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Gene Therapy For Neurometabolic Disorders

A technology of spinal cord and genetic modification, applied in the field of gene therapy for spinal cord disorders, which can solve the problems of still existing needs and loss of motor function of patients

Pending Publication Date: 2016-07-20
GENZYME CORP
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

[0008] However, there remains a need for compositions and methods of treating spinal cord abnormalities that result in loss of motor function in patients

Method used

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  • Gene Therapy For Neurometabolic Disorders
  • Gene Therapy For Neurometabolic Disorders
  • Gene Therapy For Neurometabolic Disorders

Examples

Experimental program
Comparison scheme
Effect test

Embodiment

[0101] Titration of recombinant vector

[0102] Titers of AAV vectors were measured in terms of genome copy number (genome particles per milliliter). Genomic Particle Concentration Based on Vector DNA PCR, as previously reported (Clark et al., (1999) Hum. GeneTher., 10:1031-1039; Veldwijk et al., (2002) Mol. Ther, 6:272-278). Briefly, purified AAV-ASM was treated with capsid protein digestion buffer (50 mM Tris-HCl pH 8.0, 1.0 mM EDTA, 0.5% SDS, 1.0 mg / ml proteinase K) at 50°C for 1 hour to release vector DNA. DNA samples are subjected to polymerase chain reaction (PCR) with primers that anneal to specific regions of the vector DNA, such as promoter regions, transgenes, or poly(A) sequences. PCR results are then passed in real-time Software, such as that provided by the PerkinElmer-Applied Biosystems (Foster City, CA) Prism 7700 Sequence Detection System, is used for quantification.

[0103] Vectors carrying detectable marker genes such as β-galactosidase or the green fluo...

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Abstract

This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention provides a method to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene. The viral vector delivers the transgene to a region of the deep cerebellar nuclei region of the brain. Also provided are compositions and methods to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene to the motor cortex region of the subject's brain.

Description

[0001] This application is a divisional application of an invention application with a filing date of May 2, 2006, a Chinese application number of 200680023365.X, and an invention title of "gene therapy for spinal cord disorders". Pursuant to 35 U.S.C. 119(e), this application claims U.S. Provisional Application No. 60 / 677, filed May 2, 2005, 213 and U.S. Provisional Application No. 60 / 790, filed April 8, 2006 , 217 Claims of Priority, the contents of which are incorporated herein by reference. technical field [0002] The present invention relates to compositions and methods of treatment for treating motor disorders affecting motor function in a subject, particularly motor disorders affected by diseases or injuries of the brain and / or spinal cord. Background technique [0003] Gene therapy is an emerging treatment modality for the treatment of disorders affecting the central nervous system (CNS). CNS gene therapy has been facilitated by the development of viral vectors tha...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K48/00A61P25/28A61P25/00
CPCA61K48/0075C12N2799/025A61P21/00A61P25/00A61P25/02A61P25/28C12N2750/14041C12N15/8645C07K14/475
Inventor J·道奇L·谢哈布丁M·帕西尼S·程C·R·奥赖尔登
Owner GENZYME CORP
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