Literature DB >> 16775380

Therapeutic strategies for the inherited neuropathies.

Michael E Shy1.   

Abstract

More than 30 genetic causes have been identified for the inherited neuropathies collectively referred to as Charcot-Marie-Tooth (CMT) disease. Previous therapies for CMT were limited to traditional approaches such as rehabilitation medicine, ambulation aids, and pain management. Identification of the genes causing CMT has led to improved genetic counseling and assistance in family planning. Identification of these genes is beginning to delineate common molecular pathways in multiple forms of CMT that can be exploited in future molecular therapies. Scientifically based clinical trials for CMT are currently being implemented. Techniques of gene therapy are advancing to the point that they may become feasible options for patients with CMT and other neurodegenerative diseases.

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Year:  2006        PMID： 16775380     DOI： 10.1385/nmm:8:1-2:255

Source DB:  PubMed          Journal:  Neuromolecular Med        ISSN： 1535-1084            Impact factor:   3.843


  133 in total

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Authors:  H Chao; Y Liu; J Rabinowitz; C Li; R J Samulski; C E Walsh
Journal:  Mol Ther       Date:  2000-12       Impact factor: 11.454

Review 2.  Is the therapeutic application of neurotrophic factors dead?

Authors:  Stuart C Apfel
Journal:  Ann Neurol       Date:  2002-01       Impact factor: 10.422

3.  Mutations in the mitochondrial GTPase mitofusin 2 cause Charcot-Marie-Tooth neuropathy type 2A.

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Journal:  Nat Genet       Date:  2004-04-04       Impact factor: 38.330

Review 4.  Expression of heterologous sequences in adenoviral vectors.

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Journal:  Curr Top Microbiol Immunol       Date:  1992       Impact factor: 4.291

5.  Effective gene transfer of lacZ and P0 into Schwann cells of P0-deficient mice.

Authors:  V Guénard; B Schweitzer; E Flechsig; S Hemmi; R Martini; U Suter; M Schachner
Journal:  Glia       Date:  1999-01-15       Impact factor: 7.452

6.  Curcumin treatment abrogates endoplasmic reticulum retention and aggregation-induced apoptosis associated with neuropathy-causing myelin protein zero-truncating mutants.

Authors:  Mehrdad Khajavi; Ken Inoue; Wojciech Wiszniewski; Tomoko Ohyama; G Jackson Snipes; James R Lupski
Journal:  Am J Hum Genet       Date:  2005-09-30       Impact factor: 11.025

7.  Reliability and validity of the CMT neuropathy score as a measure of disability.

Authors:  M E Shy; J Blake; K Krajewski; D R Fuerst; M Laura; A F Hahn; J Li; R A Lewis; M Reilly
Journal:  Neurology       Date:  2005-04-12       Impact factor: 9.910

8.  Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2.

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Journal:  J Virol       Date:  1998-01       Impact factor: 5.103

9.  A leucine-to-proline mutation in the putative first transmembrane domain of the 22-kDa peripheral myelin protein in the trembler-J mouse.

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Journal:  Proc Natl Acad Sci U S A       Date:  1992-05-15       Impact factor: 11.205

10.  VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model.

Authors:  Mimoun Azzouz; G Scott Ralph; Erik Storkebaum; Lucy E Walmsley; Kyriacos A Mitrophanous; Susan M Kingsman; Peter Carmeliet; Nicholas D Mazarakis
Journal:  Nature       Date:  2004-05-27       Impact factor: 49.962
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  10 in total

1.  The role of gap junctions in Charcot-Marie-Tooth disease.

Authors:  Kleopas A Kleopa
Journal:  J Neurosci       Date:  2011-12-07       Impact factor: 6.167

Review 2.  Charcot-Marie-Tooth syndrome and neurofibromatosis type 1 with multiple neurofibromas of the entire spinal nerve roots.

Authors:  David O Onu; Andrew W Hunn; Jens Peters-Willke
Journal:  BMJ Case Rep       Date:  2013-07-13

Review 3.  Signals to promote myelin formation and repair.

Authors:  Carla Taveggia; Maria Laura Feltri; Lawrence Wrabetz
Journal:  Nat Rev Neurol       Date:  2010-04-20       Impact factor: 42.937

4.  Loss of the inactive myotubularin-related phosphatase Mtmr13 leads to a Charcot-Marie-Tooth 4B2-like peripheral neuropathy in mice.

Authors:  Fred L Robinson; Ingrid R Niesman; Kristina K Beiswenger; Jack E Dixon
Journal:  Proc Natl Acad Sci U S A       Date:  2008-03-18       Impact factor: 11.205

Review 5.  Experimental therapeutics in hereditary neuropathies: the past, the present, and the future.

Authors:  David N Herrmann
Journal:  Neurotherapeutics       Date:  2008-10       Impact factor: 7.620

Review 6.  Promoting peripheral myelin repair.

Authors:  Ye Zhou; Lucia Notterpek
Journal:  Exp Neurol       Date:  2016-04-11       Impact factor: 5.330

7.  Inherited neuropathies.

Authors:  Angelo Schenone; Lucilla Nobbio; Margherita Monti Bragadin; Giulia Ursino; Marina Grandis
Journal:  Curr Treat Options Neurol       Date:  2011-04       Impact factor: 3.598

Review 8.  Molecular mechanisms of inherited demyelinating neuropathies.

Authors:  Steven S Scherer; Lawrence Wrabetz
Journal:  Glia       Date:  2008-11-01       Impact factor: 8.073

Review 9.  Pain Phenotypes in Rare Musculoskeletal and Neuromuscular Diseases.

Authors:  Anthony Tucker-Bartley; Jordan Lemme; Andrea Gomez-Morad; Nehal Shah; Miranda Veliu; Frank Birklein; Claudia Storz; Seward Rutkove; David Kronn; Alison M Boyce; Eduard Kraft; Jaymin Upadhyay
Journal:  Neurosci Biobehav Rev       Date:  2021-02-10       Impact factor: 9.052

Review 10.  Mitochondrial Dynamics: Molecular Mechanisms, Related Primary Mitochondrial Disorders and Therapeutic Approaches.

Authors:  Michela Di Nottia; Daniela Verrigni; Alessandra Torraco; Teresa Rizza; Enrico Bertini; Rosalba Carrozzo
Journal:  Genes (Basel)       Date:  2021-02-10       Impact factor: 4.096
  10 in total

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