Physics

Visual phototransduction

Macular degeneration

Retinal degeneration

Bioinformatics

Biology

Gene

Ophthalmology

Genetic enhancement

Medical physics

Peptidylprolyl isomerase

Bench to bedside

Recombinant DNA

RPE65

Retinal

Computational biology

Isomerase

Cis-trans-Isomerases

Viral vector

Genetics

Biochemistry

Medicine

Cell

Biallelic mutations in the RPE65 gene are associated with inherited retinal degenerations/dystrophies (IRD) and disrupt the visual cycle, leading to loss of vision. A new adenoviral vector-based gene therapy surgically delivered to retinal cells provides normal human RPE65 protein that can restore the visual cycle and some vision. To view this Bench to Bedside, open or download the PDF.

Gene Therapy for Retinal Degeneration