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A method of treating peripheral neuropathies and motor neuron diseases

A peripheral nervous system and disease technology, applied in nervous system diseases, neuromuscular system diseases, gene therapy, etc., can solve problems such as limiting gene expression

Inactive Publication Date: 2017-03-08
GENETHON +1
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

Furthermore, it shows that CSF (cerebrospinal fluid) injections target motor neurons and limit gene expression to CSF

Method used

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  • A method of treating peripheral neuropathies and motor neuron diseases
  • A method of treating peripheral neuropathies and motor neuron diseases
  • A method of treating peripheral neuropathies and motor neuron diseases

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Embodiment Construction

[0045] The present invention relates to a strategy for the delivery of molecules to the peripheral nervous system (PNS) and / or central nervous system (CNS) of a subject. The methods disclosed herein provide the potential for imaging, and thus visualization of tissue, through the use of markers such as the GFP protein. Alternatively, PNS and / or CNS diseases or disorders can be treated by delivering the therapeutic molecule to the tissue.

[0046] Although the present application discloses efficient delivery to the PNS, delivery to the CNS is believed to occur via retrotransport as previously reported (Zheng et al. (2010, Hum. Gen. Ther. 21(1), 87-97 ).

[0047] In one embodiment, the present invention provides a method of delivering a molecule to the peripheral nervous system (PNS) and / or central nervous system (CNS) of a subject comprising administering to said subject a Compositions. In other words, the present invention relates to a composition comprising a molecule for t...

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Abstract

A composition comprising a molecule for use in the delivery of the molecule to the peripheral nervous system (PNS) and / or to the central nervous system (CNS), wherein the composition is administered by regional infusion.

Description

[0001] Background of the invention [0002] The peripheral nervous system (PNS) is made up of nerves and neurons, including peripheral nerves and ganglia that lie outside the central nervous system (CNS) or extend from the brain and spinal cord outside the CNS. [0003] PNS has been implicated in a large number of inherited or acquired neurological disorders, such as Charcot-Marie-Tooth, diabetic, infectious, toxic, and drug-related or immune-related neuropathies. [0004] Effective clinical interventions for those diseases are very limited. Gene therapy represents a novel therapeutic strategy for PNS diseases. However, efficient PNS transgenesis remains critical for gene therapy of inherited and acquired peripheral neuropathies. [0005] Adeno-associated virus (AAV) vectors have been reported to efficiently transduce dorsal root ganglion (DRG) neurons. However, this requires delicate microneurosurgical techniques to deliver AAV to the DRG (Glatzel et al., 2000, Proc. Natl. A...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C12N15/864A61K48/00A61P25/02A61P25/00
CPCA61K48/0025A61K48/0075C12N15/86C12N2750/14141C12N2750/14143C12N2830/008A61P21/00A61P21/04A61P25/00A61P25/02A61K9/0019A61K31/7088
Inventor A·M·布伊贝洛M·K·奇尔德斯
Owner GENETHON
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