Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model

Brian K. Kaspar, Jerònia Lladó, Nushin Sherkat, Jeffrey D Rothstein, Fred H. Gage

Research output: Contribution to journalArticle

Abstract

Amyotrophic lateral sclerosis (ALS) is a progressive, lethal neuromuscular disease that is associated with the degeneration of spinal and brainstem motor neurons, leading to atrophy of limb, axial, and respiratory muscles. The cause of ALS is unknown, and there is no effective therapy. Neurotrophic factors are candidates for therapeutic evaluation in ALS. Although chronic delivery of molecules to the central nervous system has proven difficult, we recently discovered that adeno-associated virus can be retrogradely transported efficiently from muscle to motor neurons of the spinal cord. We report that insulin-like growth factor 1 prolongs life and delays disease progression, even when delivered at the time of overt disease symptoms.

Original languageEnglish (US)
Pages (from-to)839-842
Number of pages4
JournalScience
Volume301
Issue number5634
DOIs
StatePublished - Aug 8 2003

Fingerprint

Amyotrophic Lateral Sclerosis
Insulin-Like Growth Factor I
Motor Neurons
Dependovirus
Neuromuscular Diseases
Respiratory Muscles
Nerve Growth Factors
Somatomedins
Brain Stem
Atrophy
Disease Progression
Spinal Cord
Extremities
Central Nervous System
Muscles
Therapeutics

ASJC Scopus subject areas

  • General

Cite this

Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. / Kaspar, Brian K.; Lladó, Jerònia; Sherkat, Nushin; Rothstein, Jeffrey D; Gage, Fred H.

In: Science, Vol. 301, No. 5634, 08.08.2003, p. 839-842.

Research output: Contribution to journalArticle

Kaspar, BK, Lladó, J, Sherkat, N, Rothstein, JD & Gage, FH 2003, 'Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model', Science, vol. 301, no. 5634, pp. 839-842. https://doi.org/10.1126/science.1086137
Kaspar, Brian K. ; Lladó, Jerònia ; Sherkat, Nushin ; Rothstein, Jeffrey D ; Gage, Fred H. / Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. In: Science. 2003 ; Vol. 301, No. 5634. pp. 839-842.
@article{ef1353d1efe24c1abe51e1fe60efdea4,
title = "Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model",
abstract = "Amyotrophic lateral sclerosis (ALS) is a progressive, lethal neuromuscular disease that is associated with the degeneration of spinal and brainstem motor neurons, leading to atrophy of limb, axial, and respiratory muscles. The cause of ALS is unknown, and there is no effective therapy. Neurotrophic factors are candidates for therapeutic evaluation in ALS. Although chronic delivery of molecules to the central nervous system has proven difficult, we recently discovered that adeno-associated virus can be retrogradely transported efficiently from muscle to motor neurons of the spinal cord. We report that insulin-like growth factor 1 prolongs life and delays disease progression, even when delivered at the time of overt disease symptoms.",
author = "Kaspar, {Brian K.} and Jer{\`o}nia Llad{\'o} and Nushin Sherkat and Rothstein, {Jeffrey D} and Gage, {Fred H.}",
year = "2003",
month = "8",
day = "8",
doi = "10.1126/science.1086137",
language = "English (US)",
volume = "301",
pages = "839--842",
journal = "Science",
issn = "0036-8075",
publisher = "American Association for the Advancement of Science",
number = "5634",

}

TY - JOUR

T1 - Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model

AU - Kaspar, Brian K.

AU - Lladó, Jerònia

AU - Sherkat, Nushin

AU - Rothstein, Jeffrey D

AU - Gage, Fred H.

PY - 2003/8/8

Y1 - 2003/8/8

N2 - Amyotrophic lateral sclerosis (ALS) is a progressive, lethal neuromuscular disease that is associated with the degeneration of spinal and brainstem motor neurons, leading to atrophy of limb, axial, and respiratory muscles. The cause of ALS is unknown, and there is no effective therapy. Neurotrophic factors are candidates for therapeutic evaluation in ALS. Although chronic delivery of molecules to the central nervous system has proven difficult, we recently discovered that adeno-associated virus can be retrogradely transported efficiently from muscle to motor neurons of the spinal cord. We report that insulin-like growth factor 1 prolongs life and delays disease progression, even when delivered at the time of overt disease symptoms.

AB - Amyotrophic lateral sclerosis (ALS) is a progressive, lethal neuromuscular disease that is associated with the degeneration of spinal and brainstem motor neurons, leading to atrophy of limb, axial, and respiratory muscles. The cause of ALS is unknown, and there is no effective therapy. Neurotrophic factors are candidates for therapeutic evaluation in ALS. Although chronic delivery of molecules to the central nervous system has proven difficult, we recently discovered that adeno-associated virus can be retrogradely transported efficiently from muscle to motor neurons of the spinal cord. We report that insulin-like growth factor 1 prolongs life and delays disease progression, even when delivered at the time of overt disease symptoms.

UR - http://www.scopus.com/inward/record.url?scp=0042528664&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=0042528664&partnerID=8YFLogxK

U2 - 10.1126/science.1086137

DO - 10.1126/science.1086137

M3 - Article

C2 - 12907804

AN - SCOPUS:0042528664

VL - 301

SP - 839

EP - 842

JO - Science

JF - Science

SN - 0036-8075

IS - 5634

ER -