Literature DB >> 22215052

Antisense oligonucleotides shed new light on the pathogenesis and treatment of spinal muscular atrophy.

Jiing-Kuan Yee1, Ren-Jang Lin.   

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Year:  2012        PMID： 22215052      PMCID： PMC3255585          DOI： 10.1038/mt.2011.275

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


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  14 in total

Review 1.  Spinal muscular atrophy: a deficiency in a ubiquitous protein; a motor neuron-specific disease.

Authors:  Umrao R Monani
Journal:  Neuron       Date:  2005-12-22       Impact factor: 17.173

2.  Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model.

Authors:  Yimin Hua; Kentaro Sahashi; Gene Hung; Frank Rigo; Marco A Passini; C Frank Bennett; Adrian R Krainer
Journal:  Genes Dev       Date:  2010-07-12       Impact factor: 11.361

3.  IGF-I is required for normal embryonic growth in mice.

Authors:  L Powell-Braxton; P Hollingshead; C Warburton; M Dowd; S Pitts-Meek; D Dalton; N Gillett; T A Stewart
Journal:  Genes Dev       Date:  1993-12       Impact factor: 11.361

4.  CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy.

Authors:  Marco A Passini; Jie Bu; Eric M Roskelley; Amy M Richards; S Pablo Sardi; Catherine R O'Riordan; Katherine W Klinger; Lamya S Shihabuddin; Seng H Cheng
Journal:  J Clin Invest       Date:  2010-03-15       Impact factor: 14.808

5.  Splicing of a critical exon of human Survival Motor Neuron is regulated by a unique silencer element located in the last intron.

Authors:  Nirmal K Singh; Natalia N Singh; Elliot J Androphy; Ravindra N Singh
Journal:  Mol Cell Biol       Date:  2006-02       Impact factor: 4.272

6.  Combination of SMN trans-splicing and a neurotrophic factor increases the life span and body mass in a severe model of spinal muscular atrophy.

Authors:  Monir Shababi; Jacqueline Glascock; Christian L Lorson
Journal:  Hum Gene Ther       Date:  2010-12-19       Impact factor: 5.695

7.  Induced pluripotent stem cells from a spinal muscular atrophy patient.

Authors:  Allison D Ebert; Junying Yu; Ferrill F Rose; Virginia B Mattis; Christian L Lorson; James A Thomson; Clive N Svendsen
Journal:  Nature       Date:  2008-12-21       Impact factor: 49.962

Review 8.  Spinal muscular atrophy.

Authors:  Mitchell R Lunn; Ching H Wang
Journal:  Lancet       Date:  2008-06-21       Impact factor: 79.321

Review 9.  Human acid-labile subunit deficiency: clinical, endocrine and metabolic consequences.

Authors:  Horacio M Domené; Vivian Hwa; Jesús Argente; Jan M Wit; Jaan M Wit; Cecilia Camacho-Hübner; Héctor G Jasper; Jesús Pozo; Hermine A van Duyvenvoorde; Shoshana Yakar; Olga V Fofanova-Gambetti; Ron G Rosenfeld
Journal:  Horm Res       Date:  2009-09-01

Review 10.  RNA and disease.

Authors:  Thomas A Cooper; Lili Wan; Gideon Dreyfuss
Journal:  Cell       Date:  2009-02-20       Impact factor: 41.582
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  2 in total

Review 1.  Overcoming Gene-Delivery Hurdles: Physiological Considerations for Nonviral Vectors.

Authors:  Andrew B Hill; Mingfu Chen; Chih-Kuang Chen; Blaine A Pfeifer; Charles H Jones
Journal:  Trends Biotechnol       Date:  2015-12-23       Impact factor: 19.536

2.  Spinal muscular atrophy: therapeutic strategies.

Authors:  Diana Castro; Susan T Iannaccone
Journal:  Curr Treat Options Neurol       Date:  2014-11       Impact factor: 3.598
  2 in total

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