Literature DB >> 32640898

Laying the Foundation for Neuromuscular Disease Gene Therapy.

Dongsheng Duan1,2,3,4.   

Abstract

Entities:  

Year:  2020        PMID： 32640898      PMCID： PMC7462027          DOI： 10.1089/hum.2020.198

Source DB:  PubMed          Journal:  Hum Gene Ther        ISSN： 1043-0342            Impact factor:   5.695


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  8 in total

Review 1.  Adeno-associated virus vector as a platform for gene therapy delivery.

Authors:  Dan Wang; Phillip W L Tai; Guangping Gao
Journal:  Nat Rev Drug Discov       Date:  2019-05       Impact factor: 84.694

2.  Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy.

Authors:  James M Wilson; Terence R Flotte
Journal:  Hum Gene Ther       Date:  2020-07-01       Impact factor: 5.695

3.  Intralingual Administration of AAVrh10-miRSOD1 Improves Respiratory But Not Swallowing Function in a Superoxide Dismutase-1 Mouse Model of Amyotrophic Lateral Sclerosis.

Authors:  Lori A Lind; Ellyn M Andel; Angela L McCall; Justin S Dhindsa; Katherine A Johnson; Olivia E Stricklin; Christian Mueller; Mai K ElMallah; Teresa E Lever; Nicole L Nichols
Journal:  Hum Gene Ther       Date:  2020-07-13       Impact factor: 5.695

4.  Stress-Induced Mouse Model of the Cardiac Manifestations of Friedreich's Ataxia Corrected by AAV-mediated Gene Therapy.

Authors:  Christiana O Salami; Katie Jackson; Clarisse Jose; Laith Alyass; Georges-Ibrahim Cisse; Bishnu P De; Katie M Stiles; Maria J Chiuchiolo; Dolan Sondhi; Ronald G Crystal; Stephen M Kaminsky
Journal:  Hum Gene Ther       Date:  2020-08       Impact factor: 5.695

5.  Revisiting the "New" Inflammatory Toxicities of Adeno-Associated Virus Vectors.

Authors:  Terence R Flotte
Journal:  Hum Gene Ther       Date:  2020-04       Impact factor: 5.695

6.  Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

Authors:  Allison M Keeler; Terence R Flotte
Journal:  Annu Rev Virol       Date:  2019-07-05       Impact factor: 10.431

7.  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

8.  Defining Transcription Regulatory Elements in the Human Frataxin Gene: Implications for Gene Therapy.

Authors:  Jixue Li; Yanjie Li; Jun Wang; Trevor J Gonzalez; Aravind Asokan; Jill S Napierala; Marek Napierala
Journal:  Hum Gene Ther       Date:  2020-07-13       Impact factor: 4.793
  8 in total

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