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Fine-tuning FAM161A gene augmentation therapy to restore retinal function

19 Mar 2024

Abstract excerpt

For 15 years, gene therapy has been viewed as a beacon of hope for inherited retinal diseases. Many preclinical investigations have centered around vectors with maximal gene expression capabilities, yet despite efficient gene transfer, minimal physiological improvements have been observed in various ciliopathies. Retinitis pigmentosa-type 28 (RP28) is the consequence of bi-allelic null mutations in the FAM161A,...

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PMID: 38504136
DOI: 10.1038/s44321-024-00053-x

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