Zobrazeno 1 - 10
of 51
pro vyhledávání: '"Samantha L. Ginn"'
Autor:
Matthieu Drouyer, Tak-Ho Chu, Elodie Labit, Florencia Haase, Renina Gale Navarro, Deborah Nazareth, Nicole Rosin, Jessica Merjane, Suzanne Scott, Marti Cabanes-Creus, Adrian Westhaus, Erhua Zhu, Rajiv Midha, Ian E. Alexander, Jeff Biernaskie, Samantha L. Ginn, Leszek Lisowski
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 2, Pp 101234- (2024)
Gene therapies and associated technologies are transforming biomedical research and enabling novel therapeutic options for patients living with debilitating and incurable genetic disorders. The vector system based on recombinant adeno-associated vira
Externí odkaz:
https://doaj.org/article/25d01544733d4f349e805a8d768d1b0c
Autor:
Lara E. Graves, Eva B. van Dijk, Erhua Zhu, Sundar Koyyalamudi, Tiffany Wotton, Dinah Sung, Shubha Srinivasan, Samantha L. Ginn, Ian E. Alexander
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 2, Pp 101232- (2024)
Despite the availability of life-saving corticosteroids for 70 years, treatment for adrenal insufficiency is not able to recapitulate physiological diurnal cortisol secretion and results in numerous complications. Gene therapy is an attractive possib
Externí odkaz:
https://doaj.org/article/387ce34d92fb4972a41cda974893bd95
Autor:
Cindy Y. Kok, Shinya Tsurusaki, Marti Cabanes-Creus, Sindhu Igoor, Renuka Rao, Rhys Skelton, Sophia H.Y. Liao, Samantha L. Ginn, Maddison Knight, Suzanne Scott, Mario Mietzsch, Rebecca Fitzsimmons, Jessica Miller, Tamer M.A. Mohamed, Robert McKenna, James J.H. Chong, Adam P. Hill, James E. Hudson, Ian E. Alexander, Leszek Lisowski, Eddy Kizana
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 459-473 (2023)
Recombinant adeno-associated viruses (rAAVs) have emerged as one of the most promising gene therapy vectors that have been successfully used in pre-clinical models of heart disease. However, this has not translated well to humans due to species diffe
Externí odkaz:
https://doaj.org/article/0a04499d5cb345478b523f2075186f26
Autor:
Marti Cabanes-Creus, Samantha L. Ginn, Anais K. Amaya, Sophia H.Y. Liao, Adrian Westhaus, Claus V. Hallwirth, Patrick Wilmott, Jason Ward, Kimberley L. Dilworth, Giorgia Santilli, Arkadiusz Rybicki, Hiroyuki Nakai, Adrian J. Thrasher, Adrian C. Filip, Ian E. Alexander, Leszek Lisowski
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 12, Iss , Pp 71-84 (2019)
Adeno-associated virus (AAV) vectors have become one of the most widely used gene transfer tools in human gene therapy. Considerable effort is currently being focused on AAV capsid engineering strategies with the aim of developing novel variants with
Externí odkaz:
https://doaj.org/article/e371436ebdb346fc97df68b085539c0c
Autor:
Samantha L. Ginn, Anais K. Amaya, Sophia H.Y. Liao, Erhua Zhu, Sharon C. Cunningham, Michael Lee, Claus V. Hallwirth, Grant J. Logan, Szun S. Tay, Anthony J. Cesare, Hilda A. Pickett, Markus Grompe, Kimberley Dilworth, Leszek Lisowski, Ian E. Alexander
Publikováno v:
JHEP Reports, Vol 2, Iss 1, Pp - (2020)
Background & Aims: Genome editing technology has immense therapeutic potential and is likely to rapidly supplant contemporary gene addition approaches. Key advantages include the capacity to directly repair mutant loci with resultant recovery of phys
Externí odkaz:
https://doaj.org/article/81b618aa4be6420b93bda12009cd595b
Autor:
Samantha L. Ginn, Claus V. Hallwirth, Sophia H.Y. Liao, Erdahl T. Teber, Jonathan W. Arthur, Jianmin Wu, Hong Ching Lee, Szun S. Tay, Min Hu, Roger R. Reddel, Matthew P. McCormack, Adrian J. Thrasher, Marina Cavazzana, Stephen I. Alexander, Ian E. Alexander
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 6, Iss , Pp 1-14 (2017)
In early gene therapy trials for SCID-X1, using γ-retroviral vectors, T cell leukemias developed in a subset of patients secondary to insertional proto-oncogene activation. In contrast, we have reported development of T cell leukemias in SCID-X1 mic
Externí odkaz:
https://doaj.org/article/b02280667afa4fc3ad72ef3d4c6c6486
Publikováno v:
Journal of Bone and Mineral Research. 37:826-836
Osteogenesis imperfecta (OI) describes a series of genetic bone fragility disorders that can have a substantive impact on patient quality of life. The multidisciplinary approach to management of children and adults with OI primarily involves the admi
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0b64a0d307c6d00d959ae52e6198b0af
https://doi.org/10.1002/jbmr.4549
https://doi.org/10.1002/jbmr.4549
Publikováno v:
Progress in molecular biology and translational science. 182
Liver-targeted genome editing offers the prospect of life-long therapeutic benefit following a single treatment and is set to rapidly supplant conventional gene addition approaches. Combining progress in liver-targeted gene delivery with genome editi
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::165e7c09b769610ae1eee4edb6aa73bb
https://pubmed.ncbi.nlm.nih.gov/34175044
https://pubmed.ncbi.nlm.nih.gov/34175044
Autor:
Kimberley L. Dilworth, Adrian J. Thrasher, Arkadiusz Rybicki, Claus V. Hallwirth, Anais K. Amaya, Adrian C. Filip, Leszek Lisowski, Jason Ward, Sophia H.Y. Liao, Ian E. Alexander, Samantha L. Ginn, Patrick Wilmott, Hiroyuki Nakai, Giorgia Santilli, Marti Cabanes-Creus, Adrian Westhaus
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 12, Iss, Pp 71-84 (2019)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
Adeno-associated virus (AAV) vectors have become one of the most widely used gene transfer tools in human gene therapy. Considerable effort is currently being focused on AAV capsid engineering strategies with the aim of developing novel variants with
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b603777be8173bc3bfabc33565b4b7ca
http://www.sciencedirect.com/science/article/pii/S2329050118301128
http://www.sciencedirect.com/science/article/pii/S2329050118301128