Four Ways to Package Transgenes That Exceed the Size Limit of Adeno-associated Virus
Type
Blog Post
Published
Sept. 1, 2020, 1:15 p.m.
...to a mouse model of muscular dystrophy. Three different splice site donor and acceptor sequences were ...reconstituted after integration; and a sequence of stuffer DNA after the gRNA target site.
Donor B contains...greater and more consistent transgene expression.
References:
Allocca M, Doria M, Petrillo M, Colella P, Garcia-Hoyos...retinal gene therapy
Learn about the parts of an AAV transfer plasmid
Resources on Addgene.org:
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