Gene Therapy for Retinal Degeneration | Zendy

Rajendra S. Apte | Zendy

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Gene Therapy for Retinal Degeneration

Author(s) -

Rajendra S. Apte

Publication year - 2018

Publication title -

cell

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 26.304

H-Index - 776

eISSN - 1097-4172

pISSN - 0092-8674

DOI - 10.1016/j.cell.2018.03.021

Subject(s) - rpe65 , biology , retinal degeneration , genetic enhancement , retinal , visual phototransduction , cis trans isomerases , macular degeneration , viral vector , bench to bedside , gene , genetics , computational biology , bioinformatics , ophthalmology , recombinant dna , medicine , peptidylprolyl isomerase , biochemistry , physics , isomerase , medical physics

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.

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