Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models
Hakim, Chady H, Kumar, Sandeep R P, Pérez-López, Dennis O, Wasala, Nalinda B, Zhang, Dong, Yue, Yongping, Teixeira, James, Pan, Xiufang, Zhang, Keqing, Million, Emily D, Nelson, Christopher E, Metzger, Samantha, Han, Jin, Louderman, Jacqueline A, Schmidt, Florian, Feng, Feng, Grimm, Dirk, Smith, Bruce F, Yao, Gang, Yang, N Nora, Gersbach, Charles A, Chen, Shi-Jie, Herzog, Roland W, Duan, Dongsheng
Published in Nature communications (24.11.2021)
Published in Nature communications (24.11.2021)
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Journal Article
A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy
Hakim, Chady H, Wasala, Nalinda B, Pan, Xiufang, Kodippili, Kasun, Yue, Yongping, Zhang, Keqing, Yao, Gang, Haffner, Brittney, Duan, Sean X, Ramos, Julian, Schneider, Joel S, Yang, N Nora, Chamberlain, Jeffrey S, Duan, Dongsheng
Published in Molecular therapy. Methods & clinical development (15.09.2017)
Published in Molecular therapy. Methods & clinical development (15.09.2017)
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Journal Article
The evolution of heart gene delivery vectors
Wasala, Nalinda B., Shin, Jin-Hong, Duan, Dongsheng
Published in The journal of gene medicine (01.10.2011)
Published in The journal of gene medicine (01.10.2011)
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Journal Article
100-fold but not 50-fold dystrophin overexpression aggravates electrocardiographic defects in the mdx model of Duchenne muscular dystrophy
Yue, Yongping, Wasala, Nalinda B, Bostick, Brian, Duan, Dongsheng
Published in Molecular therapy. Methods & clinical development (01.01.2016)
Published in Molecular therapy. Methods & clinical development (01.01.2016)
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Journal Article
Lifelong Outcomes of Systemic Adeno-Associated Virus Micro-Dystrophin Gene Therapy in a Murine Duchenne Muscular Dystrophy Model
Wasala, Nalinda B, Yue, Yongping, Hu, Bryan, Shin, Jin-Hong, Srivastava, Arun, Yao, Gang, Duan, Dongsheng
Published in Human gene therapy (01.05.2023)
Published in Human gene therapy (01.05.2023)
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Journal Article
Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy
Wasala, Nalinda B., Yue, Yongping, Lostal, William, Wasala, Lakmini P., Niranjan, Nandita, Hajjar, Roger J., Babu, Gopal J., Duan, Dongsheng
Published in Molecular therapy (04.03.2020)
Published in Molecular therapy (04.03.2020)
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Journal Article
The Implication of Hinge 1 and Hinge 4 in Micro-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy
Wasala, Lakmini P, Watkins, Thais B, Wasala, Nalinda B, Burke, Matthew J, Yue, Yongping, Lai, Yi, Yao, Gang, Duan, Dongsheng
Published in Human gene therapy (01.05.2023)
Published in Human gene therapy (01.05.2023)
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Journal Article
Uniform low-level dystrophin expression in the heart partially preserved cardiac function in an aged mouse model of Duchenne cardiomyopathy
Wasala, Nalinda B, Yue, Yongping, Vance, Jenna, Duan, Dongsheng
Published in Journal of molecular and cellular cardiology (01.01.2017)
Published in Journal of molecular and cellular cardiology (01.01.2017)
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Journal Article
AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice
Nance, Michael E., Shi, Ruicheng, Hakim, Chady H., Wasala, Nalinda B., Yue, Yongping, Pan, Xiufang, Zhang, Tracy, Robinson, Carolyn A., Duan, Sean X., Yao, Gang, Yang, N. Nora, Chen, Shi-jie, Wagner, Kathryn R., Gersbach, Charles A., Duan, Dongsheng
Published in Molecular therapy (04.09.2019)
Published in Molecular therapy (04.09.2019)
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Journal Article
Partial restoration of cardiac function with ΔPDZ nNOS in aged mdx model of Duchenne cardiomyopathy
Lai, Yi, Zhao, Junling, Yue, Yongping, Wasala, Nalinda B, Duan, Dongsheng
Published in Human molecular genetics (15.06.2014)
Published in Human molecular genetics (15.06.2014)
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Journal Article
AAV micro-dystrophin gene therapy alleviates stress-induced cardiac death but not myocardial fibrosis in > 21-m-old mdx mice, an end-stage model of Duchenne muscular dystrophy cardiomyopathy
Bostick, Brian, Shin, Jin-Hong, Yue, Yongping, Wasala, Nalinda B, Lai, Yi, Duan, Dongsheng
Published in Journal of molecular and cellular cardiology (01.08.2012)
Published in Journal of molecular and cellular cardiology (01.08.2012)
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Journal Article
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
Wasala, Nalinda B, Shin, Jin-Hong, Lai, Yi, Yue, Yongping, Montanaro, Federica, Duan, Dongsheng
Published in Human gene therapy (01.07.2018)
Published in Human gene therapy (01.07.2018)
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Journal Article
Proteomic analysis identifies key differences in the cardiac interactomes of dystrophin and micro-dystrophin
Wang, Hong, Marrosu, Elena, Brayson, Daniel, Wasala, Nalinda B, Johnson, Eric K, Scott, Charlotte S, Yue, Yongping, Hau, Kwan-Leong, Trask, Aaron J, Froehner, Stan C, Adams, Marvin E, Zhang, Liwen, Duan, Dongsheng, Montanaro, Federica
Published in Human molecular genetics (26.06.2021)
Published in Human molecular genetics (26.06.2021)
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Journal Article
The gRNA Vector Level Determines the Outcome of Systemic AAV CRISPR Therapy for Duchenne Muscular Dystrophy
Wasala, Nalinda B, Million, Emily D, Watkins, Thais B, Wasala, Lakmini P, Han, Jin, Yue, Yongping, Lu, Baisong, Chen, Shi-Jie, Hakim, Chady H, Duan, Dongsheng
Published in Human gene therapy (01.05.2022)
Published in Human gene therapy (01.05.2022)
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Journal Article
AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice
Hakim, Chady H, Wasala, Nalinda B, Nelson, Christopher E, Wasala, Lakmini P, Yue, Yongping, Louderman, Jacqueline A, Lessa, Thais B, Dai, Aihua, Zhang, Keqing, Jenkins, Gregory J, Nance, Michael E, Pan, Xiufang, Kodippili, Kasun, Yang, N Nora, Chen, Shi-Jie, Gersbach, Charles A, Duan, Dongsheng
Published in JCI insight (06.12.2018)
Published in JCI insight (06.12.2018)
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