Literature DB >> 23135503

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Elie Dolgin.   

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Year:  2012        PMID： 23135503     DOI： 10.1038/nm1112-1602

Source DB:  PubMed          Journal:  Nat Med        ISSN： 1078-8956            Impact factor:   53.440


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  7 in total

1.  A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse.

Authors:  Paul N Porensky; Chalermchai Mitrpant; Vicki L McGovern; Adam K Bevan; Kevin D Foust; Brain K Kaspar; Stephen D Wilton; Arthur H M Burghes
Journal:  Hum Mol Genet       Date:  2011-12-20       Impact factor: 6.150

2.  Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.

Authors:  Marco A Passini; Jie Bu; Amy M Richards; Cathrine Kinnecom; S Pablo Sardi; Lisa M Stanek; Yimin Hua; Frank Rigo; John Matson; Gene Hung; Edward M Kaye; Lamya S Shihabuddin; Adrian R Krainer; C Frank Bennett; Seng H Cheng
Journal:  Sci Transl Med       Date:  2011-03-02       Impact factor: 17.956

3.  SMA CARNI-VAL trial part I: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy.

Authors:  Kathryn J Swoboda; Charles B Scott; Thomas O Crawford; Louise R Simard; Sandra P Reyna; Kristin J Krosschell; Gyula Acsadi; Bakri Elsheik; Mary K Schroth; Guy D'Anjou; Bernard LaSalle; Thomas W Prior; Susan L Sorenson; Jo Anne Maczulski; Mark B Bromberg; Gary M Chan; John T Kissel
Journal:  PLoS One       Date:  2010-08-19       Impact factor: 3.240

4.  Effects of 2,4-diaminoquinazoline derivatives on SMN expression and phenotype in a mouse model for spinal muscular atrophy.

Authors:  Matthew E R Butchbach; Jasbir Singh; Margrét Thorsteinsdóttir; Luciano Saieva; Elzbieta Slominski; John Thurmond; Thorkell Andrésson; Jun Zhang; Jonathan D Edwards; Louise R Simard; Livio Pellizzoni; Jill Jarecki; Arthur H M Burghes; Mark E Gurney
Journal:  Hum Mol Genet       Date:  2009-11-06       Impact factor: 6.150

5.  Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model.

Authors:  Yimin Hua; Kentaro Sahashi; Frank Rigo; Gene Hung; Guy Horev; C Frank Bennett; Adrian R Krainer
Journal:  Nature       Date:  2011-10-05       Impact factor: 49.962

6.  SMA CARNIVAL TRIAL PART II: a prospective, single-armed trial of L-carnitine and valproic acid in ambulatory children with spinal muscular atrophy.

Authors:  John T Kissel; Charles B Scott; Sandra P Reyna; Thomas O Crawford; Louise R Simard; Kristin J Krosschell; Gyula Acsadi; Bakri Elsheik; Mary K Schroth; Guy D'Anjou; Bernard LaSalle; Thomas W Prior; Susan Sorenson; Jo Anne Maczulski; Mark B Bromberg; Gary M Chan; Kathryn J Swoboda
Journal:  PLoS One       Date:  2011-07-06       Impact factor: 3.240

7.  Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.

Authors:  Kevin D Foust; Xueyong Wang; Vicki L McGovern; Lyndsey Braun; Adam K Bevan; Amanda M Haidet; Thanh T Le; Pablo R Morales; Mark M Rich; Arthur H M Burghes; Brian K Kaspar
Journal:  Nat Biotechnol       Date:  2010-02-28       Impact factor: 68.164
  7 in total
  2 in total

1.  Biotech comes to its 'antisenses' after hard-won drug approval.

Authors:  Kevin Jiang
Journal:  Nat Med       Date:  2013-03       Impact factor: 53.440

2.  An Integrative Transcriptomic Analysis for Identifying Novel Target Genes Corresponding to Severity Spectrum in Spinal Muscular Atrophy.

Authors:  Chung-Wei Yang; Chien-Lin Chen; Wei-Chun Chou; Ho-Chen Lin; Yuh-Jyh Jong; Li-Kai Tsai; Chun-Yu Chuang
Journal:  PLoS One       Date:  2016-06-22       Impact factor: 3.240
  2 in total

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