-T cell immunotherapy for blood

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ISSCR Issues Statement on Ethical Standards for Stem Cell-based Embryo Models

Original story from the International Society for Stem Cell Research The ISSCR is updating its Guidelines for Stem Cell Research and Clinical Translation to respond to recent scientific advances that include the use of pluripotent stem cell (PSC) to create models of early human embryo development. As the science continues to advance, it raises important

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Cancer study may accidentally help researchers create usable blood stem cells

Researchers investigating childhood leukaemia have discovered that increasing MLL gene expression in iPSCs drives hematopoietic stem cell production, so could be the target of new therapies. Researchers have identified that increasing the expression of the normal form of the MLL gene can drive production of hematopoietic stem cells (HSCs). This not only provides a protocol

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Why stem cells could be the medical innovation of the century for World Economic Forum

Source World Economic Forum The current market for stem cell therapies is growing at 36% per year and will rapidly expand when a breakthrough treatment for non-communicable disease or a lifestyle factor occurs. Funding is flowing into the sector from venture capitalists and large companies. With greater resources and an increasingly experienced talent pool, scientists

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UK accounts for over 12% of global cell and gene therapy clinical trials. CGT Catapult new report

The UK and the NHS are providing the right environment and infrastructure to allow innovative cell and gene therapies to reach patients. International companies recognise the appeal of the UK cell and gene therapy ecosystem and are sponsoring the majority of the UK commercial clinical trials which account for 77% of the total 127 ongoing trials.

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bluebird bio Announces Launch in Germany of ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene) Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalassemia Who Do Not Have β0/β0 Genotype

First agreements with statutory health insurances utilize bluebird’s innovative value-based payment model and provide coverage for ZYNTEGLO for up to 50% of patients in Germany First qualified treatment center established at University Hospital of Heidelberg to provide ZYNTEGLO to patients  bluebird bio, Inc. announced the launch in Germany of ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene), a

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The Alliance for Regenerative Medicine Demonstrates Potential for Cell and Gene Therapies to Provide 10-Year Cost Savings to the Healthcare System

ARM today announced the release of a report, “A Transformative Therapy Value Model for Rare Blood Diseases.” The report uses a refined value model to assess the impact of durable and potentially curative cell and gene therapies on health system costs.   Produced in collaboration with the Marwood Institute, this report calculates the potential cost savings that

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