Zobrazeno 1 - 10
of 89
pro vyhledávání: '"Kristy J, Brown"'
Publikováno v:
Skeletal Muscle, Vol 14, Iss 1, Pp 1-11 (2024)
Abstract Background Multiple clinical trials to assess the efficacy of AAV-directed gene transfer in participants with Duchenne muscular dystrophy (DMD) are ongoing. The success of these trials currently relies on standard functional outcome measures
Externí odkaz:
https://doaj.org/article/4757d38c542643cbadde796ef0c74dc7
Autor:
Jessica F, Boehler, Kristy J, Brown, Margaret, Beatka, J Patrick, Gonzalez, Roxana, Donisa Dreghici, Meghan, Soustek-Kramer, Sharon, McGonigle, Annie, Ganot, Timothy, Palmer, Caitlin, Lowie, Jeffrey S, Chamberlain, Michael W, Lawlor, Carl A, Morris
Publikováno v:
Neuromuscular Disorders. 33:40-49
Accelerated approval based on a likely surrogate endpoint can be life-changing for patients suffering from a rare progressive disease with unmet medical need, as it substantially hastens access to potentially lifesaving therapies. In one such example
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ae84146b67be7581f1dbbb80f22975d3
https://doi.org/10.1016/j.nmd.2022.12.007
https://doi.org/10.1016/j.nmd.2022.12.007
Autor:
Samuel Rivero-Hinojosa, Ling San Lau, Mojca Stampar, Jerome Staal, Huizhen Zhang, Heather Gordish-Dressman, Paul A. Northcott, Stefan M. Pfister, Michael D. Taylor, Kristy J. Brown, Brian R. Rood
Publikováno v:
Acta Neuropathologica Communications, Vol 6, Iss 1, Pp 1-19 (2018)
Abstract Genomic characterization has begun to redefine diagnostic classifications of cancers. However, it remains a challenge to infer disease phenotypes from genomic alterations alone. To help realize the promise of genomics, we have performed a qu
Externí odkaz:
https://doaj.org/article/b2ef1794bc3d4451bd0ab50e6ec884e1
Autor:
Jeffrey S. Chamberlain, Melissa Robb, Serge Braun, Kristy J. Brown, Olivier Danos, Annie Ganot, Pedro Gonzalez-Alegre, Nina Hunter, Craig McDonald, Carl Morris, Mark Tobolowsky, Kathryn R. Wagner, Olivia Ziolkowski, Dongsheng Duan
Publikováno v:
Human gene therapy, vol 34, iss 9-10
Duchenne muscular dystrophy (DMD) is a serious, rare genetic disease, affecting primarily boys. It is caused by mutations in the DMD gene and is characterized by progressive muscle degeneration that results in loss of function and early death due to
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d4cd9d835233f4a077af22bfee836cfc
https://escholarship.org/uc/item/0v56c0vk
https://escholarship.org/uc/item/0v56c0vk
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
Autor:
Christopher B. Tully, Terence A. Partridge, Utkarsh J. Dang, Ravi Hindupur, Davi A. G. Mázala, James S. Novak, Kristy J. Brown, Yetrib Hathout, Emily H. Canessa, Alyson A. Fiorillo, Kanneboyina Nagaraju, Rita Spathis
Publikováno v:
Journal of Neuromuscular Diseases
Recently, the Food and Drug Administration granted accelerated approvals for four exon skipping therapies –Eteplirsen, Golodirsen, Viltolarsen, and Casimersen –for Duchenne Muscular Dystrophy (DMD). However, these treatments have only demonstrate
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::aeeee445cf10ae357bc01250c19e86ef
https://doi.org/10.3233/jnd-210696
https://doi.org/10.3233/jnd-210696
Autor:
Ryan M. Kendra, Joanna E. Parkes, Kristy J. Brown, Emily M. Ott, John Mcdonald, Rita Spathis, Ning Li, Kanneboyina Nagaraju, Michael W. Lawlor, Melissa Morales
Publikováno v:
Journal of Neuromuscular Diseases. 8:S325-S340
Background: AAV-based gene therapy is an attractive approach to treat Duchenne muscular dystrophy (DMD) patients. Although the long-term consequences of a gene therapy approach for DMD are unknown, there is evidence in both DMD patients and animal mo
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d42506ca2b90f8d54ac554b9907d21ff
https://doi.org/10.3233/jnd-210706
https://doi.org/10.3233/jnd-210706
Autor:
Alyson A. Fiorillo, Christopher R. Heier, James S. Novak, Christopher B. Tully, Kristy J. Brown, Kitipong Uaesoontrachoon, Maria C. Vila, Peter P. Ngheim, Luca Bello, Joe N. Kornegay, Corrado Angelini, Terence A. Partridge, Kanneboyina Nagaraju, Eric P. Hoffman
Publikováno v:
Cell Reports, Vol 12, Iss 10, Pp 1678-1690 (2015)
The amount and distribution of dystrophin protein in myofibers and muscle is highly variable in Becker muscular dystrophy and in exon-skipping trials for Duchenne muscular dystrophy. Here, we investigate a molecular basis for this variability. In mus
Externí odkaz:
https://doaj.org/article/de3f64e4552f4c9aa4b50be9b61059d2
Autor:
Aiping Zhang, Kitipong Uaesoontrachoon, Conner Shaughnessy, Jharna R. Das, Sree Rayavarapu, Kristy J. Brown, Patricio E. Ray, Kanneboyina Nagaraju, John N. van den Anker, Eric P. Hoffman, Yetrib Hathout
Publikováno v:
Toxicology Reports, Vol 2, Iss C, Pp 838-849 (2015)
Phosphorodiamidate morpholino oligonucleotides (PMO) are used as a promising exon-skipping gene therapy for Duchenne muscular dystrophy (DMD). One potential complication of high dose PMO therapy is its transient accumulation in the kidneys. Therefore
Externí odkaz:
https://doaj.org/article/125cba0abb4b4b699ceae9c3fc968255
Effect of endurance exercise on microRNAs in myositis skeletal muscle-A randomized controlled study.
Autor:
Jessica F Boehler, Marshall W Hogarth, Matthew D Barberio, James S Novak, Svetlana Ghimbovschi, Kristy J Brown, Li Alemo Munters, Ingela Loell, Yi-Wen Chen, Heather Gordish-Dressman, Helene Alexanderson, Ingrid E Lundberg, Kanneboyina Nagaraju
Publikováno v:
PLoS ONE, Vol 12, Iss 8, p e0183292 (2017)
To identify changes in skeletal muscle microRNA expression after endurance exercise and associate the identified microRNAs with mRNA and protein expression to disease-specific pathways in polymyositis (PM) and dermatomyositis (DM) patients.Following
Externí odkaz:
https://doaj.org/article/d90075eef8b740c6bdb09916a05214c1