Massimo Giunti

TL;DR: It is found that dual trans‐splicing and hybrid vectors transduce efficiently mouse and pig photoreceptors to levels that, albeit lower than those achieved with a single AAV, resulted in significant improvement of the retinal phenotype of mouse models of STGD and USH1B.

TL;DR: The data support the potential of dual AAV vectors for large gene reconstitution in the cone-enriched pig retina that is a relevant preclinical model and shows that subretinal delivery in pigs ofDual AAV trans-splicing and hybrid vectors successfully reconstitute the expression of the large genes ABCA4 and MYO7A mutated in STGD and USH1B.

TL;DR: The data presented here on AAV-mediated transduction of the cone-enriched porcine retina may affect the development of gene-based therapies for rare and common severe photoreceptor diseases.

TL;DR: Dual AAV hybrid ABCA4 vectors including homologous ITR2, the photoreceptor-specific G protein-coupled receptor kinase 1 promoter, the AK region of homology and the CL1 degradation signal are developed and shown that upon subretinal administration these vectors are both safe in pigs and effective in Abca4−/− mice.

TL;DR: This study provides new important results about the evolutionary dynamics of CPV infections in cats, showing that CPV has presumably started a new process of readaptation in feline hosts.