Literature DB >> 34297918

A hop, skip, and a jump to evade USH2A deaf-blindness mutations.

Stéphanie A Mauriac1, Gwenaëlle S G Géléoc2.   

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Year:  2021        PMID： 34297918      PMCID： PMC8353237          DOI： 10.1016/j.ymthe.2021.07.008

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   12.910


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  8 in total

1.  Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy.

Authors:  Hirofumi Komaki; Tetsuya Nagata; Takashi Saito; Satoru Masuda; Eri Takeshita; Masayuki Sasaki; Hisateru Tachimori; Harumasa Nakamura; Yoshitsugu Aoki; Shin'ichi Takeda
Journal:  Sci Transl Med       Date:  2018-04-18       Impact factor: 17.956

2.  Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript.

Authors:  Steve D Wilton; Abbie M Fall; Penny L Harding; Graham McClorey; Catherine Coleman; Susan Fletcher
Journal:  Mol Ther       Date:  2007-02-06       Impact factor: 11.454

3.  Usherin defects lead to early-onset retinal dysfunction in zebrafish.

Authors:  Margo Dona; Ralph Slijkerman; Kimberly Lerner; Sanne Broekman; Jeremy Wegner; Taylor Howat; Theo Peters; Lisette Hetterschijt; Nanda Boon; Erik de Vrieze; Nasrin Sorusch; Uwe Wolfrum; Hannie Kremer; Stephan Neuhauss; Jingjing Zang; Maarten Kamermans; Monte Westerfield; Jennifer Phillips; Erwin van Wijk
Journal:  Exp Eye Res       Date:  2018-05-16       Impact factor: 3.467

4.  In Vivo Assessment of Potential Therapeutic Approaches for USH2A-Associated Diseases.

Authors:  Nachiket D Pendse; Veronica Lamas; Basil S Pawlyk; Morgan L Maeder; Zheng-Yi Chen; Eric A Pierce; Qin Liu
Journal:  Adv Exp Med Biol       Date:  2019       Impact factor: 2.622

5.  Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations.

Authors:  Kalyan Dulla; Ralph Slijkerman; Hester C van Diepen; Silvia Albert; Margo Dona; Wouter Beumer; Janne J Turunen; Hee Lam Chan; Iris A Schulkens; Lars Vorthoren; Cathaline den Besten; Levi Buil; Iris Schmidt; Jiayi Miao; Hanka Venselaar; Jingjing Zang; Stephan C F Neuhauss; Theo Peters; Sanne Broekman; Ronald Pennings; Hannie Kremer; Gerard Platenburg; Peter Adamson; Erik de Vrieze; Erwin van Wijk
Journal:  Mol Ther       Date:  2021-04-23       Impact factor: 12.910

Review 6.  Disease mechanisms and gene therapy for Usher syndrome.

Authors:  Gwenaelle G S Géléoc; Aziz El-Amraoui
Journal:  Hear Res       Date:  2020-03-04       Impact factor: 3.208

7.  Genomic Landscape and Mutational Signatures of Deafness-Associated Genes.

Authors:  Hela Azaiez; Kevin T Booth; Sean S Ephraim; Bradley Crone; Elizabeth A Black-Ziegelbein; Robert J Marini; A Eliot Shearer; Christina M Sloan-Heggen; Diana Kolbe; Thomas Casavant; Michael J Schnieders; Carla Nishimura; Terry Braun; Richard J H Smith
Journal:  Am J Hum Genet       Date:  2018-09-20       Impact factor: 11.025

Review 8.  USH2A-retinopathy: From genetics to therapeutics.

Authors:  Lyes Toualbi; Maria Toms; Mariya Moosajee
Journal:  Exp Eye Res       Date:  2020-10-27       Impact factor: 3.467
  8 in total

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