The trigeminal retrograde transfer pathway in the treatment of neurodegeneration

Stephanos Kyrkanides; Meixiang Yang; Ross H. Tallents; Jen nie H. Miller; Sabine M. Brouxhon; John A. Olschowka

doi:10.1016/j.jneuroim.2009.02.006

The trigeminal retrograde transfer pathway in the treatment of neurodegeneration

Stephanos Kyrkanides, Meixiang Yang, Ross H. Tallents, Jen nie H. Miller, Sabine M. Brouxhon, John A. Olschowka

Oral Health Science

Research output: Contribution to journal › Article › peer-review

10 Scopus citations

Abstract

The trigeminal sensory system was evaluated for the retrograde transfer of gene therapy vectors into the CNS. The feline immunodeficiency viral vector, FIV(HEXB), encoding for the human HEXB gene, was injected intra-articularly in the temporomandibular joint of 12 week-old HexB^-/- mice displaying clinical and histopathological signs of Sandhoff disease. This treatment regiment reduced GM₂ storage and ameliorated neuroinflammation in the brain of HexB^-/- mice, as well as attenuated behavioral deficits. In conclusion, retrograde transfer along trigeminal sensory nerves may prove to be a valuable route of gene therapy administration for the treatment of lysosomal storage disorders and other neurodegenerative diseases.

Original language	English
Pages (from-to)	139-142
Number of pages	4
Journal	Journal of Neuroimmunology
Volume	209
Issue number	1-2
DOIs	https://doi.org/10.1016/j.jneuroim.2009.02.006
State	Published - Apr 30 2009

Bibliographical note

Funding Information:
This work was funded in part by grant NS048339 from the National Institutes of Health.

Keywords

GM gangliosidosis
Mouse, Gene therapy
Sandhoff disease
β-hexosaminidase

ASJC Scopus subject areas

Immunology and Allergy
Immunology
Neurology
Clinical Neurology

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.1016/j.jneuroim.2009.02.006

Cite this

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title = "The trigeminal retrograde transfer pathway in the treatment of neurodegeneration",

abstract = "The trigeminal sensory system was evaluated for the retrograde transfer of gene therapy vectors into the CNS. The feline immunodeficiency viral vector, FIV(HEXB), encoding for the human HEXB gene, was injected intra-articularly in the temporomandibular joint of 12 week-old HexB-/- mice displaying clinical and histopathological signs of Sandhoff disease. This treatment regiment reduced GM2 storage and ameliorated neuroinflammation in the brain of HexB-/- mice, as well as attenuated behavioral deficits. In conclusion, retrograde transfer along trigeminal sensory nerves may prove to be a valuable route of gene therapy administration for the treatment of lysosomal storage disorders and other neurodegenerative diseases.",

keywords = "GM gangliosidosis, Mouse, Gene therapy, Sandhoff disease, β-hexosaminidase",

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doi = "10.1016/j.jneuroim.2009.02.006",

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AU - Kyrkanides, Stephanos

AU - Yang, Meixiang

AU - Tallents, Ross H.

AU - Miller, Jen nie H.

AU - Brouxhon, Sabine M.

AU - Olschowka, John A.

N1 - Funding Information: This work was funded in part by grant NS048339 from the National Institutes of Health.

PY - 2009/4/30

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AB - The trigeminal sensory system was evaluated for the retrograde transfer of gene therapy vectors into the CNS. The feline immunodeficiency viral vector, FIV(HEXB), encoding for the human HEXB gene, was injected intra-articularly in the temporomandibular joint of 12 week-old HexB-/- mice displaying clinical and histopathological signs of Sandhoff disease. This treatment regiment reduced GM2 storage and ameliorated neuroinflammation in the brain of HexB-/- mice, as well as attenuated behavioral deficits. In conclusion, retrograde transfer along trigeminal sensory nerves may prove to be a valuable route of gene therapy administration for the treatment of lysosomal storage disorders and other neurodegenerative diseases.

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KW - Sandhoff disease

KW - β-hexosaminidase

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The trigeminal retrograde transfer pathway in the treatment of neurodegeneration

Abstract

Bibliographical note

Keywords

ASJC Scopus subject areas

UN SDGs

Access to Document

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