Zobrazeno 1 - 10
of 14
pro vyhledávání: '"Lakmini P. Wasala"'
Autor:
Chady H. Hakim, Nathalie Clément, Lakmini P. Wasala, Hsiao T. Yang, Yongping Yue, Keqing Zhang, Kasun Kodippili, Laura Adamson-Small, Xiufang Pan, Joel S. Schneider, N. Nora Yang, Jeffrey S. Chamberlain, Barry J. Byrne, Dongsheng Duan
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss , Pp 664-678 (2020)
Vector production scale-up is a major barrier in systemic adeno-associated virus (AAV) gene therapy. Many scalable manufacturing methods have been developed. However, the potency of the vectors generated by these methods has rarely been compared with
Externí odkaz:
https://doaj.org/article/c9a8a753dbc949b9af502c5c8edc3335
Autor:
Nalinda B. Wasala, Emily D. Million, Thais B. Watkins, Lakmini P. Wasala, Jin Han, Yongping Yue, Baisong Lu, Shi-jie Chen, Chady H. Hakim, Dongsheng Duan
Publikováno v:
Hum Gene Ther
Adeno-associated virus (AAV)-mediated clustered regularly interspaced short palindromic repeats (CRISPR) editing holds promise to restore missing dystrophin in Duchenne muscular dystrophy (DMD). Intramuscular coinjection of CRISPR-associated protein
Autor:
Lakmini P. Wasala, Thais B. Watkins, Nalinda B. Wasala, Matthew J. Burke, Yongping Yue, Yi Lai, Gang Yao, Dongsheng Duan
Publikováno v:
Human gene therapy.
Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by dystrophin deficiency. Dystrophin consists of the amino terminus, central rod domain with 24 spectrin-like repeats and four hinges (H), cysteine-rich domain, and carboxyl terminus.
Autor:
Dongsheng Duan, Yongping Yue, Lakmini P. Wasala, Xiufang Pan, Jeffrey S. Chamberlain, N. Nora Yang, Chady H. Hakim, Nathalie Clement, Joel S. Schneider, Keqing Zhang, Barry J. Byrne, Kasun Kodippili, Laura Adamson-Small, Hsiao T. Yang
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss, Pp 664-678 (2020)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
Vector production scale-up is a major barrier in systemic adeno-associated virus (AAV) gene therapy. Many scalable manufacturing methods have been developed. However, the potency of the vectors generated by these methods has rarely been compared with
Autor:
Keqing Zhang, Jacob F. Karp, Junling Zhao, Yongping Yue, Yi Lai, Aman Patel, Lakmini P. Wasala, Dongsheng Duan
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss, Pp 856-868 (2020)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
Adeno-associated virus (AAV) is one of the most important gene delivery vehicles for in vivo gene therapy. Intramuscular (i.m.) and intravascular (i.v.) injection are commonly used for AAV gene transfer. Unfortunately, the fate of AAV vectors followi
Autor:
Melissa G. Mitchum, Richard S. Hussey, Eric L. Davis, Lakmini P. Wasala, Jianying Wang, Gregory Yeckel, Thomas J. Baum, Pramod Kaitheri Kandoth
Publikováno v:
Molecular Plant Pathology
While numerous effectors that suppress plant immunity have been identified from bacteria, fungi, and oomycete pathogens, relatively little is known for nematode effectors. Several dozen effectors have been reported from the soybean cyst nematode (SCN
Autor:
David J. Pintel, Xiufang Pan, Ngoc Tam Tran, Phillip W. L. Tai, Yongping Yue, Dongsheng Duan, Maria Boftsi, Keqing Zhang, Lakmini P. Wasala
Publikováno v:
Gene therapy. 29(6)
Inverted terminal repeats (ITRs) are the only wild-type components retained in the genome of adeno-associated virus (AAV) vectors. To determine whether ITR modification is a viable approach for AAV vector engineering, we rationally deleted all CpG mo
Autor:
Nalinda B. Wasala, Yongping Yue, Nandita Niranjan, Roger J. Hajjar, Dongsheng Duan, Gopal J. Babu, Lakmini P. Wasala, William Lostal
Publikováno v:
Mol Ther
Loss of dystrophin leads to Duchenne muscular dystrophy (DMD). A pathogenic feature of DMD is the significant elevation of cytosolic calcium. Supraphysiological calcium triggers protein degradation, membrane damage, and eventually muscle death and dy
Publikováno v:
Methods in Molecular Biology ISBN: 9781493990641
Many diseases affect multiple tissues and/or organ systems, or affect tissues that are broadly distributed. For these diseases, an effective gene therapy will require systemic delivery of the therapeutic vector to all affected locations. Adeno-associ
Autor:
Yongping Yue, Lakmini P. Wasala, Nora Yang, Junling Zhao, Kasun Kodippili, Yi Lai, Dongsheng Duan, Xiufang Pan, Keqing Zhang, Chady H. Hakim
Publikováno v:
Human Molecular Genetics. 25:3647-3653
Dystrophin is a large sub-sarcolemmal protein. Its absence leads to Duchenne muscular dystrophy (DMD). Binding to the sarcolemma is essential for dystrophin to protect muscle from contraction-induced injury. It has long been thought that membrane bin