First pilot study of intrastromal rAAV-PAX6 gene therapy suggests improved corneal thickness and transcription correction in aniridic mouse

Molecular Therapy Advances 2026 Open access

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Authors: Sif G. Kaad, Tess C. Lengyell, Seyedeh Zeinab Mirjalili Mohanna, Diana Djaksigulova, Andrea J. Korecki, Elizabeth M. Simpson
DOI: 10.1016/j.omta.2026.201786
Cited by: 1
References: 63

Abstract

. We explore a variety of hypothesizes to explain these initial results, including that the virally delivered PAX6 is acting non-cell autonomously, moving from the non-dividing stroma into the epithelium, causing structural and molecular improvement.

Fields of Study & Subjects

Retinal Development and Disorders Developmental Biology and Gene Regulation Corneal Surgery and Treatments

Publication details

Journal / source
Molecular Therapy Advances
Publisher
Elsevier BV
Volume
34
Issue
3
Pages
201786–201786
Publication date
17 June 2026
Language
EN