Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?
Author:
Affiliation:
1. Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK
2. Oxford Eye Hospital, Oxford University Hospitals NHS Trust, Oxford, UK
Funder
University of Oxford
Rhodes Foundation
National Institute for Health Research (NIHR) Oxford Biomedical Research Centre
Wellcome Trust
Publisher
Informa UK Limited
Subject
Pharmacology (medical),Health Policy,Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
Link
https://www.tandfonline.com/doi/pdf/10.1080/21678707.2021.1882300
Reference84 articles.
1. Rab escort protein 1 (REP1) in intracellular traffic: a functional and pathophysiological overview
2. Deficient Geranylgeranylation of Ram/Rab27 in Choroideremia
3. Retinal Degeneration in Choroideremia: Deficiency of Rab Geranylgeranyl Transferase
4. Correction: Conditional Ablation of the Choroideremia Gene Causes Age-Related Changes in Mouse Retinal Pigment Epithelium
5. Independent degeneration of photoreceptors and retinal pigment epithelium in conditional knockout mouse models of choroideremia
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1. A Prospective, Observational, Non-interventional Clinical Study of Participants With Choroideremia: The NIGHT Study;American Journal of Ophthalmology;2024-07
2. Choroideremia: Toward Regulatory Approval of Retinal Gene Therapy;Cold Spring Harbor Perspectives in Medicine;2023-06-05
3. AAV2-Mediated Gene Therapy for Choroideremia: 5-Year Results and Alternate Anti-sense Oligonucleotide Therapy;American Journal of Ophthalmology;2023-04
4. Molecular Therapy for Choroideremia: Pre-clinical and Clinical Progress to Date;Molecular Diagnosis & Therapy;2021-10-18
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