C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/62 (2006.01) A61K 38/17 (2006.01) A61K 38/30 (2006.01) A61K 47/48 (2006.01) A61P 25/00 (2006.01) C07K 1/00 (2006.01) C07K 14/47 (2006.01) C07K 14/65 (2006.01) C07K 19/00 (2006.01) C12N 15/12 (2006.01) C12N 15/16 (2006.01) C12P 21/00 (2006.01) A61K 38/00 (2006.01)
Patent
CA 2463473
Disclosed are methods and compositions for targeting therapeutic proteins to the brain. Methods and compositions of the invention involve associating an IGF moiety with a therapeutic protein in order to target the therapeutic protein to the brain. Soluble fusion proteins that include an IGF targeting moiety are transported to neural tissue in the brain from blood. Methods and compositions of the invention include therapeutic applications for treating lysosomal storage diseases. The invention also provides nucleic acids and cells for expressing IGF fusion proteins.
Cette invention a trait à des techniques et aux compositions correspondantes visant à apporter au cerveau des protéines thérapeutiques par ciblage. Ces techniques consistent à associer un fragment de facteur de croissance semblable à l'insuline (IGF) à une protéine thérapeutique, afin de cibler celle-ci sur le cerveau. Des protéines hybrides solubles renfermant un fragment d'IGF sont transportées par le sang vers le tissu nerveux du cerveau. Cette invention porte également sur des applications thérapeutiques visant au traitement de maladies lysosomales. Elle concerne, de surcroît, des acides nucléiques ainsi que des cellules exprimant ces protéines hybrides/IGF.
Lebowitz Jonathan H.
Sly William S.
Biomarin Pharmaceutical Inc.
Fasken Martineau Dumoulin Llp
Symbiontics Inc.
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