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Triple Vectors Expand AAV Transfer Capacity in the Retina

6 Dec 2017

Abstract excerpt

Retinal gene transfer with adeno-associated viral (AAV) vectors holds great promise for the treatment of inherited retinal degenerations (IRDs). One limit of AAV is its transfer capacity of about 5 kb, which can be expanded to about 9 kb, using dual AAV vectors. This strategy would still not suffice for treatment of IRDs such as Usher syndrome type 1D or Alström syndrome type I (ALMS) due to mutations in CDH23 or...

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PMID: 29292161
DOI: 10.1016/j.ymthe.2017.11.019

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