Literature DB >> 17336076

Neurturin gene therapy improves motor function and prevents death of striatal neurons in a 3-nitropropionic acid rat model of Huntington's disease.

Shilpa Ramaswamy1, Jodi L McBride, Christopher D Herzog, Eugene Brandon, Mehdi Gasmi, Raymond T Bartus, Jeffrey H Kordower.   

Abstract

Huntington's disease (HD) is a devastating neurodegenerative disease characterized by the selective loss of neurons in the striatum and cerebral cortex. This study tested the hypothesis that an adenoassociated viral (AAV2) vector encoding for the trophic factor neurturin (NTN) could provide neuroprotection in the rat 3-nitropropionic acid (3NP) model of HD. Rats received AAV2-NTN (CERE-120), AAV2-eGFP or Vehicle, followed 4 weeks later by the mitochondrial toxin 3NP. 3NP induced motor impairments were observed on the rotarod test, the platform test, and a clinical rating scale in all groups. However, each of these deficits was attenuated by AAV2-NTN (CERE-120). Stereological counts revealed a significant protection of NeuN-ir striatal neurons from 3NP toxicity by AAV2-NTN. These data support the concept that AAV2-NTN might be a valuable treatment for patients with Huntington's disease.

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Year:  2007        PMID: 17336076     DOI: 10.1016/j.nbd.2007.01.003

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


  13 in total

Review 1.  Differential vulnerability of neurons in Huntington's disease: the role of cell type-specific features.

Authors:  Ina Han; YiMei You; Jeffrey H Kordower; Scott T Brady; Gerardo A Morfini
Journal:  J Neurochem       Date:  2010-03-17       Impact factor: 5.372

Review 2.  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 3.  Gene therapy in mouse models of huntington disease.

Authors:  Amber L Southwell; Paul H Patterson
Journal:  Neuroscientist       Date:  2011-04       Impact factor: 7.519

4.  Selective A2A receptor antagonist SCH 58261 modulates striatal oxidative stress and alleviates toxicity induced by 3-Nitropropionic acid in male Wistar rats.

Authors:  Cristiani F Bortolatto; Angélica S Reis; Mikaela P Pinz; Guilherme T Voss; Renata L Oliveira; Ane G Vogt; Silvane Roman; Cristiano R Jesse; Cristiane Luchese; Ethel A Wilhelm
Journal:  Metab Brain Dis       Date:  2017-08-09       Impact factor: 3.584

Review 5.  Large animal models of neurological disorders for gene therapy.

Authors:  Christine Gagliardi; Bruce A Bunnell
Journal:  ILAR J       Date:  2009

Review 6.  Therapeutic approaches to preventing cell death in Huntington disease.

Authors:  Anna Kaplan; Brent R Stockwell
Journal:  Prog Neurobiol       Date:  2012-08-28       Impact factor: 11.685

Review 7.  Advances in gene therapy for movement disorders.

Authors:  Hideki Mochizuki; Toru Yasuda; M Maral Mouradian
Journal:  Neurotherapeutics       Date:  2008-04       Impact factor: 7.620

8.  Quinacrine and Niclosamide Promote Neurite Growth in Midbrain Dopaminergic Neurons Through the Canonical BMP-Smad Pathway and Protect Against Neurotoxin and α-Synuclein-Induced Neurodegeneration.

Authors:  Susan R Goulding; Martin Lévesque; Aideen M Sullivan; Louise M Collins; Gerard W O'Keeffe
Journal:  Mol Neurobiol       Date:  2021-03-12       Impact factor: 5.590

9.  Huntington's Disease: From Mutant Huntingtin Protein to Neurotrophic Factor Therapy.

Authors:  Youssef Sari
Journal:  Int J Biomed Sci       Date:  2011-06

10.  Nucleic Acid-Based Therapy Approaches for Huntington's Disease.

Authors:  Tatyana Vagner; Deborah Young; Alexandre Mouravlev
Journal:  Neurol Res Int       Date:  2012-01-11
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