Zobrazeno 1 - 10
of 145
pro vyhledávání: '"Stephen D, Hauschka"'
Autor:
Jennifer B. Kwon, Adarsh R. Ettyreddy, Ashish Vankara, Joel D. Bohning, Garth Devlin, Stephen D. Hauschka, Aravind Asokan, Charles A. Gersbach
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 19, Iss , Pp 320-329 (2020)
Delivery of therapeutic transgenes with adeno-associated viral (AAV) vectors for treatment of myopathies has yielded encouraging results in animal models and early clinical studies. Although certain AAV serotypes efficiently target muscle fibers, tra
Externí odkaz:
https://doaj.org/article/027bf4f182644f8e99d5b59e596aa1d1
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:
Niclas E. Bengtsson, John K. Hall, Guy L. Odom, Michael P. Phelps, Colin R. Andrus, R. David Hawkins, Stephen D. Hauschka, Joel R. Chamberlain, Jeffrey S. Chamberlain
Publikováno v:
Nature Communications, Vol 8, Iss 1, Pp 1-10 (2017)
CRISPR/Cas9-mediated gene editing is an emerging strategy to treat Duchenne muscular dystrophy. Here the authors develop multiple CRISPR/Cas9-based approaches to correct different dystrophin gene mutations, and show significant restoration of dystrop
Externí odkaz:
https://doaj.org/article/42b84f4d2d7e485cb3c0f8f3e7eaf353
Autor:
Kassandra S. Thomson, PhD, Guy L. Odom, PhD, Charles E. Murry, MD, PhD, Gregory G. Mahairas, PhD, Farid Moussavi-Harami, MD, Sam L. Teichman, MD, Xiaolan Chen, PhD, Stephen D. Hauschka, PhD, Jeffrey S. Chamberlain, PhD, Michael Regnier, PhD
Publikováno v:
JACC: Basic to Translational Science, Vol 1, Iss 7, Pp 666-679 (2016)
Despite recent advances, chronic heart failure remains a significant and growing unmet medical need, reaching epidemic proportions carrying substantial morbidity, mortality, and costs. A safe and convenient therapeutic agent that produces sustained i
Externí odkaz:
https://doaj.org/article/d156eb416a674b9bb2f4b5a526ecf031
Autor:
Ketaki N Mhatre, Julie Mathieu, Amy Martinson, Galina Flint, Leslie P. Blakley, Arash Tabesh, Hans Reinecke, Xiulan Yang, Xuan Guan, Eesha Murali, Jordan M Klaiman, Guy L Odom, Mary Beth Brown, Rong Tian, Stephen D Hauschka, Daniel Raftery, Farid Moussavi-Harami, Michael Regnier, Charles E Murry
Transplanted human pluripotent stem cell-derived cardiomyocytes (hPSC-CMs) improve ventricular performance when delivered acutely post-myocardial infarction but are ineffective in chronic myocardial infarction/heart failure. 2’-deoxy-ATP (dATP) act
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::e665b470ba9e0494d5530ab7fecca44b
https://doi.org/10.1101/2023.04.24.538108
https://doi.org/10.1101/2023.04.24.538108
Autor:
Sharla M. Birch, Michael W. Lawlor, Thomas J. Conlon, Lee-Jae Guo, Julie M. Crudele, Eleanor C. Hawkins, Peter P. Nghiem, Mihye Ahn, Hui Meng, Margaret J. Beatka, Brittany A. Fickau, Juan C. Prieto, Martin A. Styner, Michael J. Struharik, Courtney Shanks, Kristy J. Brown, Diane Golebiowski, Amanda K. Bettis, Cynthia J. Balog-Alvarez, Nathalie Clement, Kirsten E. Coleman, Manuela Corti, Xiufang Pan, Stephen D. Hauschka, J. Patrick Gonzalez, Carl A. Morris, Joel S. Schneider, Dongsheng Duan, Jeffrey S. Chamberlain, Barry J. Byrne, Joe. N. Kornegay
Publikováno v:
Science translational medicine. 15(677)
Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by the absence of dystrophin, a membrane-stabilizing protein encoded by the DMD gene. Although mouse models of DMD provide insight into the potential of a corrective the
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::286334212cdab7cd1ec6f09084ab2d3f
https://pubmed.ncbi.nlm.nih.gov/36599002
https://pubmed.ncbi.nlm.nih.gov/36599002
Publikováno v:
Mol Ther
Gene editing is often touted as a permanent method for correcting mutations, but its long-term benefits in Duchenne muscular dystrophy (DMD) may depend on sufficiently high editing efficiencies to halt muscle degeneration. Here, we explored the persi
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::418b4b5a189776174b4dca1a60b402ff
https://doi.org/10.1016/j.ymthe.2020.11.003
https://doi.org/10.1016/j.ymthe.2020.11.003
Autor:
Svyatoslav Dvoretskiy, M. Carmen Valero, Samuel Lapp, M S Stephen D Hauschka, Yu-Fu Wu, Heather D. Huntsman, Marni D. Boppart, Ziad S. Mahmassani, Dean J. Burkin, Koyal Garg
Publikováno v:
J Gerontol A Biol Sci Med Sci
Anabolic resistance to a mechanical stimulus may contribute to the loss of skeletal muscle mass observed with age. In this study, young and aged mice were injected with saline or human LM-111 (1 mg/kg). One week later, the myotendinous junction of th
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::895deec7083669b4f52689c765fdaf65
https://doi.org/10.1093/gerona/glaa308
https://doi.org/10.1093/gerona/glaa308
Autor:
Niclas E. Bengtsson, Julie M. Crudele, Jordan M. Klaiman, Christine L. Halbert, Stephen D. Hauschka, Jeffrey S. Chamberlain
Publikováno v:
Mol Ther
Gene editing has shown promise for correcting or bypassing dystrophin mutations in Duchenne muscular dystrophy (DMD). However, preclinical studies have focused on young animals with limited muscle fibrosis and wasting, thereby favoring muscle transdu
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1f33fd42d19d0dd0ec239a07eb87d1d4
https://europepmc.org/articles/PMC9171147/
https://europepmc.org/articles/PMC9171147/
Autor:
Julian Ramos, James M. Allen, Jeffrey S. Chamberlain, Niclas E. Bengtsson, Stephen D. Hauschka, Katrin Hollinger
Publikováno v:
Molecular Therapy. 27:623-635
Gene therapies using adeno-associated viral (AAV) vectors have advanced into clinical trials for several diseases, including Duchenne muscular dystrophy (DMD). A limitation of AAV is the carrying capacity (∼5 kb) available for genes and regulatory
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::1c5b10c4a8af9439076fdddad3811c20
https://doi.org/10.1016/j.ymthe.2019.01.002
https://doi.org/10.1016/j.ymthe.2019.01.002