Literature DB >> 26633828

Moving toward a gene therapy for Huntington's disease.

J C Glorioso1, J B Cohen1, D L Carlisle2, I Munoz-Sanjuan3, R M Friedlander2.   

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Year:  2015        PMID： 26633828     DOI： 10.1038/gt.2015.102

Source DB:  PubMed          Journal:  Gene Ther        ISSN： 0969-7128            Impact factor:   5.250


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  43 in total

Review 1.  Complexity and heterogeneity: what drives the ever-changing brain in Huntington's disease?

Authors:  H Diana Rosas; David H Salat; Stephanie Y Lee; Alexandra K Zaleta; Nathanael Hevelone; Steven M Hersch
Journal:  Ann N Y Acad Sci       Date:  2008-12       Impact factor: 5.691

2.  RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model.

Authors:  Scott Q Harper; Patrick D Staber; Xiaohua He; Steven L Eliason; Inês H Martins; Qinwen Mao; Linda Yang; Robert M Kotin; Henry L Paulson; Beverly L Davidson
Journal:  Proc Natl Acad Sci U S A       Date:  2005-04-05       Impact factor: 11.205

3.  Recombinant variant of ciliary neurotrophic factor for weight loss in obese adults: a randomized, dose-ranging study.

Authors:  Mark P Ettinger; Thomas W Littlejohn; Sherwyn L Schwartz; Stuart R Weiss; Harris H McIlwain; Steven B Heymsfield; George A Bray; William G Roberts; Eugene R Heyman; Nancy Stambler; Stanley Heshka; Catherine Vicary; Hans-Peter Guler
Journal:  JAMA       Date:  2003-04-09       Impact factor: 56.272

4.  Interventional MRI-guided putaminal delivery of AAV2-GDNF for a planned clinical trial in Parkinson's disease.

Authors:  R Mark Richardson; Adrian P Kells; Kathryn H Rosenbluth; Ernesto Aguilar Salegio; Massimo S Fiandaca; Paul S Larson; Philip A Starr; Alastair J Martin; Russell R Lonser; Howard J Federoff; John R Forsayeth; Krystof S Bankiewicz
Journal:  Mol Ther       Date:  2011-02-22       Impact factor: 11.454

Review 5.  Animal models of Huntington's disease.

Authors:  Shilpa Ramaswamy; Jodi L McBride; Jeffrey H Kordower
Journal:  ILAR J       Date:  2007

6.  A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference.

Authors:  Maria Stella Lombardi; Leonie Jaspers; Christine Spronkmans; Cinzia Gellera; Franco Taroni; Emilio Di Maria; Stefano Di Donato; William F Kaemmerer
Journal:  Exp Neurol       Date:  2009-03-13       Impact factor: 5.330

7.  Allele-specific silencing of mutant Huntington's disease gene.

Authors:  Yu Zhang; Joshua Engelman; Robert M Friedlander
Journal:  J Neurochem       Date:  2009-01       Impact factor: 5.372

8.  Intrastriatal CERE-120 (AAV-Neurturin) protects striatal and cortical neurons and delays motor deficits in a transgenic mouse model of Huntington's disease.

Authors:  Shilpa Ramaswamy; Jodi L McBride; Ina Han; Elizabeth M Berry-Kravis; Lili Zhou; Christopher D Herzog; Mehdi Gasmi; Raymond T Bartus; Jeffrey H Kordower
Journal:  Neurobiol Dis       Date:  2008-12-25       Impact factor: 5.996

9.  Inhibition of mitochondrial protein import by mutant huntingtin.

Authors:  Hiroko Yano; Sergei V Baranov; Oxana V Baranova; Jinho Kim; Yanchun Pan; Svitlana Yablonska; Diane L Carlisle; Robert J Ferrante; Albert H Kim; Robert M Friedlander
Journal:  Nat Neurosci       Date:  2014-05-18       Impact factor: 24.884

10.  Adeno-associated virus type 6 is retrogradely transported in the non-human primate brain.

Authors:  W San Sebastian; L Samaranch; G Heller; A P Kells; J Bringas; P Pivirotto; J Forsayeth; K S Bankiewicz
Journal:  Gene Ther       Date:  2013-09-26       Impact factor: 5.250
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  6 in total

1.  Why Gene Editors Like CRISPR/Cas May Be a Game-Changer for Neuroweapons.

Authors:  Diane DiEuliis; James Giordano
Journal:  Health Secur       Date:  2017-06-02

Review 2.  Herpes Simplex Virus Vectors for Gene Transfer to the Central Nervous System.

Authors:  Sara Artusi; Yoshitaka Miyagawa; William F Goins; Justus B Cohen; Joseph C Glorioso
Journal:  Diseases       Date:  2018-08-14

Review 3.  Applying switchable Cas9 variants to in vivo gene editing for therapeutic applications.

Authors:  Emily M Mills; Victoria L Barlow; Louis Y P Luk; Yu-Hsuan Tsai
Journal:  Cell Biol Toxicol       Date:  2019-08-15       Impact factor: 6.691

4.  GAPDH controls extracellular vesicle biogenesis and enhances the therapeutic potential of EV mediated siRNA delivery to the brain.

Authors:  Ghulam Hassan Dar; Cláudia C Mendes; Wei-Li Kuan; Alfina A Speciale; Mariana Conceição; André Görgens; Inna Uliyakina; Miguel J Lobo; Wooi F Lim; Samir El Andaloussi; Imre Mäger; Thomas C Roberts; Roger A Barker; Deborah C I Goberdhan; Clive Wilson; Matthew J A Wood
Journal:  Nat Commun       Date:  2021-11-18       Impact factor: 14.919

5.  Gene Therapy for Huntington's Disease: The Final Strategy for a Cure?

Authors:  Seulgi Byun; Mijung Lee; Manho Kim
Journal:  J Mov Disord       Date:  2021-11-17

6.  Rethinking Functional Outcome Measures: The Development of a Novel Upper Limb Token Transfer Test to Assess Basal Ganglia Dysfunction.

Authors:  Susanne P Clinch; Monica Busse; Mariah J Lelos; Anne E Rosser
Journal:  Front Neurosci       Date:  2018-05-30       Impact factor: 5.152
  6 in total

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