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This is one of the first studies of a CRISPR-based in vivo human gene editing therapy, where the editing takes place inside the human body. [266] The first injection of the CRISPR-Cas System was confirmed in March 2020. [267] Exagamglogene autotemcel, a CRISPR-based human gene editing therapy, was used for sickle cell and thalassemia in ...
Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency (no longer commercially available); Axicabtagene ciloleucel (Yescarta): treatment for large B-cell lymphoma [1]
A more recent focus of the journal includes Cell Therapy, with genetic modification, in essence ex vivo gene therapy. Previous issues have focused on the latest developments in gene transfer, gene expression and regulation, development of novel gene delivery vectors, and ex vivo gene therapies, animal models, and human therapeutic applications.
Gene knockdown is an experimental technique by which the expression of one or more of an organism's genes is reduced. The reduction can occur either through genetic modification or by treatment with a reagent such as a short DNA or RNA oligonucleotide that has a sequence complementary to either gene or an mRNA transcript.
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In 1999, Wilson led a clinical trial at the Institute for Human Gene Therapy using an adenoviral vector that resulted in the death of Jesse Gelsinger.As a result, the government banned him from working on FDA-regulated human clinical trials for five years and shut down the institute, which led to a shift in his research focus towards a study of adeno-associated viruses (AAV).
Modifying human embryos to give the CCR5 Δ32 allele protects them from the disease. An other use would be to cure genetic disorders. In the first study published regarding human germline engineering, the researchers attempted to edit the HBB gene which codes for the human β-globin protein. HBB mutations produce β-thalassaemia, which can be ...
On January 9, 2020, the company reported positive results from a Phase 1/2 trial for its investigational gene therapy for retinitis pigmentosa. [11] A few weeks later, the company reported interim six-month data from the dose-escalation cohorts of its ongoing Phase 1/2 clinical programs in patients with achromatopsia due to mutation in the ACHM ...