Abstract
Hematopoietic stem cell (HSC) gene therapy has effectively become a therapeutic option largely due to the resounding clinical successes in patients with primary immunodeficiencies (PIDs) such as X-linked severe immunodeficiency (SCID-X1), adenosine deaminase deficiency (ADA-SCID), Wiskott-Aldrich syndrome (WAS), and chronic granulomatous disease (CGD). HSC gene therapy is also being investigated in patients with metabolic diseases such as X-linked adrenoleukodystrophy (ALD) and metachromatic leukodystrophy (MLD) and inherited blood disorders such as β-thalassemia and sickle cell disease (SCD) where some therapeutic benefits have been reported more recently. Safer and more efficient self-inactivating (SIN) γ-retroviral and lentiviral vectors have been developed to overcome the genotoxicity imparted by γ-retroviral vectors with intact long terminal repeat (LTR). The discovery and maturation of gene-editing platforms, including zinc-finger nuclease (ZFN), transcription activator-like effector nucleases (TALEN), and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9, offer exciting prospective strategies for further improving gene therapy by targeting the repair of diseased genes.
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