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Gene therapy for spinal disorders

A spinal cord, transgenic technology, applied in the field of gene therapy for spinal cord disorders, can solve the problems of patients with loss of motor function and still existing needs, etc.

Inactive Publication Date: 2008-06-25
GENZYME CORP
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

[0008] [08] 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 spinal disorders
  • Gene therapy for spinal disorders
  • Gene therapy for spinal disorders

Examples

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Embodiment

[0099] [099] The following examples provide illustrative embodiments of the invention. One of ordinary skill in the art will recognize numerous modifications and variations that could be implemented without altering the spirit or scope of the invention. Such modifications and changes are included within the scope of the present invention. These examples do not limit the invention.

[0100] Example

[0101] Titration of recombinant vector

[0100] Titers of AAV vectors were measured in terms of genome copy number (genome particles per milliliter). Genomic Particle Concentration Based on Taqman of Vector DNA PCR, as previously reported (Clark et al., (1999) Hum. Gene Ther., 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 ...

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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] [01] According to Section 119(e) of Volume 35 of the United States Code, this application requires U.S. Provisional Application No.60 / 677,213 filed on May 2, 2005 and U.S. Provisional Application No.60 / 60 / 213 filed on April 8, 2006 790,217 claims of priority, the contents of which are incorporated herein by reference. technical field [0002] [02] The present invention relates to compositions and methods of treatment for the treatment of movement disorders affecting motor function in a subject, particularly those affected by diseases or injuries of the brain and / or spinal cord. Background technique [0003] [03] 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 that efficiently infect postmitotic neurons. The central nervous system consists of the spinal cord and brain. The spinal cord conducts sensory information from the...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K48/00C12N15/86
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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