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

Physiology and Biophysics

Stony Brook University
University of Rochester

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

12 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

Keywords

β-hexosaminidase
GM gangliosidosis
Mouse, Gene therapy
Sandhoff disease

Access to Document

10.1016/j.jneuroim.2009.02.006

Cite this

@article{ca07a9acf8da4670aa92b1f9e410b91a,

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

author = "Stephanos Kyrkanides and Meixiang Yang and Tallents, \{Ross H.\} and Miller, \{Jen nie H.\} and Brouxhon, \{Sabine M.\} and Olschowka, \{John A.\}",

year = "2009",

month = apr,

day = "30",

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

language = "English",

volume = "209",

pages = "139--142",

journal = "Journal of Neuroimmunology",

issn = "0165-5728",

number = "1-2",

}

TY - JOUR

T1 - The trigeminal retrograde transfer pathway in the treatment of neurodegeneration

AU - Kyrkanides, Stephanos

AU - Yang, Meixiang

AU - Tallents, Ross H.

AU - Miller, Jen nie H.

AU - Brouxhon, Sabine M.

AU - Olschowka, John A.

PY - 2009/4/30

Y1 - 2009/4/30

N2 - 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.

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.

KW - β-hexosaminidase

KW - GM gangliosidosis

KW - Mouse, Gene therapy

KW - Sandhoff disease

UR - https://www.scopus.com/pages/publications/64549099776

U2 - 10.1016/j.jneuroim.2009.02.006

DO - 10.1016/j.jneuroim.2009.02.006

M3 - Article

C2 - 19278737

AN - SCOPUS:64549099776

SN - 0165-5728

VL - 209

SP - 139

EP - 142

JO - Journal of Neuroimmunology

JF - Journal of Neuroimmunology

IS - 1-2

ER -

The trigeminal retrograde transfer pathway in the treatment of neurodegeneration

Abstract

Keywords

Access to Document

Other files and links

Fingerprint

Cite this