Antisense oligonucleotide therapy for proline-23-histidine autosomal dominant retinitis pigmentosa

To discuss antisense oligonucleotide (ASON) therapy for autosomal dominant retinitis pigmentosa (adRP) caused by the proline-23-histidine (P23H) mutation in the rhodopsin gene. Viral and nonviral therapies to treat adRP are currently under investigation. A promising therapeutic option is a nonviral...

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Bibliographic Details
Published inCurrent opinion in ophthalmology Vol. 34; no. 3; p. 226
Main Authors Justin, Grant A, Girach, Aniz, Maldonado, Ramiro S
Format Journal Article
LanguageEnglish
Published United States 01.05.2023
Subjects
Animals
Histidine - genetics
Humans
Mutation
Oligonucleotides, Antisense - therapeutic use
Proline - genetics
Retinitis Pigmentosa - drug therapy
Retinitis Pigmentosa - genetics
Rhodopsin - genetics
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Summary:To discuss antisense oligonucleotide (ASON) therapy for autosomal dominant retinitis pigmentosa (adRP) caused by the proline-23-histidine (P23H) mutation in the rhodopsin gene. 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. 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.
ISSN:1531-7021
DOI:10.1097/ICU.0000000000000947