Literature DB >> 26718928

Standard Operating Procedures (SOPs) for Evaluating the Heart in Preclinical Studies of Duchenne Muscular Dystrophy.

Dongsheng Duan1,2, Jill A Rafael-Fortney3, Alison Blain4, David A Kass5, Elizabeth M McNally6, Joseph M Metzger7, Christopher F Spurney8, Kathi Kinnett9.   

Abstract

A recent working group meeting focused on contemporary cardiac issues in Duchenne muscular dystrophy (DMD) was hosted by the National Heart, Lung, and Blood Institute in collaboration with the Parent Project Muscular Dystrophy. An outcome of this meeting was to provide freely available detailed protocols for preclinical animal studies. The goal of these protocols is to improve the quality and reproducibility of cardiac preclinical studies aimed at developing new therapeutics for the prevention and treatment of DMD cardiomyopathy.

Entities:  

Keywords:  Cardiology; Cardiomyopathy; Duchenne; Preclinical; Standard operating procedures (SOPs)

Mesh：

Year:  2015        PMID： 26718928      PMCID： PMC5509361          DOI： 10.1007/s12265-015-9669-6

Source DB:  PubMed          Journal:  J Cardiovasc Transl Res        ISSN： 1937-5387            Impact factor:   4.132


  5 in total

1.  Failure to replicate the STAP cell phenomenon.

Authors:  Alejandro De Los Angeles; Francesco Ferrari; Yuko Fujiwara; Ronald Mathieu; Soohyun Lee; Semin Lee; Ho-Chou Tu; Samantha Ross; Stephanie Chou; Minh Nguyen; Zhaoting Wu; Thorold W Theunissen; Benjamin E Powell; Sumeth Imsoonthornruksa; Jiekai Chen; Marti Borkent; Vladislav Krupalnik; Ernesto Lujan; Marius Wernig; Jacob H Hanna; Konrad Hochedlinger; Duanqing Pei; Rudolf Jaenisch; Hongkui Deng; Stuart H Orkin; Peter J Park; George Q Daley
Journal:  Nature       Date:  2015-09-24       Impact factor: 49.962

2.  Contemporary cardiac issues in Duchenne muscular dystrophy. Working Group of the National Heart, Lung, and Blood Institute in collaboration with Parent Project Muscular Dystrophy.

Authors:  Elizabeth M McNally; Jonathan R Kaltman; D Woodrow Benson; Charles E Canter; Linda H Cripe; Dongsheng Duan; Jonathan D Finder; William J Groh; Eric P Hoffman; Daniel P Judge; Naomi Kertesz; Kathi Kinnett; Roxanne Kirsch; Joseph M Metzger; Gail D Pearson; Jill A Rafael-Fortney; Subha V Raman; Christopher F Spurney; Shari L Targum; Kathryn R Wagner; Larry W Markham
Journal:  Circulation       Date:  2015-05-05       Impact factor: 29.690

Review 3.  Reproducibility in science: improving the standard for basic and preclinical research.

Authors:  C Glenn Begley; John P A Ioannidis
Journal:  Circ Res       Date:  2015-01-02       Impact factor: 17.367

4.  Policy: NIH plans to enhance reproducibility.

Authors:  Francis S Collins; Lawrence A Tabak
Journal:  Nature       Date:  2014-01-30       Impact factor: 49.962

Review 5.  Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy.

Authors:  Joe W McGreevy; Chady H Hakim; Mark A McIntosh; Dongsheng Duan
Journal:  Dis Model Mech       Date:  2015-03       Impact factor: 5.758
  5 in total
  9 in total

1.  AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice.

Authors:  Chady H Hakim; Nalinda B Wasala; Christopher E Nelson; Lakmini P Wasala; Yongping Yue; Jacqueline A Louderman; Thais B Lessa; Aihua Dai; Keqing Zhang; Gregory J Jenkins; Michael E Nance; Xiufang Pan; Kasun Kodippili; N Nora Yang; Shi-Jie Chen; Charles A Gersbach; Dongsheng Duan
Journal:  JCI Insight       Date:  2018-12-06

2.  Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy.

Authors:  Nalinda B Wasala; Yongping Yue; William Lostal; Lakmini P Wasala; Nandita Niranjan; Roger J Hajjar; Gopal J Babu; Dongsheng Duan
Journal:  Mol Ther       Date:  2020-01-10       Impact factor: 11.454

Review 3.  Duchenne muscular dystrophy animal models for high-throughput drug discovery and precision medicine.

Authors:  Nalinda B Wasala; Shi-Jie Chen; Dongsheng Duan
Journal:  Expert Opin Drug Discov       Date:  2020-01-30       Impact factor: 6.098

4.  Cardiac-Specific Expression of ΔH2-R15 Mini-Dystrophin Normalized All Electrocardiogram Abnormalities and the End-Diastolic Volume in a 23-Month-Old Mouse Model of Duchenne Dilated Cardiomyopathy.

Authors:  Nalinda B Wasala; Jin-Hong Shin; Yi Lai; Yongping Yue; Federica Montanaro; Dongsheng Duan
Journal:  Hum Gene Ther       Date:  2018-03-22       Impact factor: 5.695

5.  Uniform low-level dystrophin expression in the heart partially preserved cardiac function in an aged mouse model of Duchenne cardiomyopathy.

Authors:  Nalinda B Wasala; Yongping Yue; Jenna Vance; Dongsheng Duan
Journal:  J Mol Cell Cardiol       Date:  2016-11-29       Impact factor: 5.000

6.  Questions Answered and Unanswered by the First CRISPR Editing Study in a Canine Model of Duchenne Muscular Dystrophy.

Authors:  Nalinda B Wasala; Chady H Hakim; Shi-Jie Chen; N Nora Yang; Dongsheng Duan
Journal:  Hum Gene Ther       Date:  2019-02-26       Impact factor: 5.695

7.  A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy.

Authors:  Chady H Hakim; Nalinda B Wasala; Xiufang Pan; Kasun Kodippili; Yongping Yue; Keqing Zhang; Gang Yao; Brittney Haffner; Sean X Duan; Julian Ramos; Joel S Schneider; N Nora Yang; Jeffrey S Chamberlain; Dongsheng Duan
Journal:  Mol Ther Methods Clin Dev       Date:  2017-07-27       Impact factor: 6.698

8.  Ryanodine channel complex stabilizer compound S48168/ARM210 as a disease modifier in dystrophin-deficient mdx mice: proof-of-concept study and independent validation of efficacy.

Authors:  Roberta Francesca Capogrosso; Paola Mantuano; Kitipong Uaesoontrachoon; Anna Cozzoli; Arcangela Giustino; Todd Dow; Sadish Srinivassane; Marina Filipovic; Christina Bell; Jack Vandermeulen; Ada Maria Massari; Michela De Bellis; Elena Conte; Sabata Pierno; Giulia Maria Camerino; Antonella Liantonio; Kanneboyina Nagaraju; Annamaria De Luca
Journal:  FASEB J       Date:  2018-01-03       Impact factor: 5.834

9.  "Of Mice and Measures": A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic.

Authors:  Heather Gordish-Dressman; Raffaella Willmann; Laura Dalle Pazze; Arati Kreibich; Maaike van Putten; Ahlke Heydemann; Laurent Bogdanik; Cathleen Lutz; Kay Davies; Alexis R Demonbreun; Dongsheng Duan; David Elsey; So-Ichiro Fukada; Mahasweta Girgenrath; J Patrick Gonzalez; Miranda D Grounds; Andy Nichols; Terry Partridge; Marco Passini; Francesca Sanarica; Frederick J Schnell; Dominic J Wells; Toshifumi Yokota; Courtney S Young; Zhong Zhong; Christopher Spurney; Melissa Spencer; Annamaria De Luca; Kanneboyina Nagaraju; Annemieke Aartsma-Rus
Journal:  J Neuromuscul Dis       Date:  2018
  9 in total

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