Zobrazeno 1 - 10
of 330
pro vyhledávání: '"Jeffrey S. Chamberlain"'
Autor:
Marcelo dos Santos Voltani Lorena, Estela Kato dos Santos, Renato Ferretti, G. A. Nagana Gowda, Guy L. Odom, Jeffrey S. Chamberlain, Cintia Yuri Matsumura
Publikováno v:
Skeletal Muscle, Vol 13, Iss 1, Pp 1-13 (2023)
Abstract Background Duchenne muscular dystrophy (DMD) is a severe form of muscular dystrophy without an effective treatment, caused by mutations in the DMD gene, leading to the absence of dystrophin. DMD results in muscle weakness, loss of ambulation
Externí odkaz:
https://doaj.org/article/bee194a80f0f4dbb94252899775bccf4
Autor:
Michael R. Emami, Alejandro Espinoza, Courtney S. Young, Feiyang Ma, Philip K. Farahat, Philip L. Felgner, Jeffrey S. Chamberlain, Xiangmin Xu, April D. Pyle, Matteo Pellegrini, S. Armando Villalta, Melissa J. Spencer
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 90-102 (2023)
High systemic doses of adeno-associated viruses (AAVs) have been associated with immune-related serious adverse events (SAEs). Although AAV was well tolerated in preclinical models, SAEs were observed in clinical trials, indicating the need for impro
Externí odkaz:
https://doaj.org/article/5f866c643097449da902b95f356bd37b
Autor:
Halli Benasutti, Joseph W. Maricelli, Jane Seto, John Hall, Christine Halbert, Jacqueline Wicki, Lydia Heusgen, Nicholas Purvis, Michael Regnier, David C. Lin, Buel D. Rodgers, Jeffrey S. Chamberlain
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 65-80 (2023)
Limb-girdle muscular dystrophy type R9 (LGMDR9) is a muscle-wasting disease that begins in the hip and shoulder regions of the body. This disease is caused by mutations in fukutin-related protein (FKRP), a glycosyltransferase critical for maintaining
Externí odkaz:
https://doaj.org/article/a9db696fcc7143e8a0480dc385b78ddf
Autor:
Arden B. Piepho, Jeovanna Lowe, Laurel R. Cumby, Lisa E. Dorn, Dana M. Lake, Neha Rastogi, Megan D. Gertzen, Sarah L. Sturgill, Guy L. Odom, Mark T. Ziolo, Federica Accornero, Jeffrey S. Chamberlain, Jill A. Rafael-Fortney
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 28, Iss , Pp 344-354 (2023)
Micro-dystrophin gene replacement therapies for Duchenne muscular dystrophy (DMD) are currently in clinical trials, but have not been thoroughly investigated for their efficacy on cardiomyopathy progression to heart failure. We previously validated F
Externí odkaz:
https://doaj.org/article/112376132c914e0588ee19091b2c6a91
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:
Frank Li, Justin Kolb, Julie Crudele, Paola Tonino, Zaynab Hourani, John E. Smith, Jeffrey S. Chamberlain, Henk Granzier
Publikováno v:
Skeletal Muscle, Vol 10, Iss 1, Pp 1-20 (2020)
Abstract Background Nebulin is a critical thin filament-binding protein that spans from the Z-disk of the skeletal muscle sarcomere to near the pointed end of the thin filament. Its massive size and actin-binding property allows it to provide the thi
Externí odkaz:
https://doaj.org/article/667904beb85d45bf8b0810fff5560535
Autor:
Stephen C. Kolwicz, Jr., PhD, John K. Hall, PhD, Farid Moussavi-Harami, MD, Xiolan Chen, PhD, Stephen D. Hauschka, PhD, Jeffrey S. Chamberlain, PhD, Michael Regnier, PhD, Guy L. Odom, PhD
Publikováno v:
JACC: Basic to Translational Science, Vol 4, Iss 7, Pp 778-791 (2019)
Summary: Mutations in the gene encoding for dystrophin leads to structural and functional deterioration of cardiomyocytes and is a hallmark of cardiomyopathy in Duchenne muscular dystrophy (DMD) patients. Administration of recombinant adeno-associate
Externí odkaz:
https://doaj.org/article/4212bb9493db490e828183607656df25
Autor:
Killian S. Hanlon, Benjamin P. Kleinstiver, Sara P. Garcia, Mikołaj P. Zaborowski, Adrienn Volak, Stefan E. Spirig, Alissa Muller, Alexander A. Sousa, Shengdar Q. Tsai, Niclas E. Bengtsson, Camilla Lööv, Martin Ingelsson, Jeffrey S. Chamberlain, David P. Corey, Martin J. Aryee, J. Keith Joung, Xandra O. Breakefield, Casey A. Maguire, Bence György
Publikováno v:
Nature Communications, Vol 10, Iss 1, Pp 1-11 (2019)
In-depth characterization of adeno-associated virus (AAV)-mediated CRISPR delivery is still lacking. Here, the authors show high levels of integration into Cas9-induced double-strand breaks (DSBs) in therapeutically relevant genes in vivo.
Externí odkaz:
https://doaj.org/article/d463e02a3bdc40c4aca343f4b409877a
Autor:
Tahnee L. Kennedy, Simon Guiraud, Ben Edwards, Sarah Squire, Lee Moir, Arran Babbs, Guy Odom, Diane Golebiowski, Joel Schneider, Jeffrey S. Chamberlain, Kay E. Davies
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 11, Iss , Pp 92-105 (2018)
Duchenne muscular dystrophy (DMD) is an X-linked muscle-wasting disease caused by mutations in the dystrophin gene. DMD boys are wheelchair-bound around 12 years and generally survive into their twenties. There is currently no effective treatment exc
Externí odkaz:
https://doaj.org/article/b4cdbb1c898240c9ae805bf8b86d8caa
Autor:
Zachary M. Howard, Lisa E. Dorn, Jeovanna Lowe, Megan D. Gertzen, Pierce Ciccone, Neha Rastogi, Guy L. Odom, Federica Accornero, Jeffrey S. Chamberlain, Jill A. Rafael-Fortney
Publikováno v:
JCI Insight, Vol 6, Iss 7 (2021)
Gene replacement for Duchenne muscular dystrophy (DMD) with micro-dystrophins has entered clinical trials, but efficacy in preventing heart failure is unknown. Although most patients with DMD die from heart failure, cardiomyopathy is undetectable unt
Externí odkaz:
https://doaj.org/article/edf8afa3bfcf4133a3ecce62112683f8