Literature DB >> 27078106

Gene therapy, CMT1X, and the inherited neuropathies.

Michael E Shy1.   

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Year:  2016        PMID： 27078106      PMCID： PMC4855541          DOI： 10.1073/pnas.1604005113

Source DB:  PubMed          Journal:  Proc Natl Acad Sci U S A        ISSN： 0027-8424            Impact factor:   11.205


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  18 in total

Review 1.  Characterization of targeted connexin32-deficient mice: a model for the human Charcot-Marie-Tooth (X-type) inherited disease.

Authors:  K Willecke; A Temme; B Teubner; T Ott
Journal:  Ann N Y Acad Sci       Date:  1999-09-14       Impact factor: 5.691

2.  Fates of neurotrophins after retrograde axonal transport: phosphorylation of p75NTR is a sorting signal for delayed degradation.

Authors:  Rafal Butowt; Christopher S von Bartheld
Journal:  J Neurosci       Date:  2009-08-26       Impact factor: 6.167

3.  In vivo introduction of transgenes into mouse sciatic nerve cells in situ using viral vectors.

Authors:  Sergio Gonzalez; Ruani N Fernando; Claire Perrin-Tricaud; Nicolas Tricaud
Journal:  Nat Protoc       Date:  2014-04-24       Impact factor: 13.491

4.  In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.

Authors:  Christopher E Nelson; Chady H Hakim; David G Ousterout; Pratiksha I Thakore; Eirik A Moreb; Ruth M Castellanos Rivera; Sarina Madhavan; Xiufang Pan; F Ann Ran; Winston X Yan; Aravind Asokan; Feng Zhang; Dongsheng Duan; Charles A Gersbach
Journal:  Science       Date:  2015-12-31       Impact factor: 47.728

5.  MpzR98C arrests Schwann cell development in a mouse model of early-onset Charcot-Marie-Tooth disease type 1B.

Authors:  Mario A C Saporta; Brian R Shy; Agnes Patzko; Yunhong Bai; Maria Pennuto; Cinzia Ferri; Elisa Tinelli; Paola Saveri; Dan Kirschner; Michelle Crowther; Cherie Southwood; Xingyao Wu; Alexander Gow; M Laura Feltri; Lawrence Wrabetz; Michael E Shy
Journal:  Brain       Date:  2012-06-10       Impact factor: 13.501

6.  Intraneural GJB1 gene delivery improves nerve pathology in a model of X-linked Charcot-Marie-Tooth disease.

Authors:  Irene Sargiannidou; Alexia Kagiava; Stavros Bashiardes; Jan Richter; Christina Christodoulou; Steven S Scherer; Kleopas A Kleopa
Journal:  Ann Neurol       Date:  2015-06-30       Impact factor: 10.422

7.  Mitogen-expanded Schwann cells retain the capacity to myelinate regenerating axons after transplantation into rat sciatic nerve.

Authors:  M L Feltri; S S Scherer; L Wrabetz; J Kamholz; M E Shy
Journal:  Proc Natl Acad Sci U S A       Date:  1992-09-15       Impact factor: 11.205

8.  Ablation of the UPR-mediator CHOP restores motor function and reduces demyelination in Charcot-Marie-Tooth 1B mice.

Authors:  Maria Pennuto; Elisa Tinelli; Mariachiara Malaguti; Ubaldo Del Carro; Maurizio D'Antonio; David Ron; Angelo Quattrini; M Laura Feltri; Lawrence Wrabetz
Journal:  Neuron       Date:  2008-02-07       Impact factor: 17.173

9.  CMT subtypes and disease burden in patients enrolled in the Inherited Neuropathies Consortium natural history study: a cross-sectional analysis.

Authors:  V Fridman; B Bundy; M M Reilly; D Pareyson; C Bacon; J Burns; J Day; S Feely; R S Finkel; T Grider; C A Kirk; D N Herrmann; M Laurá; J Li; T Lloyd; C J Sumner; F Muntoni; G Piscosquito; S Ramchandren; R Shy; C E Siskind; S W Yum; I Moroni; E Pagliano; S Zuchner; S S Scherer; M E Shy
Journal:  J Neurol Neurosurg Psychiatry       Date:  2014-11-27       Impact factor: 10.154

10.  Comparative analysis of HIV-1-based lentiviral vectors bearing lyssavirus glycoproteins for neuronal gene transfer.

Authors:  Thais Federici; Robert Kutner; Xian-Yang Zhang; Hitoshi Kuroda; Noël Tordo; Nicholas M Boulis; Jakob Reiser
Journal:  Genet Vaccines Ther       Date:  2009-01-13
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