Benitec, Inc.

Genzyme Corporation

Spark Therapeutics, Inc.

Linda Couto

David A Suhy

Alexander A Kolykhalov

Petrus W Roelvink

Peter C Colosi

Xiaobing Qian

Methods for assaying function and/or activity and/or potency of isomerohydrolase proteins are provided.

Relative potency assay for viral vector encoding isomerohydrolases

The present invention provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases or disorders.

RNAi expression constructs

One form of a composition has two types of recombinant adeno-associated virus. The first type encodes a portion of Factor VIII operably linked to an expression control element; and the second type encodes a different portion of Factor VIII operably linked to an expression control element. The first and second nucleotide sequences collectively encode a functional Factor VIII protein. Another form of the composition is a recombinant adeno-associated virus containing a nucleotide sequence encoding functional Factor VIII light or heavy chain operably linked to a tissue-specific promoter.

Adeno-associated vector compositions for expression of Factor VIII

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Pleasanton, CA, United States of America

Linda Couto