Concordant but Varied Phenotypes among Duchenne Muscular Dystrophy Patient-Specific Myoblasts Derived using a Human iPSC-Based Model
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- 作者：In Young Choi1 ; 14 ; HoTae Lim1 ; 14 ; Kenneth Estrellas2 ; 7 ; Jyothi Mula3 ; 7 ; Tatiana V. Cohen3 ; 7 ; Yuanfan Zhang2 ; 7 ; Christopher J. Donnelly3 ; 4 ; 5 ; 15 ; Jean-Philippe Richard3 ; Yong Jun Kim1 ; 3 ; 8 ; Hyesoo Kim1 ; 6 ; Yasuhiro Kazuki9 ; 10 ; Mitsuo Oshimura9 ; Hongmei Lisa Li11 ; Akitsu Hotta11 ; 12 ; 13 ; Jeffrey Rothstein2 ; 3 ; 4 ; 5 ; Nicholas Maragakis3 ; Kathryn R. Wagner2 ; 3 ; 5 ; 7 ; wagnerk@kennedykrieger.org" class="auth_mail" title="E-mail the corresponding author ; Gabsang Lee1 ; 3 ; 5 ; glee48@jhmi.edu" class="auth_mail" title="E-mail the corresponding author
- 刊名：Cell Reports
- 出版年：2016
- 出版时间：7 June 2016
- 年：2016
- 卷：15
- 期：10
- 页码：2301-2312
- 全文大小：4176 K
文摘
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Isolation of functional myoblasts from multiple hiPSC lines using a defined system
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Concordant but heterogeneous phenotypes among myoblasts from DMD patients
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Genetic and pharmacological rescue of DMD-related phenotypes
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Myotube formation in DMD-myoblasts and genetically corrected isogenic myoblasts