AAV-RPGR Gene Therapy Rescues Opsin Mislocalisation in a Human Retinal Organoid Model of RPGR-Associated X-Linked Retinitis Pigmentosa
Author:
Affiliation:
1. MeiraGTx UK II, 34-38 Provost Street, London N1 7NG, UK
2. Moorfields Eye Hospital, 162 City Road, London EC1V 2PD, UK
3. University College London Institute of Ophthalmology, London EC1V 9LF, UK
Abstract
Funder
MeiraGTx
Publisher
MDPI AG
Link
https://www.mdpi.com/1422-0067/25/3/1839/pdf
Reference34 articles.
1. RPGR-Related Retinopathy: Clinical Features, Molecular Genetics, and Gene Replacement Therapy;Georgiou;Cold Spring Harb. Perspect. Med.,2023
2. A Systematic Literature Review of Disease Progression Reported in RPGR-Associated X-Linked Retinitis Pigmentosa;Lam;Retina,2023
3. A Gene (RPGR) with Homology to the RCC1 Guanine Nucleotide Exchange Factor Is Mutated in X-Linked Retinitis Pigmentosa (RP3);Meindl;Nat. Genet.,1996
4. Mutational Analysis of RPGR and RP2 Genes in Japanese Patients with Retinitis Pigmentosa: Identification of Four Mutations;Jin;Mol. Vis.,2006
5. Mutational Hot Spot within a New RPGR Exon in X-Linked Retinitis Pigmentosa;Vervoort;Nat. Genet.,2000
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1. Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-Associated X-Linked Retinitis Pigmentosa;American Journal of Ophthalmology;2024-11
2. Gene Therapy for Retinitis Pigmentosa: Current Challenges and New Progress;Biomolecules;2024-07-25
3. Gene Therapies in Clinical Development to Treat Retinal Disorders;Molecular Diagnosis & Therapy;2024-07-02
4. Inherited Retinal Diseases and Retinal Organoids as Preclinical Cell Models for Inherited Retinal Disease Research;Genes;2024-05-28
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