Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina

Volume: 11, Issue: 492
Published: May 15, 2019
Abstract
Retinal gene therapy with adeno-associated viral (AAV) vectors holds promises for treating inherited and noninherited diseases of the eye. Although clinical data suggest that retinal gene therapy is safe and effective, delivery of large genes is hindered by the limited AAV cargo capacity. Protein trans-splicing mediated by split inteins is used by single-cell organisms to reconstitute proteins. Here, we show that delivery of multiple AAV vectors...
Paper Details
Title
Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina
Published Date
May 15, 2019
Volume
11
Issue
492
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