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Gene therapy for RAB28: What can we learn from zebrafish?
Ailis L Moran1, John D Fehilly1, Oliver Blacque1
1UCD School of Biomolecular and Biomedical Science, University College Dublin, Dublin, Ireland; UCD Conway Institute, University College Dublin, Dublin, Ireland.
Vision Research
|June 15, 2023
Summary
Gene therapy shows promise for inherited retinal degenerations (IRDs) by restoring RAB28 gene function in cone photoreceptors. Zebrafish models offer valuable insights for developing therapies and diagnosing patient variants.
Area of Science:
- Ophthalmology
- Genetics
- Molecular Biology
Background:
- Inherited retinal degenerations (IRDs) represent a significant unmet need, with 281 known causative genes.
- RAB28 gene mutations cause autosomal recessive cone-rod dystrophy (arCORD) in humans.
- The eye's unique properties make it a prime target for gene therapy interventions.
Approach:
- This review examines RAB28 biology and its role in arCORD.
- It explores the utility of zebrafish models for preclinical gene therapy development.
- The study assesses zebrafish as a diagnostic tool for patient variants of unknown significance (VUS).
Key Points:
- Restoring wild-type zebrafish Rab28 in cone photoreceptors rescued outer segment phagocytosis defects in rab28 knockouts.
- This preclinical finding supports the potential of RAB28 gene restoration for treating arCORD.
- Zebrafish models provide a platform for evaluating gene therapy efficacy and diagnosing VUS.
Conclusions:
- Gene therapy targeting RAB28 holds promise for treating arCORD.
- Zebrafish serve as a valuable model for advancing IRD gene therapy research.
- Utilizing zebrafish can aid in understanding patient-specific RAB28 variants and inform clinical strategies.
