Intravitreal Delivery of rAAV2tYF-CB-hRS1 Vector for Gene Augmentation Therapy in Patients with X-Linked Retinoschisis
Author:
Funder
Applied Genetic Technologies Corporation
Publisher
Elsevier BV
Subject
Ophthalmology
Reference42 articles.
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2. X-linked juvenile retinoschisis: clinical diagnosis, genetic analysis, and molecular mechanisms;Molday;Prog Retin Eye Res,2012
3. Carbonic anhydrase inhibitors in patients with X-linked retinoschisis: effects on macular morphology and function;Testa;Ophthalmic Genet,2019
4. Prolonged recovery of retinal structure/function after gene therapy in an Rs1h-deficient mouse model of x-linked juvenile retinoschisis;Min;Mol Ther,2005
5. Retinoschisin gene therapy and natural history in the Rs1h-KO mouse: long-term rescue from retinal degeneration;Kjellstrom;Invest Ophthalmol Vis Sci,2007
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2. Investigating the role of Caspase-1 in a mouse model of Juvenile X-linked Retinoschisis;Frontiers in Medicine;2024-06-13
3. Retinal organoids with X-linked retinoschisis RS1 (E72K) mutation exhibit a photoreceptor developmental delay and are rescued by gene augmentation therapy;Stem Cell Research & Therapy;2024-05-31
4. Intravitreal Delivery of rAAV2-hSyn-hRS1 Results in Retinal Ganglion Cell-Specific Gene Expression and Retinal Improvement in the Rs1-KO Mouse;Human Gene Therapy;2024-05-01
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