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 ...
1. Etranacogene dezaparvovec gene therapy was more effective than prophylactic factor IX infusion at reducing annualized bleeding rate amongst patients ...
1. Topical gene therapy beremagene geperpavec (B-VEC) was superior to placebo in achieving complete wound healing in patients with dystrophic ...
1. Hemophilia B patients treated with the adeno-associated virus (AAV) gene therapy produced dose-dependent sustained factor IX levels. 2. All ...
1. The adeno-associated virus 5 (AAV5)-based gene therapy, valoctocogene roxaparvovec (AAV5-hFVIII-SQ), resulted in sustained factor VIII production, reduced bleeding rates, ...
1. Betibeglogene autotemcel (beti-cel) gene therapy resulted in sustained hemoglobin A with a T87Q amino acid substitution (HbAT87Q) in non-β0/β0 ...
1. Treatment with autologous CD34+ hematopoietic stem and progenitor cells transduced with human ADA via a lentiviral vector showed sustained ...
1. A one-time infusion of a recombinant viral vector with a highly expressed factor IX gene resulted in sustained endogenous ...
1. In this study of seven individuals with Wiskott-Aldrich Syndrome (WAS), gene therapy improved eczema, susceptibility to infection, autoimmunity, and ...
Image: PD 1. Two patients with X-linked severe combined immunodeficiency (SCID) were treated with a retroviral-vector containing γc receptor for ...
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