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Forever Friends, a spin-out company from Oxford University, hopes to dramatically expand the lifespan of dogs using new gene-editing technology. This startup wants to use gene-editing to extend ...
Gene therapy [83] uses genetically modified viruses to deliver genes which can cure disease in humans. Although gene therapy is still relatively new, it has had some successes. It has been used to treat genetic disorders such as severe combined immunodeficiency [84] and Leber's congenital amaurosis. [85]
Gene therapy may be classified into two types by the type of cell it affects: somatic cell and germline gene therapy. In somatic cell gene therapy (SCGT), the therapeutic genes are transferred into any cell other than a gamete , germ cell , gametocyte , or undifferentiated stem cell .
Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency (no longer commercially available); Axicabtagene ciloleucel (Yescarta): treatment for large B-cell lymphoma [1]
Nyvlt-art/Shutterstock By Ludwig Burger and Ben Hirschler The Western world's first gene therapy drug is set to go on sale in Germany with a 1.1 million euro ($1.4 million) price tag, a new record ...
The grandaddy of gene-editing stocks recently provided an encouraging update, but it isn't the only experimental hemophilia therapy out there.
Recombinant adeno-associated virus (rAAV) based genome engineering is a genome editing platform centered on the use of recombinant AAV vectors that enables insertion, deletion or substitution of DNA sequences into the genomes of live mammalian cells.
NEW YORK/ZURICH, Dec 20 (Reuters) - Biogen Inc's Spinraza treatment for spinal muscular atrophy and Swiss drugmaker Novartis AG's experimental gene therapy are both expensive, but the gene therapy ...