The new landscape of retinal gene therapy.
Am J Med Genet C Semin Med Genet
; 184(3): 846-859, 2020 09.
Article
in En
| MEDLINE
| ID: mdl-32888388
ABSTRACT
Novel therapeutics for inherited retinal dystrophies (IRDs) have rapidly evolved since groundbreaking clinical trials for LCA due to RPE65 mutations led to the first FDA-approved in vivo gene therapy. Since then, advancements in viral vectors have led to more efficient AAV transduction and developed other viral vectors for gene augmentation therapy of large gene targets. Furthermore, significant developments in gene editing and RNA modulation technologies have introduced novel capabilities for treatment of autosomal dominant diseases, intronic mutations, and/or large genes otherwise unable to be treated with current viral vectors. We highlight strategies currently being evaluated in gene therapy clinical trials and promising preclinical developments for IRDs.
Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Genetic Therapy
/
Cis-trans-Isomerases
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Retinal Dystrophies
/
Gene Editing
Limits:
Humans
Language:
En
Journal:
Am J Med Genet C Semin Med Genet
Journal subject:
GENETICA MEDICA
Year:
2020
Document type:
Article
Affiliation country:
United States