WebMay 4, 2024 · The new gene therapy being trialed focuses on correcting a defect in the CNGA3 gene. The treatment is somewhat similar to one of the first FDA-approved gene therapies, for vision loss, called ... WebDec 19, 2024 · Three gene therapy phase I/II trials are currently being conducted in human patients in the USA, the UK, and Germany. This review details the AAV gene therapy treatments of achromatopsia to date. We also present novel data showing rescue of a Cnga3-/- mouse model using an rAAV.CBA.CNGA3 vector.
Achromatopsia: Symptoms, Causes and Treatment
WebNo authorized therapy for ACHM exists, but research activities have intensified over the past decade and have led to several preclinical gene therapy studies that have shown functional and morphological improvements in animal models of ACHM. WebMay 14, 2024 · The preliminary results of 2 ongoing phase 1/2 clinical trials evaluating the effects of subretinal gene therapies for achromatopsia (ACHM) indicate that both … 8項 英語
Pipeline - MeiraGTx
WebMar 18, 2024 · The affected dogs have thus become valuable models for preclinical studies of gene therapy for problems such as eye diseases, immunodeficiency, lysosomal storage diseases, hemophilia, and muscular dystrophy. ... Michalakis S, Schön C, Becirovic E, Biel M (2024) Gene therapy for achromatopsia. J Gene Med 19(3). 10.1002/jgm.2944 ; … WebJan 10, 2024 · Current research in Achromatopsia 1) Gene therapy. Gene therapy . aims to halt retinal degeneration by replacing the mutated gene with a normal healthy copy. … WebThe potential for the treatment of achromatopsia in humans with gene therapy shows great promise. INTRODUCTION. Achromatopsia, also known as rod monochromacy, is present in about 1:30 000 births. It is an autosomal-recessive genetic disease defined by loss of cone cell function in the retina, classically presenting with color blindness ... 8顆球 天平