Literature DB >> 21784507

Exon-skipping therapy for Duchenne muscular dystrophy.

Akinori Nakamura1, Shin'ichi Takeda.   

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Year:  2011        PMID： 21784507     DOI： 10.1016/S0140-6736(11)61028-3

Source DB:  PubMed          Journal:  Lancet        ISSN： 0140-6736            Impact factor:   79.321


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  7 in total

Review 1.  Moving towards successful exon-skipping therapy for Duchenne muscular dystrophy.

Authors:  Akinori Nakamura
Journal:  J Hum Genet       Date:  2017-06-01       Impact factor: 3.172

2.  Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.

Authors:  Yoshitsugu Aoki; Toshifumi Yokota; Tetsuya Nagata; Akinori Nakamura; Jun Tanihata; Takashi Saito; Stephanie M R Duguez; Kanneboyina Nagaraju; Eric P Hoffman; Terence Partridge; Shin'ichi Takeda
Journal:  Proc Natl Acad Sci U S A       Date:  2012-08-06       Impact factor: 11.205

3.  Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair.

Authors:  Kunwoo Lee; Michael Conboy; Hyo Min Park; Fuguo Jiang; Hyun Jin Kim; Mark A Dewitt; Vanessa A Mackley; Kevin Chang; Anirudh Rao; Colin Skinner; Tamanna Shobha; Melod Mehdipour; Hui Liu; Wen-Chin Huang; Freeman Lan; Nicolas L Bray; Song Li; Jacob E Corn; Kazunori Kataoka; Jennifer A Doudna; Irina Conboy; Niren Murthy
Journal:  Nat Biomed Eng       Date:  2017-10-02       Impact factor: 25.671

4.  Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice.

Authors:  Yoshitsugu Aoki; Tetsuya Nagata; Toshifumi Yokota; Akinori Nakamura; Matthew J A Wood; Terence Partridge; Shin'ichi Takeda
Journal:  Hum Mol Genet       Date:  2013-07-23       Impact factor: 6.150

Review 5.  RNA Targeting in Inherited Neuromuscular Disorders: Novel Therapeutic Strategies to Counteract Mis-Splicing.

Authors:  Veronica Verdile; Gloria Guizzo; Gabriele Ferrante; Maria Paola Paronetto
Journal:  Cells       Date:  2021-10-22       Impact factor: 6.600

Review 6.  RNA-based therapeutics: an overview and prospectus.

Authors:  Yiran Zhu; Liyuan Zhu; Xian Wang; Hongchuan Jin
Journal:  Cell Death Dis       Date:  2022-07-23       Impact factor: 9.685

Review 7.  The emerging agenda of stratified medicine in neurology.

Authors:  Paul M Matthews; Paul Edison; Olivia C Geraghty; Michael R Johnson
Journal:  Nat Rev Neurol       Date:  2013-12-10       Impact factor: 42.937
  7 in total

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