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)
Get full text
Journal Article
Automatic characterization of stride parameters in canines with a single wearable inertial sensor
Jenkins, Gregory J, Hakim, Chady H, Yang, N Nora, Yao, Gang, Duan, Dongsheng
Published in PloS one (14.06.2018)
Published in PloS one (14.06.2018)
Get full text
Journal Article
Extensor carpi ulnaris muscle shows unexpected slow-to-fast fiber-type switch in Duchenne muscular dystrophy dogs
Hakim, Chady H, Yang, Hsiao T, Burke, Matthew J, Teixeira, James, Jenkins, Gregory J, Yang, N Nora, Yao, Gang, Duan, Dongsheng
Published in Disease models & mechanisms (01.12.2021)
Published in Disease models & mechanisms (01.12.2021)
Get full text
Journal Article
Dystrophin contains multiple independent membrane-binding domains
Zhao, Junling, Kodippili, Kasun, Yue, Yongping, Hakim, Chady H, Wasala, Lakmini, Pan, Xiufang, Zhang, Keqing, Yang, Nora N, Duan, Dongsheng, Lai, Yi
Published in Human molecular genetics (01.09.2016)
Published in Human molecular genetics (01.09.2016)
Get full text
Journal Article
Nanotherapy for Duchenne muscular dystrophy
Nance, Michael E, Hakim, Chady H, Yang, N Nora, Duan, Dongsheng
Published in Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology (01.03.2018)
Published in Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology (01.03.2018)
Get more information
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)
Get full text
Journal Article
Micro-dystrophin AAV Vectors Made by Transient Transfection and Herpesvirus System Are Equally Potent in Treating mdx Mouse Muscle Disease
Hakim, Chady H., Clément, Nathalie, Wasala, Lakmini P., Yang, Hsiao T., Yue, Yongping, Zhang, Keqing, Kodippili, Kasun, Adamson-Small, Laura, Pan, Xiufang, Schneider, Joel S., Yang, N. Nora, Chamberlain, Jeffrey S., Byrne, Barry J., Duan, Dongsheng
Published in Molecular therapy. Methods & clinical development (11.09.2020)
Published in Molecular therapy. Methods & clinical development (11.09.2020)
Get full text
Journal Article
Widespread severe myodegeneration in a compound heterozygote female dog with dystrophin deficiency
Fortin, Jessica S., Hakim, Chady H., Korte, Scott, Yang, N. Nora, Fitzgerald, Scott D., Johnson, Gayle C., Smith, Bruce F., Duan, Dongsheng
Published in Veterinary medicine and science (01.05.2021)
Published in Veterinary medicine and science (01.05.2021)
Get full text
Journal Article
Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine Model
Kodippili, Kasun, Hakim, Chady H, Pan, Xiufang, Yang, Hsiao T, Yue, Yongping, Zhang, Yadong, Shin, Jin-Hong, Yang, N Nora, Duan, Dongsheng
Published in Human gene therapy (01.03.2018)
Published in Human gene therapy (01.03.2018)
Get more information
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)
Get full text
Journal Article
An improved method for studying mouse diaphragm function
Hakim, Chady H., Lessa, Thais B., Jenkins, Gregory J., Yang, Nora N., Ambrosio, Carlos E., Duan, Dongsheng
Published in Scientific reports (19.12.2019)
Published in Scientific reports (19.12.2019)
Get full text
Journal Article
Nitric oxide‐dependent attenuation of noradrenaline‐induced vasoconstriction is impaired in the canine model of Duchenne muscular dystrophy
Kodippili, Kasun, Hakim, Chady H., Yang, Hsiao T., Pan, Xiufang, Yang, N. Nora, Laughlin, Maurice H., Terjung, Ronald L., Duan, Dongsheng
Published in The Journal of physiology (01.11.2018)
Published in The Journal of physiology (01.11.2018)
Get full text
Journal Article
High prevalence of plasma lipid abnormalities in human and canine Duchenne and Becker muscular dystrophies depicts a new type of primary genetic dyslipidemia
White, Zoe, Hakim, Chady H., Theret, Marine, Yang, N. Nora, Rossi, Fabio, Cox, Dan, Francis, Gordon A., Straub, Volker, Selby, Kathryn, Panagiotopoulos, Constadina, Duan, Dongsheng, Bernatchez, Pascal
Published in Journal of clinical lipidology (01.07.2020)
Published in Journal of clinical lipidology (01.07.2020)
Get full text
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)
Get full text
Journal Article
Financing translation: analysis of the NCATS rare-diseases portfolio
Fagnan, David E, Yang, N Nora, McKew, John C, Lo, Andrew W
Published in Science translational medicine (25.02.2015)
Published in Science translational medicine (25.02.2015)
Get more information
Journal Article
Repurposing a novel parathyroid hormone analogue to treat hypoparathyroidism
Krishnan, Venkatesh, Ma, Yanfei L, Chen, Catherine Z, Thorne, Natasha, Bullock, Heather, Tawa, Gregory, Javella‐Cauley, Christy, Chu, Shaoyou, Li, Weiming, Kohn, Wayne, Adrian, Mary D, Benson, Charles, Liu, Lifei, Sato, Masahiko, Zheng, Wei, Pilon, Andre M, Yang, N. Nora, Bryant, Henry U
Published in British journal of pharmacology (01.01.2018)
Published in British journal of pharmacology (01.01.2018)
Get full text
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)
Get full text
Journal Article