Mayo Clinic offers a unique regenerative medicine approach for repairing knee cartilage, which can be completed in a single surgery. The Food and Drug Administration approved the use of this technique, known as recycled cartilage auto/allo implantation (RECLAIM), in a trial utilizing the stem cell bank in the Mayo Clinic Center for Regenerative Medicine. “Mayo
Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A published Gene therapy with valoctocogene roxaparvovec reduced annualized rates of bleeding events and led to discontinuation of prophylactic factor VIII among patients with hemophilia A, according to results of a prospective study with multiyear follow-up published in The New England Journal of Medicine. “The treatment burden is
Novel spinal therapy/delivery approach prevented disease onset in neurodegenerative ALS disease model in adult mice and blocked progression in animals already showing disease symptoms Writing in Nature Medicine, an international team headed by researchers at University of California San Diego School of Medicine describe a new way to effectively deliver a gene-silencing vector to adult amyotrophic
Before gene therapy can be used to treat renal diseases, delivery of therapeutic genes to the kidney must become much more efficient. A novel approach in which three different gene delivery vectors were injected intravenously and directly into the kidneys of mice was reported in an article published in Human Gene Therapy, a peer-reviewed journal from
Early results suggest that prime editors are cleaner than CRISPR–Cas9 and more versatile than base editors, but many questions remain. A paper recently published in Nature from David Liu and co-workers discloses a ‘prime’ gene-editing system many years in the making. In principle, the system, comprising a catalytically impaired Cas9 enzyme and an engineered reverse transcriptase, may
Four months after treating them, Yasuhiro Shiga, MD, PhD, checked on his rats. Walking into the lab, he carried minimal expectations. Treating spinal cord injuries with stem cells had been tried by many people, many times before, with modest success at best. The endpoint he was specifically there to measure — pain levels — hadn’t
Drugs that manipulate the code of life are finally changing lives Source Scientific American The idea for gene therapy—a type of DNA-based medicine that inserts a healthy gene into cells to replace a mutated, disease-causing variant—was first published in 1972. After decades of disputed results, treatment failures and some deaths in experimental trials, the first
The strategy has long been seen as having enormous potential to cure diseases Paving the way for solving a major safety issue associated with gene therapies, scientists have developed a special molecular switch that could be embedded into such therapies to allow doctors to control dosing. The feat, published in the journal Nature Biotechnology, offers
Mutations in the RIPK1 gene responsible for CRIA syndrome. Over the last 20 years, three families have been unsuspectingly linked by an unknown illness. Researchers at the National Human Genome Research Institute (NHGRI), part of the National Institutes of Health, and other organizations have now identified the cause of the illness, a new disease called
Potential 1st Gene Therapy in Europe Directed at Any Type of Hemophilia Application to be Reviewed Under Accelerated Assessment BioMarin Pharmaceutical announced today that the European Medicines Agency (EMA) validated the Company’s Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. The MAA review will commence in January
