C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 5/10 (2006.01) A61K 31/70 (2006.01) A61K 35/34 (2006.01) A61K 38/18 (2006.01) A61K 38/47 (2006.01) A61K 48/00 (2006.01) C07K 14/505 (2006.01) C12N 9/24 (2006.01) C12N 15/86 (2006.01) C12N 15/864 (2006.01) A61K 38/00 (2006.01)
Patent
CA 2243470
The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue in the treatment of anemia 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 a delivered nucleotide sequence encoding erythropoietin, and for in vivo secretion thereof 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 en vue du traitement de l'anémie. 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 d'une séquence nucléotidique administrée codant pour l'érythropoïétine et pour sa sécrétion in vivo à partir de cellules transduites de muscles, conduisant à une administration par voie générale.
Kurtzman Gary J.
Podsakoff Gregory M.
Avigen Inc.
Smart & Biggar
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