Literature DB >> 31558355

Neuromuscular Gene Therapy: Catching the Wave of Positive Early Studies Is Expected to Crest in Product Approvals.

Barry J Byrne1.   

Abstract

Mesh：

Year:  2019        PMID： 31558355      PMCID： PMC6822225          DOI： 10.1016/j.ymthe.2019.09.011

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


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  5 in total

1.  Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning.

Authors:  Manuela Corti; Brian Cleaver; Nathalie Clément; Thomas J Conlon; Kaitlyn J Faris; Gensheng Wang; Janet Benson; Alice F Tarantal; Davis Fuller; Roland W Herzog; Barry J Byrne
Journal:  Hum Gene Ther Clin Dev       Date:  2015-09       Impact factor: 5.032

2.  Myocarditis following adeno-associated viral gene expression of human soluble TNF receptor (TNFRII-Fc) in baboon hearts.

Authors:  C F McTiernan; M A Mathier; X Zhu; X Xiao; E Klein; C H Swan; H Mehdi; G Gibson; A M Trichel; J C Glorioso; A M Feldman; K R McCurry; B London
Journal:  Gene Ther       Date:  2007-09-13       Impact factor: 5.250

Review 3.  Newborn screening for Pompe disease: synthesis of the evidence and development of screening recommendations.

Authors:  Alex R Kemper; Wuh-Liang Hwu; Michele Lloyd-Puryear; Priya S Kishnani
Journal:  Pediatrics       Date:  2007-11       Impact factor: 7.124

Review 4.  Pompe disease: early diagnosis and early treatment make a difference.

Authors:  Yin-Hsiu Chien; Wuh-Liang Hwu; Ni-Chung Lee
Journal:  Pediatr Neonatol       Date:  2013-04-28       Impact factor: 2.083

5.  Maximizing the Benefit of Life-Saving Treatments for Pompe Disease, Spinal Muscular Atrophy, and Duchenne Muscular Dystrophy Through Newborn Screening: Essential Steps.

Authors:  Mei Baker; Robert Griggs; Barry Byrne; Anne M Connolly; Richard Finkel; Lucja Grajkowska; Amanda Haidet-Phillips; Laura Hagerty; Robert Ostrander; Lianna Orlando; Kathryn Swoboda; Michael Watson; R Rodney Howell
Journal:  JAMA Neurol       Date:  2019-08-01       Impact factor: 18.302
  5 in total
  1 in total

Review 1.  Mesenchymal Stem Cells as a Gene Delivery Tool: Promise, Problems, and Prospects.

Authors:  Noha Attia; Mohamed Mashal; Gustavo Puras; Jose Luis Pedraz
Journal:  Pharmaceutics       Date:  2021-06-07       Impact factor: 6.321
  1 in total

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