Intraperitoneal inoculation of Sandhoff mouse neonates with an HIV-1 based lentiviral vector exacerbates the attendant neuroinflammation and disease phenotype

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

doi:10.1016/j.jneuroim.2007.05.010

Intraperitoneal inoculation of Sandhoff mouse neonates with an HIV-1 based lentiviral vector exacerbates the attendant neuroinflammation and disease phenotype

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

Physiology and Biophysics

University of Rochester

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

10 Scopus citations

Abstract

We aimed to evaluate the efficacy of VSV-G pseudotyped, defective HIV-1 based lentiviral vectors for the neonatal transfer of therapeutic genes following systemic administration in Sandhoff mouse pups. Despite transgene expression in mouse brains, these animals presented with significant exacerbation and acceleration of the disease neurological phenotype. We observed an increase and acceleration in the presence of MHC-II and CD45+ cells in their brains, along with neuroinflammation, but not in control heterozygous or wild type littermates that also received the same treatment.

Original language	English
Pages (from-to)	39-47
Number of pages	9
Journal	Journal of Neuroimmunology
Volume	188
Issue number	1-2
DOIs	https://doi.org/10.1016/j.jneuroim.2007.05.010
State	Published - Aug 2007

Keywords

β-hexosaminidase
Gene therapy
Lentiviral vectors
Lysosomal storage disorder
Sandhoff disease

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10.1016/j.jneuroim.2007.05.010

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title = "Intraperitoneal inoculation of Sandhoff mouse neonates with an HIV-1 based lentiviral vector exacerbates the attendant neuroinflammation and disease phenotype",

abstract = "We aimed to evaluate the efficacy of VSV-G pseudotyped, defective HIV-1 based lentiviral vectors for the neonatal transfer of therapeutic genes following systemic administration in Sandhoff mouse pups. Despite transgene expression in mouse brains, these animals presented with significant exacerbation and acceleration of the disease neurological phenotype. We observed an increase and acceleration in the presence of MHC-II and CD45+ cells in their brains, along with neuroinflammation, but not in control heterozygous or wild type littermates that also received the same treatment.",

keywords = "β-hexosaminidase, Gene therapy, Lentiviral vectors, Lysosomal storage disorder, Sandhoff disease",

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

year = "2007",

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doi = "10.1016/j.jneuroim.2007.05.010",

language = "English",

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T1 - Intraperitoneal inoculation of Sandhoff mouse neonates with an HIV-1 based lentiviral vector exacerbates the attendant neuroinflammation and disease phenotype

AU - Kyrkanides, Stephanos

AU - Miller, Jen nie H.

AU - Tallents, Ross H.

AU - Brouxhon, Sabine M.

AU - Centola, Gina M.

AU - Olschowka, John A.

PY - 2007/8

Y1 - 2007/8

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AB - We aimed to evaluate the efficacy of VSV-G pseudotyped, defective HIV-1 based lentiviral vectors for the neonatal transfer of therapeutic genes following systemic administration in Sandhoff mouse pups. Despite transgene expression in mouse brains, these animals presented with significant exacerbation and acceleration of the disease neurological phenotype. We observed an increase and acceleration in the presence of MHC-II and CD45+ cells in their brains, along with neuroinflammation, but not in control heterozygous or wild type littermates that also received the same treatment.

KW - β-hexosaminidase

KW - Gene therapy

KW - Lentiviral vectors

KW - Lysosomal storage disorder

KW - Sandhoff disease

UR - https://www.scopus.com/pages/publications/34447633012

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AN - SCOPUS:34447633012

SN - 0165-5728

VL - 188

SP - 39

EP - 47

JO - Journal of Neuroimmunology

JF - Journal of Neuroimmunology

IS - 1-2

ER -

Intraperitoneal inoculation of Sandhoff mouse neonates with an HIV-1 based lentiviral vector exacerbates the attendant neuroinflammation and disease phenotype

Abstract

Keywords

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