C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/864 (2006.01) A61K 48/00 (2006.01) C07K 14/015 (2006.01) C12N 5/10 (2006.01) A61K 38/00 (2006.01)
Patent
CA 2500523
Disclosed are improved VP2-modified recombinant adeno-associated viral (rAAV) vectors, expression systems, and rAAV virions that are fully virulent, yet lack functional VP2 protein expression. Also disclosed are pharmaceutical compositions, virus particles, host cells, and pharmaceutical formulations that comprise these modified vectors useful in the expression of therapeutic proteins, polypeptides, peptides, antisense oligonucleotides and/or ribozymes in the cells and tissues of selected mammals, including, for example, human tissues and host cells.
L'invention concerne des vecteurs viraux adéno-associés recombinants (rAAV) améliorés modifiés par VP2, des systèmes d'expression, et des virions de rAAV entièrement virulents mais sont cependant dépourvus de l'expression fonctionnelle de la protéine VP2. L'invention concerne également des compositions pharmaceutiques, des particules virales, des cellules hôtes et des préparations pharmaceutiques comprenant ces vecteurs modifiés utiles dans l'expression de protéines thérapeutiques, de polypeptides, de peptides, d'oligonucléotides antisens et/ou de ribozymes dans des cellules et des tissus de mammifères choisis, y compris, par exemple, des tissus et cellules hôtes humains.
Muzyczka Nicholas
Opie Shaun R.
Warrington Kenneth H.
Torys Llp
University Of Florida Research Foundation Inc.
LandOfFree
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