Invention Grant
US07807618B2 Methods and compositions for delivering enzymes and nucleic acid molecules to brain, bone and other tissues
有权
将酶和核酸分子递送到脑,骨骼和其他组织的方法和组合物
- Patent Title: Methods and compositions for delivering enzymes and nucleic acid molecules to brain, bone and other tissues
- Patent Title (中): 将酶和核酸分子递送到脑,骨骼和其他组织的方法和组合物
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Application No.: US10386144Application Date: 2003-03-11
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Publication No.: US07807618B2Publication Date: 2010-10-05
- Inventor: Reuben Matalon
- Applicant: Reuben Matalon
- Applicant Address: US TX Austin
- Assignee: The Board of Regents of the University of Texas System
- Current Assignee: The Board of Regents of the University of Texas System
- Current Assignee Address: US TX Austin
- Agent Benjamin Aaron Adler
- Main IPC: A01N37/18
- IPC: A01N37/18 ; A61K38/00 ; A61K49/00

Abstract:
Disclosed are methods for delivering an enzyme to a subject's brain or bone. The methods include administering a hyaluronidase to the subject and administering the enzyme to the subject. The hyaluronidase and the enzyme are administered to the subject under conditions effective to deliver the enzyme to the subject's brain or bone. Compositions and kits which include hyaluronidase and an enzyme are also disclosed, as are methods for increasing blood-brain barrier permeability in a subject. Also disclosed are methods, compositions, and kits for delivering genes or other nucleic acid molecules to a subject's brain or bone, as well as methods, compositions, and kits for delivering enzymes to a subject's tissues. The methods, compositions, and kits are disclosed as being useful in treating or preventing a variety of enzyme deficiency diseases, such as those affecting brain and/or bone, e.g., as Canavan's disease, Fabry disease, Gaucher's disease, various forms of mucopolysaccharidosis (e.g., Hurler's syndrome, Scheie syndrome, Hurler-Scheie syndrome, Sanfillippo A syndrome, Morquio A syndrome, Morquio B syndrome, etc.), Niemann-Pick disease, Schindler disease, and Pompe disease.
Public/Granted literature
Information query
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