Αποτελέσματα Αναζήτησης
I. Ex vivo gene therapy: This involves the transfer of genes in cultured cells (e.g., bone marrow cells) which are then reintroduced into the patient. II. In vivo gene therapy: The direct delivery of genes into the cells of a particular tissue is referred to as in vivo gene therapy.
8 Νοε 2023 · Severe combined immune deficiency (SCID) was the first clinical success with gene therapy. SCID is the most severe human IEI, with absent T and B lymphocyte function making the infant...
In this review, we provide historical and biological perspectives on the advent and clinical implementation of ex vivo retroviral gene therapy, focusing on the milestones during the development of HSCs-based GTs and immunotherapies that led to the clinical approvals.
Since 2000, gene therapy has become an option to treat severe combined immunodeficiency (SCID), a rare but lethal condition characterized by fully defective T lymphocyte differentiation caused by pathogenic variants in least 18 genes.
Ex vivo retrovirally mediated gene therapy has been shown within the last 20 yr to correct the T cell immunodeficiency caused by γc-deficiency (SCID X1) and adenosine deaminase (ADA) deficiency. The rationale was brought up by the observation of the revertant of SCIDX1 and ADA deficiency as a kind of natural gene therapy.
In 2016, the European Commission granted market approval to GlaxoSmithKline (GSK) for ex vivo hematopoietic stem cell (HSC) gene therapy for the treatment of adenosine deaminase (ADA)‐deficient severe combined immunodeficiency (SCID), a very rare congenital disorder of the immune system.
12 Μαρ 2010 · In all, 20 patients with classic SCID-X1 disease and 27 with ADA deficiency underwent ex vivo gene therapy with γ-retroviral vectors in the period of 1999–2009.
