TY - JOUR
T1 - Gene Therapy for Retinal Degeneration
AU - Apte, Rajendra S.
N1 - Funding Information:
Dr. Apte is supported by the Starr Foundation, the Glenn Foundation for Medical Research, the Kuzma Family Fund, the Research to Prevent Blindness Nelson Trust Award for Retinitis Pigmentosa, and an unrestricted grant from Research to Prevent Blindness to the Department of Ophthalmology, Washington University School of Medicine, St. Louis, MO.
Publisher Copyright:
© 2018
PY - 2018/3/22
Y1 - 2018/3/22
N2 - 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. 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.
AB - 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. 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.
UR - http://www.scopus.com/inward/record.url?scp=85044132232&partnerID=8YFLogxK
U2 - 10.1016/j.cell.2018.03.021
DO - 10.1016/j.cell.2018.03.021
M3 - Short survey
C2 - 29570997
AN - SCOPUS:85044132232
VL - 173
SP - 5
JO - Cell
JF - Cell
SN - 0092-8674
IS - 1
ER -