Antisense oligonucleotide therapy for proline-23-histidine autosomal dominant retinitis pigmentosa. Issue 3 (14th May 2023)

• Record Type: Journal Article
• Authors: Justin, Grant A.; Girach, Aniz; Maldonado, Ramiro S.
• Is Part Of: Current opinion in ophthalmology. Volume 34:Issue 3(2023)
• Page Start: 226
• Page End: 231
• Publication Date: 2023-05-14
• Subjects: adeno-associated virus -- antisense oligonucleotides -- genetic therapy -- retinitis pigmentosa -- rhodopsin; Ophthalmology -- Periodicals; Eye Diseases -- Indexes; Eye Diseases -- Periodicals; Review Literature -- Indexes; Review Literature -- Periodicals; Vision Disorders -- Indexes; Vision Disorders -- Periodicals; 617.7
• DOI: 10.1097/ICU.0000000000000947
• Languages: English
• ISSNs: 1040-8738

Abstract

Abstract : Purpose of review: To discuss antisense oligonucleotide (ASON) therapy for autosomal dominant retinitis pigmentosa (adRP) caused by the proline-23-histidine (P23H) mutation in the rhodopsin gene. Recent findings: Viral and nonviral therapies to treat adRP are currently under investigation. A promising therapeutic option is a nonviral approach using ASONs. This form of genetic therapy has demonstrated a dose-dependent and highly selective reduction of P23H mutant rhodopsin mRNA in animal models, and it is currently being investigated as a human phase 1/2 clinical trial. Summary: There are promising new therapies to treat adRP. ASON has shown encouraging results in animal models and has undergone a phase 1 clinical trial. ASON does not use a viral vector, is delivered with standard intravitreal injection, and its effects are reversible.