Arlene V. Drack

A splice-switching antisense oligonucleotide delivered via intravitreal injection mitigates early retinal dysfunction in a pig model of CLN3 Batten disease.

This study shows that a hypertonic buffer reduces retinal cysts and improves cone function in Rs1-KO mice, suggesting an osmolarity-dependent therapeutic effect.

This paper describes a method to deliver large genes using AAV vectors and the CRE-lox system, enabling efficient reconstitution of therapeutic genes beyond AAV packaging limits.

This paper presents a novel porcine model of CLN3 Batten disease that recapitulates clinical phenotypes observed in humans.

This study develops a BBS10 knockout mouse model to investigate retinal degeneration, finding progressive loss of cone function and thinning of the outer nuclear layer.