Work

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

Year: 2026

Type: article

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.

Source: Molecular Therapy Advances

DOI: 10.1016/j.omta.2026.201786

Authors: Sif G. Kaad, Tess C. Lengyell, Seyedeh Zeinab Mirjalili Mohanna, Diana Djaksigulova, Andrea J. Korecki, Elizabeth M. Simpson

Institutions: University of British Columbia, BC Children's Hospital

Language: English

Topic: Retinal Development and Disorders

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: hybrid

Funders: BC Children's Hospital, Canadian Institutes of Health Research