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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