Literature DB >> 32605399

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

James M Wilson1, Terence R Flotte2,3.   

Abstract

Entities:  

Year:  2020        PMID： 32605399     DOI： 10.1089/hum.2020.182

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


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  45 in total

1.  Laying the Foundation for Neuromuscular Disease Gene Therapy.

Authors:  Dongsheng Duan
Journal:  Hum Gene Ther       Date:  2020-07-28       Impact factor: 5.695

Review 2.  Gene therapy and gene correction: targets, progress, and challenges for treating human diseases.

Authors:  Matthew R Cring; Val C Sheffield
Journal:  Gene Ther       Date:  2020-10-09       Impact factor: 5.250

3.  Advanced therapeutic strategy for hereditary neuromuscular diseases.

Authors:  Wuh-Liang Hwu; Shin-Ichi Muramatsu; Yin-Hsiu Chien; Barry J Byrne
Journal:  Mol Ther       Date:  2021-12-11       Impact factor: 11.454

Review 4.  Myogenic Cell Transplantation in Genetic and Acquired Diseases of Skeletal Muscle.

Authors:  Olivier Boyer; Gillian Butler-Browne; Hector Chinoy; Giulio Cossu; Francesco Galli; James B Lilleker; Alessandro Magli; Vincent Mouly; Rita C R Perlingeiro; Stefano C Previtali; Maurilio Sampaolesi; Hubert Smeets; Verena Schoewel-Wolf; Simone Spuler; Yvan Torrente; Florence Van Tienen
Journal:  Front Genet       Date:  2021-08-02       Impact factor: 4.599

5.  Adenine Base Editing In Vivo with a Single Adeno-Associated Virus Vector.

Authors:  Han Zhang; Nathan Bamidele; Pengpeng Liu; Ogooluwa Ojelabi; Xin D Gao; Tomás Rodriguez; Haoyang Cheng; Karen Kelly; Jonathan K Watts; Jun Xie; Guangping Gao; Scot A Wolfe; Wen Xue; Erik J Sontheimer
Journal:  GEN Biotechnol       Date:  2022-06-14

Review 6.  CRISPR Modeling and Correction of Cardiovascular Disease.

Authors:  Ning Liu; Eric N Olson
Journal:  Circ Res       Date:  2022-06-09       Impact factor: 23.213

7.  Updates on Cardiac Gene Therapy Research and Methods: Overview of Cardiac Gene Therapy.

Authors:  Francisco J Romeo; Spyros A Marvopoulos; Kiyotake Ishikawa
Journal:  Methods Mol Biol       Date:  2022

Review 8.  Best of most possible worlds: Hybrid gene therapy vectors based on parvoviruses and heterologous viruses.

Authors:  Julia Fakhiri; Dirk Grimm
Journal:  Mol Ther       Date:  2021-04-05       Impact factor: 11.454

Review 9.  Gene-based therapies for neurodegenerative diseases.

Authors:  Jichao Sun; Subhojit Roy
Journal:  Nat Neurosci       Date:  2021-02-01       Impact factor: 24.884

Review 10.  Overcoming innate immune barriers that impede AAV gene therapy vectors.

Authors:  Manish Muhuri; Yukiko Maeda; Hong Ma; Sanjay Ram; Katherine A Fitzgerald; Phillip Wl Tai; Guangping Gao
Journal:  J Clin Invest       Date:  2021-01-04       Impact factor: 14.808
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