C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/86 (2006.01) A61K 38/47 (2006.01) A61K 48/00 (2006.01) C12N 7/01 (2006.01) C12N 9/24 (2006.01) C12N 15/56 (2006.01) C12N 15/864 (2006.01)
Patent
CA 2243261
The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue is disclosed. The invention allows for the direct, in vivo injection of recombinant AAV virions into muscle tissue, e.g., by intramuscular injection, as well as for the in vitro transduction of muscle cells which can subsequently be introduced into a subject for treatment. The invention provides for sustained, high-level expression of the delivered gene and for in vivo secretion of the therapeutic protein from transduced muscle cells such that systemic delivery is achieved.
L'invention porte sur l'emploi de virions de virus adéno-associés (AAV) de recombinaison pour fournir des molécules d'ADN aux cellules et aux tissus musculaires. Elle recourt à une injection directe in vivo de virions d'AAV de recombinaison (par exemple par injection intramusculaire) dans les tissus musculaires et à la transduction in vitro de cellules de muscles qui sont ensuite administrées au patient pour le traiter. Elle porte par ailleurs sur l'expression continue à haut niveau du gène administré et sur la sécrétion in vivo de la protéine thérapeutique à partir de cellules transduites de muscles, entraînant la libération systémique.
Byrne Barry J.
Kessler Paul D.
Kurtzman Gary J.
Podsakoff Gregory M.
Avigen Inc.
Johns Hopkins University
Smart & Biggar
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