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Early and late stage gene therapy interventions for inherited retinal degenerations.
Prog Retin Eye Res. 2021 May 29 [Online ahead of print]PR

Abstract

Inherited and age-related retinal degeneration is the hallmark of a large group of heterogeneous diseases and is the main cause of untreatable blindness today. Genetic factors play a major pathogenic role in retinal degenerations for both monogenic diseases (such as retinitis pigmentosa) and complex diseases with established genetic risk factors (such as age-related macular degeneration). Progress in genotyping techniques and back of the eye imaging are completing our understanding of these diseases and their manifestations in patient populations suffering from retinal degenerations. It is clear that whatever the genetic cause, the majority of vision loss in retinal diseases results from the loss of photoreceptor function. The timing and circumstances surrounding the loss of photoreceptor function determine the adequate therapeutic approach to use for each patient. Among such approaches, gene therapy is rapidly becoming a therapeutic reality applicable in the clinic. This massive move from laboratory work towards clinical application has been propelled by the advances in our understanding of disease genetics and mechanisms, gene delivery vectors, gene editing systems, and compensatory strategies for loss of photoreceptor function. Here, we provide an overview of existing modalities of retinal gene therapy and their relevance based on the needs of patient populations suffering from inherited retinal degenerations.

Authors+Show Affiliations

Sorbonne Université, INSERM, CNRS, Institut de la Vision, F-75012, Paris, France.Sorbonne Université, INSERM, CNRS, Institut de la Vision, F-75012, Paris, France.Sorbonne Université, INSERM, CNRS, Institut de la Vision, F-75012, Paris, France.Sorbonne Université, INSERM, CNRS, Institut de la Vision, F-75012, Paris, France.Sorbonne Université, INSERM, CNRS, Institut de la Vision, F-75012, Paris, France; Department of Ophthalmology, The University of Pittsburgh School of Medicine, Pittsburgh, PA, 15213, United States; CHNO des Quinze-Vingts, INSERM-DGOS CIC 1423, F-75012, Paris, France; Fondation Ophtalmologique Rothschild, F-75019, Paris, France.Sorbonne Université, INSERM, CNRS, Institut de la Vision, F-75012, Paris, France. Electronic address: deniz.dalkara@inserm.fr.

Pub Type(s)

Journal Article
Review

Language

eng

PubMed ID

34058340

Citation

Botto, Catherine, et al. "Early and Late Stage Gene Therapy Interventions for Inherited Retinal Degenerations." Progress in Retinal and Eye Research, 2021, p. 100975.
Botto C, Rucli M, Tekinsoy MD, et al. Early and late stage gene therapy interventions for inherited retinal degenerations. Prog Retin Eye Res. 2021.
Botto, C., Rucli, M., Tekinsoy, M. D., Pulman, J., Sahel, J. A., & Dalkara, D. (2021). Early and late stage gene therapy interventions for inherited retinal degenerations. Progress in Retinal and Eye Research, 100975. https://doi.org/10.1016/j.preteyeres.2021.100975
Botto C, et al. Early and Late Stage Gene Therapy Interventions for Inherited Retinal Degenerations. Prog Retin Eye Res. 2021 May 29;100975. PubMed PMID: 34058340.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Early and late stage gene therapy interventions for inherited retinal degenerations. AU - Botto,Catherine, AU - Rucli,Marco, AU - Tekinsoy,Müge Defne, AU - Pulman,Juliette, AU - Sahel,José-Alain, AU - Dalkara,Deniz, Y1 - 2021/05/29/ PY - 2021/02/18/received PY - 2021/05/18/revised PY - 2021/05/21/accepted PY - 2021/6/1/pubmed PY - 2021/6/1/medline PY - 2021/5/31/entrez KW - AAV KW - CRISPR/Cas9 KW - Gene therapy KW - Neuroprotection KW - Optogenetics KW - Retina SP - 100975 EP - 100975 JF - Progress in retinal and eye research JO - Prog Retin Eye Res N2 - Inherited and age-related retinal degeneration is the hallmark of a large group of heterogeneous diseases and is the main cause of untreatable blindness today. Genetic factors play a major pathogenic role in retinal degenerations for both monogenic diseases (such as retinitis pigmentosa) and complex diseases with established genetic risk factors (such as age-related macular degeneration). Progress in genotyping techniques and back of the eye imaging are completing our understanding of these diseases and their manifestations in patient populations suffering from retinal degenerations. It is clear that whatever the genetic cause, the majority of vision loss in retinal diseases results from the loss of photoreceptor function. The timing and circumstances surrounding the loss of photoreceptor function determine the adequate therapeutic approach to use for each patient. Among such approaches, gene therapy is rapidly becoming a therapeutic reality applicable in the clinic. This massive move from laboratory work towards clinical application has been propelled by the advances in our understanding of disease genetics and mechanisms, gene delivery vectors, gene editing systems, and compensatory strategies for loss of photoreceptor function. Here, we provide an overview of existing modalities of retinal gene therapy and their relevance based on the needs of patient populations suffering from inherited retinal degenerations. SN - 1873-1635 UR - https://www.unboundmedicine.com/medline/citation/34058340/Early_and_late_stage_gene_therapy_interventions_for_inherited_retinal_degenerations. L2 - https://linkinghub.elsevier.com/retrieve/pii/S1350-9462(21)00036-7 DB - PRIME DP - Unbound Medicine ER -
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