Literature DB >> 34554886

A Cautiously Optimistic Outlook of a Designer Therapy for 1% of Duchenne Muscular Dystrophy Patients.

Dongsheng Duan1,2,3,4.   

Abstract

Entities:  

Mesh：

Year:  2021        PMID： 34554886      PMCID： PMC8575061          DOI： 10.1089/hum.2021.29179.ddu

Source DB:  PubMed          Journal:  Hum Gene Ther        ISSN： 1043-0342            Impact factor:   4.793


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  15 in total

Review 1.  Therapeutic developments for Duchenne muscular dystrophy.

Authors:  Ingrid E C Verhaart; Annemieke Aartsma-Rus
Journal:  Nat Rev Neurol       Date:  2019-07       Impact factor: 42.937

2.  Micro-Dystrophin Gene Therapy Goes Systemic in Duchenne Muscular Dystrophy Patients.

Authors:  Dongsheng Duan
Journal:  Hum Gene Ther       Date:  2018-04-05       Impact factor: 5.695

3.  Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.

Authors:  Jerry R Mendell; Samiah Al-Zaidy; Richard Shell; W Dave Arnold; Louise R Rodino-Klapac; Thomas W Prior; Linda Lowes; Lindsay Alfano; Katherine Berry; Kathleen Church; John T Kissel; Sukumar Nagendran; James L'Italien; Douglas M Sproule; Courtney Wells; Jessica A Cardenas; Marjet D Heitzer; Allan Kaspar; Sarah Corcoran; Lyndsey Braun; Shibi Likhite; Carlos Miranda; Kathrin Meyer; K D Foust; Arthur H M Burghes; Brian K Kaspar
Journal:  N Engl J Med       Date:  2017-11-02       Impact factor: 91.245

Review 4.  Duchenne muscular dystrophy.

Authors:  Dongsheng Duan; Nathalie Goemans; Shin'ichi Takeda; Eugenio Mercuri; Annemieke Aartsma-Rus
Journal:  Nat Rev Dis Primers       Date:  2021-02-18       Impact factor: 52.329

5.  Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN.

Authors:  Christian Hinderer; Nathan Katz; Elizabeth L Buza; Cecilia Dyer; Tamara Goode; Peter Bell; Laura K Richman; James M Wilson
Journal:  Hum Gene Ther       Date:  2018-02-12       Impact factor: 5.695

6.  Toward the correction of muscular dystrophy by gene editing.

Authors:  Eric N Olson
Journal:  Proc Natl Acad Sci U S A       Date:  2021-04-30       Impact factor: 11.205

7.  MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates.

Authors:  Juliette Hordeaux; Elizabeth L Buza; Brianne Jeffrey; Chunjuan Song; Tahsin Jahan; Yuan Yuan; Yanqing Zhu; Peter Bell; Mingyao Li; Jessica A Chichester; Roberto Calcedo; James M Wilson
Journal:  Sci Transl Med       Date:  2020-11-11       Impact factor: 17.956

8.  Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology.

Authors:  Juliette Hordeaux; Elizabeth L Buza; Cecilia Dyer; Tamara Goode; Thomas W Mitchell; Laura Richman; Nathan Denton; Christian Hinderer; Nathan Katz; Ralf Schmid; Rod Miller; Gourav R Choudhury; Makoto Horiuchi; Kalyani Nambiar; Hanying Yan; Mingyao Li; James M Wilson
Journal:  Hum Gene Ther       Date:  2020-07-31       Impact factor: 5.695

9.  Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping.

Authors:  Liubov V Gushchina; Emma C Frair; Natalie Rohan; Adrienne J Bradley; Tabatha R Simmons; Hemantkumar D Chavan; Hsin-Jung Chou; Michelle Eggers; Megan A Waldrop; Nicolas Wein; Kevin M Flanigan
Journal:  Hum Gene Ther       Date:  2021-05-07       Impact factor: 5.695

10.  Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.

Authors:  Nicolas Wein; Adeline Vulin; Maria S Falzarano; Christina Al-Khalili Szigyarto; Baijayanta Maiti; Andrew Findlay; Kristin N Heller; Mathias Uhlén; Baskar Bakthavachalu; Sonia Messina; Giuseppe Vita; Chiara Passarelli; Simona Brioschi; Matteo Bovolenta; Marcella Neri; Francesca Gualandi; Steve D Wilton; Louise R Rodino-Klapac; Lin Yang; Diane M Dunn; Daniel R Schoenberg; Robert B Weiss; Michael T Howard; Alessandra Ferlini; Kevin M Flanigan
Journal:  Nat Med       Date:  2014-08-10       Impact factor: 53.440
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