Gene therapy improves bilirubin levels in Crigler-Najjar syndrome
1. In this randomized controlled trial, a high dose of GNT0003 gene therapy was associated with decreased serum bilirubin and ...
Tag: gene therapy
Etranacogene dezaparvovec gene therapy reduces bleeding in severe hemophilia B
1. Etranacogene dezaparvovec gene therapy was more effective than prophylactic factor IX infusion at reducing annualized bleeding rate amongst patients ...
Beremagene geperpavec improves wound healing in dystrophic epidermolysis bullosa
1. Topical gene therapy beremagene geperpavec (B-VEC) was superior to placebo in achieving complete wound healing in patients with dystrophic ...
Low-dose AAVS3 gene therapy is efficacious for treating patients with hemophilia B
1. Hemophilia B patients treated with the adeno-associated virus (AAV) gene therapy produced dose-dependent sustained factor IX levels. 2. All ...
Valoctocogene roxaparvovec gene therapy efficacious for hemophilia A treatment
1. The adeno-associated virus 5 (AAV5)-based gene therapy, valoctocogene roxaparvovec (AAV5-hFVIII-SQ), resulted in sustained factor VIII production, reduced bleeding rates, ...
Betibeglogene autotemcel gene therapy effective for β-thalassemia
1. Betibeglogene autotemcel (beti-cel) gene therapy resulted in sustained hemoglobin A with a T87Q amino acid substitution (HbAT87Q) in non-β0/β0 ...
Lentiviral gene therapy treatment for adenosine deaminase deficiency
1. Treatment with autologous CD34+ hematopoietic stem and progenitor cells transduced with human ADA via a lentiviral vector showed sustained ...
Recombinant viral vector treatment sustains endogenous factor IX activity in patients with hemophilia B
1. A one-time infusion of a recombinant viral vector with a highly expressed factor IX gene resulted in sustained endogenous ...
Gene therapy may be helpful for Wiskott-Aldrich Syndrome
1. In this study of seven individuals with Wiskott-Aldrich Syndrome (WAS), gene therapy improved eczema, susceptibility to infection, autoimmunity, and ...
Experimental gene therapy results in full clinical reversal of X-linked SCID [Classics Series]
Image: PD 1. Two patients with X-linked severe combined immunodeficiency (SCID) were treated with a retroviral-vector containing γc receptor for ...
