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

Grant A. Justin, Aniz Girach, Ramiro S. Maldonado*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review


Purpose of reviewTo discuss antisense oligonucleotide (ASON) therapy for autosomal dominant retinitis pigmentosa (adRP) caused by the proline-23-histidine (P23H) mutation in the rhodopsin gene.Recent findingsViral 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.SummaryThere 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.

Original languageEnglish
Pages (from-to)226-231
Number of pages6
JournalCurrent Opinion in Ophthalmology
Issue number3
StatePublished - 1 May 2023
Externally publishedYes


  • adeno-Associated virus
  • antisense oligonucleotides
  • genetic therapy
  • retinitis pigmentosa
  • rhodopsin


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