Selecta Biosciences, Inc.
METHODS AND COMPOSITIONS OF OTC CONSTRUCTS AND VECTORS
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Abstract:
Provided herein are methods and compositions related to nucleic acids encoding ornithine transcarbamylase (OTC), such as nucleic acids comprising an OTC codon-optimized sequence, as well as related vectors, such as AAV vectors. Also, provided are methods for administering AAV vectors that comprise a sequence that encodes an enzyme associated with an urea cycle disorder and an expression control sequence, in combination with synthetic nanocarriers coupled to an immunosuppressant.
Status:
Application
Type:
Utility
Filling date:
16 Jul 2019
Issue date:
6 Feb 2020