Title:
ASSISTED ENZYME REPLACEMENT THERAPY
Document Type and Number:
WIPO Patent Application WO/2011/034951
Kind Code:
A3
Abstract:
Reagents and methods useful for the synthesis of conjugates comprising guanidinylated cyclic acetals are provided. Also provided are methods for increasing the cellular uptake of various therapeutic compounds and treatment modalities using these conjugates.
Inventors:
ESKO JEFFREY D (US)
TOR YITZHAK (US)
TOR YITZHAK (US)
Application Number:
PCT/US2010/048968
Publication Date:
October 13, 2011
Filing Date:
September 15, 2010
Export Citation:
Assignee:
UNIV CALIFORNIA (US)
ESKO JEFFREY D (US)
TOR YITZHAK (US)
ESKO JEFFREY D (US)
TOR YITZHAK (US)
International Classes:
A61K31/7036; A61K31/352; A61K31/70; A61K31/7048; A61K38/43; A61P3/00; A61P43/00
Foreign References:
| US6537785B1 | 2003-03-25 |
Other References:
ELSON-SCHWAB,L. ET AL.: "Guanidinylated Neomycin Delievers Large, Bioactive Cargo into Cells through a Heparan sulfate-dependent Pathway", JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 282, no. 18, 2007, pages 13585 - 13591
LUEDTKE,N.W. ET AL.: "Cellular Uptake of Aminoglycosides, Guanidinoglycosides, and Poly-arginine", J. AM. CHEM. SOC., vol. 125, 2003, pages 12374 - 12375
DESNICK,R.J.: "Enzyme Replacement and Enhancement Therapies for Lysosomal Disease", J. INHERIT. METAB. DIS., vol. 27, 2004, pages 385 - 410
WINCHESTER,B. ET AL.: "The Molecular Basis of Lysosomal Storage Diseases and Their Treatment", BIOCHEMICAL SOCIETY TRANSACTIONS, vol. 28, 2000, pages 150 - 153
LUEDTKE,N.W. ET AL.: "Cellular Uptake of Aminoglycosides, Guanidinoglycosides, and Poly-arginine", J. AM. CHEM. SOC., vol. 125, 2003, pages 12374 - 12375
DESNICK,R.J.: "Enzyme Replacement and Enhancement Therapies for Lysosomal Disease", J. INHERIT. METAB. DIS., vol. 27, 2004, pages 385 - 410
WINCHESTER,B. ET AL.: "The Molecular Basis of Lysosomal Storage Diseases and Their Treatment", BIOCHEMICAL SOCIETY TRANSACTIONS, vol. 28, 2000, pages 150 - 153
Attorney, Agent or Firm:
LAVOIE, Teresa A. et al. (P. O. Box 1022Minneapolis, Minnesota, US)
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