Literature DB >> 33476580

Challenges at the Crossroads: Myopathy Trials in 2020 Hindsight.

Christopher Greer1, Benjamin Kozyak2, Hansell Stedman3.   

Abstract

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Mesh：

Year:  2021        PMID： 33476580      PMCID： PMC7854349          DOI： 10.1016/j.ymthe.2021.01.012

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


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  6 in total

1.  Specific sequences of infectious challenge lead to secondary hemophagocytic lymphohistiocytosis-like disease in mice.

Authors:  Andrew Wang; Scott D Pope; Jason S Weinstein; Shuang Yu; Cuiling Zhang; Carmen J Booth; Ruslan Medzhitov
Journal:  Proc Natl Acad Sci U S A       Date:  2019-01-23       Impact factor: 11.205

2.  Subacute Liver Failure Following Gene Replacement Therapy for Spinal Muscular Atrophy Type 1.

Authors:  Amy G Feldman; Julie A Parsons; Cullen M Dutmer; Aravindhan Veerapandiyan; Einar Hafberg; Nolan Maloney; Cara L Mack
Journal:  J Pediatr       Date:  2020-05-28       Impact factor: 4.406

3.  Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models.

Authors:  Yafeng Song; Leon Morales; Alock S Malik; Andrew F Mead; Christopher D Greer; Marilyn A Mitchell; Mihail T Petrov; Leonard T Su; Margaret E Choi; Shira T Rosenblum; Xiangping Lu; Daniel J VanBelzen; Ranjith K Krishnankutty; Frederick J Balzer; Emanuele Loro; Robert French; Kathleen J Propert; Shangzhen Zhou; Benjamin W Kozyak; Peter P Nghiem; Tejvir S Khurana; Joe N Kornegay; Hansell H Stedman
Journal:  Nat Med       Date:  2019-10-07       Impact factor: 53.440

4.  Thinking Beyond HLH: Clinical Features of Patients with Concurrent Presentation of Hemophagocytic Lymphohistiocytosis and Thrombotic Microangiopathy.

Authors:  Nicholas J Gloude; Christopher E Dandoy; Stella M Davies; Kasiani C Myers; Michael B Jordan; Rebecca A Marsh; Ashish Kumar; Jack Bleesing; Ashley Teusink-Cross; Sonata Jodele
Journal:  J Clin Immunol       Date:  2020-05-23       Impact factor: 8.317

5.  Re: "Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy" by Wilson and Flotte.

Authors:  Perry B Shieh; Carsten G Bönnemann; Wolfgang Müller-Felber; Astrid Blaschek; James J Dowling; Nancy L Kuntz; Andreea M Seferian
Journal:  Hum Gene Ther       Date:  2020-08       Impact factor: 5.695

6.  Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial.

Authors:  Jerry R Mendell; Zarife Sahenk; Kelly Lehman; Carrie Nease; Linda P Lowes; Natalie F Miller; Megan A Iammarino; Lindsay N Alfano; Amanda Nicholl; Samiah Al-Zaidy; Sarah Lewis; Kathleen Church; Richard Shell; Linda H Cripe; Rachael A Potter; Danielle A Griffin; Eric Pozsgai; Ashish Dugar; Mark Hogan; Louise R Rodino-Klapac
Journal:  JAMA Neurol       Date:  2020-09-01       Impact factor: 18.302
  6 in total
  1 in total

Review 1.  Adeno-Associated Virus (AAV) Gene Delivery: Dissecting Molecular Interactions upon Cell Entry.

Authors:  Edward E Large; Mark A Silveria; Grant M Zane; Onellah Weerakoon; Michael S Chapman
Journal:  Viruses       Date:  2021-07-10       Impact factor: 5.048
  1 in total

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