Zobrazeno 1 - 10
of 64
pro vyhledávání: '"Rachael A Potter"'
Autor:
Rachael A. Potter, Ellyn L. Peterson, Danielle Griffin, Grace Cooper Olson, Sarah Lewis, Kyle Cochran, Jerry R. Mendell, Louise R. Rodino-Klapac
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 1, Pp 101195- (2024)
Patients with pre-existing immunity to adeno-associated virus (AAV) are currently unable to receive systemic gene transfer therapies. In this nonhuman primate study, we investigated the impact of immunosuppression strategies on gene transfer therapy
Externí odkaz:
https://doaj.org/article/cf55b910bf664423b112cf5813d36f07
Autor:
Young-Eun Seo, Stephen H. Baine, Amber N. Kempton, Oliver C. Rogers, Sarah Lewis, Kaitlin Adegboye, Alex Haile, Danielle A. Griffin, Ellyn L. Peterson, Eric R. Pozsgai, Rachael A. Potter, Louise R. Rodino-Klapac
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 28, Iss , Pp 284-299 (2023)
Limb-girdle muscular dystrophy (LGMD) type 2C/R5 results from mutations in the γ-sarcoglycan (SGCG) gene and is characterized by muscle weakness and progressive wasting. Loss of functional γ-sarcoglycan protein in the dystrophin-associated protein
Externí odkaz:
https://doaj.org/article/454f7bfe844c427ebf5263c914d5ec4d
Autor:
Rachael A. Potter, Danielle A. Griffin, Kristin N. Heller, Jerry R. Mendell, Louise R. Rodino-Klapac
Publikováno v:
Biology Open, Vol 12, Iss 9 (2023)
Externí odkaz:
https://doaj.org/article/718925be3ff24eaabe94799ea3ca29bd
Autor:
Jerry R. Mendell, Perry B. Shieh, Craig M. McDonald, Zarife Sahenk, Kelly J. Lehman, Linda P. Lowes, Natalie F. Reash, Megan A. Iammarino, Lindsay N. Alfano, Brenna Sabo, Jeremy D. Woods, Christy L. Skura, Howard C. Mao, Loretta A. Staudt, Danielle A. Griffin, Sarah Lewis, Shufang Wang, Rachael A. Potter, Teji Singh, Louise R. Rodino-Klapac
Publikováno v:
Frontiers in Cell and Developmental Biology, Vol 11 (2023)
Introduction: Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy designed for targeted expression of SRP-9001 dystrophin protein, a shortened dystrophin retaining key functional domains of the wild-type protein.Method
Externí odkaz:
https://doaj.org/article/b25a8c7747044bbf987c9093a8d8d79b
Autor:
Erin O’Rourke, Jerry R. Mendell, Zarife Sahenk, Kelly J. Lehman, Linda P. Lowes, Natalie F. Reash, Megan Iammarino, Lindsay Alfano, Sarah Lewis, Kathleen Church, Richard Shell, Rachael A. Potter, Danielle Griffin, Mark J. Hogan, Shufang Wang, Stefanie Mason, Eddie Darton, Louise Rodino-Klapac
Publikováno v:
Genetics in Medicine Open, Vol 1, Iss 1, Pp 100342- (2023)
Externí odkaz:
https://doaj.org/article/fb9e1b284811470595a4cfbc12d4ff7e
Publikováno v:
Computers in Human Behavior Reports, Vol 5, Iss , Pp 100160- (2022)
This paper explores practices and policies for managing information communication technologies (ICTs) and ensuring healthy and productive work environments in higher education settings. This was informed by: (1) semi-structured interviews with 12 sen
Externí odkaz:
https://doaj.org/article/82aedabdd80d425e966a6fb8e4202f9a
Autor:
Jerry R. Mendell, Eric R. Pozsgai, Rachael A. Potter, Danielle A. Griffin, Louise R. Rodino-Klapac, Zarife Sahenk, Kelly J. Lehman
Publikováno v:
Neurodegenerative Disease Management. 11:411-429
Limb-girdle muscular dystrophies (LGMDs) represent a major group of muscle disorders. Treatment is sorely needed and currently expanding based on safety and efficacy adopting principles of single-dosing gene therapy for monogenic autosomal recessive
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1a25fd46b66addc212d2bf2b512855e7
https://doi.org/10.2217/nmt-2020-0066
https://doi.org/10.2217/nmt-2020-0066
Publikováno v:
Journal of Neuromuscular Diseases
Recombinant micro-dystrophin genes are designed to treat Duchenne muscular dystrophy (DMD) by retaining dystrophin domains believed to play key functional roles while fitting the packaging capacity of adeno-associated virus vectors. Domains R1-3 are
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5f20e27eeba9964488eb7dfb2072d06a
http://europepmc.org/articles/PMC8385511
http://europepmc.org/articles/PMC8385511
Autor:
Eric Pozsgai, Danielle A. Griffin, Kristin N. Heller, Rachael A Potter, Louise R. Rodino-Klapac, E. Peterson
Publikováno v:
Human Gene Therapy
Limb-girdle muscular dystrophy type 2D/R3 (LGMD2D/R3) is a progressive muscular dystrophy that manifests with muscle weakness, respiratory abnormalities, and in rare cases cardiomyopathy. LGMD2D/R3 is caused by mutations in the SGCA gene resulting in
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::22791b5bf32f83e99fd025133ba9e748
https://doi.org/10.1089/hum.2019.199
https://doi.org/10.1089/hum.2019.199
Autor:
Danielle A. Griffin, Rachael A Potter, Jerry R. Mendell, Louise R. Rodino-Klapac, Emma K Clark, E. Peterson, Kristin N. Heller
Publikováno v:
Human Gene Therapy. 32:375-389
Duchenne muscular dystrophy (DMD) is a rare, X-linked, fatal, degenerative neuromuscular disease caused by mutations in the DMD gene. More than 2,000 mutations of the DMD gene are responsible for p...
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::2e07e8da95be6d222a8403932a5663c4
https://doi.org/10.1089/hum.2019.255
https://doi.org/10.1089/hum.2019.255