The CRISPR (clustered regularly interspaced short palindromic repeats)-based genome editing technology is an emerging RNA-guided nuclease system initially identified from the microbial adaptive immune systems. In recent years, the CRISPR system has been reprogrammed to target specific regions of the eukaryotic genome and has become a powerful tool for genetic engineering. Researchers have explored many
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
