Literature DB >> 22222669

Gene therapy for Huntington's disease.

Shilpa Ramaswamy1, Jeffrey H Kordower.   

Abstract

Huntington's disease (HD) is a neurodegenerative disease for which there is no cure. Therapies that are efficacious in animal models have to date shown benefit for humans. One potential powerful approach is gene therapy. The ideal method of administration of gene therapy has been hotly debated and viral vectors have provided one method of long-term and wide-spread delivery to the brain. Trophic factors to protect cells from degeneration and RNAi to reduce mutant huntingtin (mHtt) protein expression are 2 main classes of compounds that demonstrate benefit in animal models. This review will examine some commonly used adeno-associated viral (AAV) vectors and discuss some therapies that hold promise for HD.
Copyright © 2011. Published by Elsevier Inc.

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Year:  2011        PMID： 22222669     DOI： 10.1016/j.nbd.2011.12.030

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


  22 in total

1.  Inhibition of astrocyte FAK-JNK signaling promotes subventricular zone neurogenesis through CNTF.

Authors:  Cuihong Jia; Matthew P Keasey; Chiharu Lovins; Theo Hagg
Journal:  Glia       Date:  2018-11       Impact factor: 7.452

2.  Intrajugular vein delivery of AAV9-RNAi prevents neuropathological changes and weight loss in Huntington's disease mice.

Authors:  Brett D Dufour; Catherine A Smith; Randall L Clark; Timothy R Walker; Jodi L McBride
Journal:  Mol Ther       Date:  2014-01-06       Impact factor: 11.454

3.  Transduction of the central nervous system after intracerebroventricular injection of adeno-associated viral vectors in neonatal and juvenile mice.

Authors:  Shervin Gholizadeh; Sujeenthar Tharmalingam; Margarita E Macaldaz; David R Hampson
Journal:  Hum Gene Ther Methods       Date:  2013-08-03       Impact factor: 2.396

Review 4.  Beyond traditional pharmacology: new tools and approaches.

Authors:  E V Gurevich; V V Gurevich
Journal:  Br J Pharmacol       Date:  2015-06-10       Impact factor: 8.739

5.  Nanomaterial based drug delivery systems for the treatment of neurodegenerative diseases.

Authors:  Shima Masoudi Asil; Jyoti Ahlawat; Gileydis Guillama Barroso; Mahesh Narayan
Journal:  Biomater Sci       Date:  2020-07-08       Impact factor: 6.843

Review 6.  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

7.  Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice.

Authors:  Mireia Garriga-Canut; Carmen Agustín-Pavón; Frank Herrmann; Aurora Sánchez; Mara Dierssen; Cristina Fillat; Mark Isalan
Journal:  Proc Natl Acad Sci U S A       Date:  2012-10-10       Impact factor: 11.205

8.  Interaction between nonviral reprogrammed fibroblast stem cells and trophic factors for brain repair.

Authors:  G Liu; H Anisman; J Bobyn; S Hayley
Journal:  Mol Neurobiol       Date:  2014-03-28       Impact factor: 5.590

Review 9.  BDNF mediates adaptive brain and body responses to energetic challenges.

Authors:  Krisztina Marosi; Mark P Mattson
Journal:  Trends Endocrinol Metab       Date:  2013-12-19       Impact factor: 12.015

10.  AAV9 delivering a modified human Mullerian inhibiting substance as a gene therapy in patient-derived xenografts of ovarian cancer.

Authors:  David Pépin; Amanda Sosulski; Lihua Zhang; Dan Wang; Vinod Vathipadiekal; Katherine Hendren; Caroline M Coletti; Aaron Yu; Cesar M Castro; Michael J Birrer; Guangping Gao; Patricia K Donahoe
Journal:  Proc Natl Acad Sci U S A       Date:  2015-07-27       Impact factor: 11.205
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