Literature DB >> 19135533

Intraspinal cord delivery of IGF-I mediated by adeno-associated virus 2 is neuroprotective in a rat model of familial ALS.

Colin K Franz1, Thais Federici, Jun Yang, Carey Backus, Sang Su Oh, Qingshan Teng, Erin Carlton, Kathie M Bishop, Mehdi Gasmi, Raymond T Bartus, Eva L Feldman, Nicholas M Boulis.   

Abstract

BACKGROUND: Amyotrophic lateral sclerosis (ALS) is a devastating disease that is characterized by the progressive loss of motor neurons. Patients with ALS usually die from respiratory failure due to respiratory muscle paralysis. Consequently, therapies aimed at preserving segmental function of the respiratory motor neurons could extend life for these patients. Insulin-like growth factor-I (IGF-I) is known to be a potent survival factor for motor neurons. In this study we induced high levels of IGF-I expression in the cervical spinal cord of hSOD1(G93A) rats with intraspinal cord (ISC) injections of an adeno-associated virus serotype 2 vector (CERE-130). This approach reduced the extent of motor neuron loss in the treated segments of the spinal cord. However, a corresponding preservation of motor function was observed in male, but not female, hSOD1(G93A) rats. We conclude that ISC injection of CERE-130 has the potential to protect motor neurons and preserve neuromuscular function in ALS.

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Year:  2008        PMID: 19135533     DOI: 10.1016/j.nbd.2008.12.003

Source DB:  PubMed          Journal:  Neurobiol Dis        ISSN: 0969-9961            Impact factor:   5.996


  24 in total

Review 1.  Viral vectors for neurotrophic factor delivery: a gene therapy approach for neurodegenerative diseases of the CNS.

Authors:  Seung T Lim; Mikko Airavaara; Brandon K Harvey
Journal:  Pharmacol Res       Date:  2009-10-17       Impact factor: 7.658

Review 2.  Progress in gene therapy for neurological disorders.

Authors:  Michele Simonato; Jean Bennett; Nicholas M Boulis; Maria G Castro; David J Fink; William F Goins; Steven J Gray; Pedro R Lowenstein; Luk H Vandenberghe; Thomas J Wilson; John H Wolfe; Joseph C Glorioso
Journal:  Nat Rev Neurol       Date:  2013-04-23       Impact factor: 42.937

Review 3.  Pharmacology of manipulating lean body mass.

Authors:  Patricio V Sepulveda; Ernest D Bush; Keith Baar
Journal:  Clin Exp Pharmacol Physiol       Date:  2015-01       Impact factor: 2.557

Review 4.  Antioxidant gene therapy against neuronal cell death.

Authors:  Juliana Navarro-Yepes; Laura Zavala-Flores; Annadurai Anandhan; Fang Wang; Maciej Skotak; Namas Chandra; Ming Li; Aglaia Pappa; Daniel Martinez-Fong; Luz Maria Del Razo; Betzabet Quintanilla-Vega; Rodrigo Franco
Journal:  Pharmacol Ther       Date:  2013-12-12       Impact factor: 12.310

Review 5.  Dysregulation of IGF-1/GLP-1 signaling in the progression of ALS: potential target activators and influences on neurological dysfunctions.

Authors:  Ambika Shandilya; Sidharth Mehan
Journal:  Neurol Sci       Date:  2021-05-21       Impact factor: 3.307

6.  Neuroprotection by gene therapy targeting mutant SOD1 in individual pools of motor neurons does not translate into therapeutic benefit in fALS mice.

Authors:  Chris Towne; Veronica Setola; Bernard L Schneider; Patrick Aebischer
Journal:  Mol Ther       Date:  2010-11-23       Impact factor: 11.454

7.  Neurotrophic growth factors for the treatment of amyotrophic lateral sclerosis: where do we stand?

Authors:  Alexandre Henriques; Claudia Pitzer; Armin Schneider
Journal:  Front Neurosci       Date:  2010-06-11       Impact factor: 4.677

Review 8.  Potential new complication in drug therapy development for amyotrophic lateral sclerosis.

Authors:  Svitlana Garbuzova-Davis; Avery Thomson; Crupa Kurien; R Douglas Shytle; Paul R Sanberg
Journal:  Expert Rev Neurother       Date:  2016-07-15       Impact factor: 4.618

Review 9.  Viral vectors and delivery strategies for CNS gene therapy.

Authors:  Steven J Gray; Kenton T Woodard; R Jude Samulski
Journal:  Ther Deliv       Date:  2010-10

10.  A conditioning lesion provides selective protection in a rat model of Amyotrophic Lateral Sclerosis.

Authors:  Colin K Franz; Eric T Quach; Christina A Krudy; Thais Federici; Michele A Kliem; Brooke R Snyder; Bethwel Raore; Nicholas M Boulis
Journal:  PLoS One       Date:  2009-10-06       Impact factor: 3.240

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