Zobrazeno 1 - 10
of 472
pro vyhledávání: '"K. Amaya"'
Autor:
Marti Cabanes-Creus, Renina Gale Navarro, Erhua Zhu, Grober Baltazar, Sophia H.Y. Liao, Matthieu Drouyer, Anais K. Amaya, Suzanne Scott, Loan Hanh Nguyen, Adrian Westhaus, Matthias Hebben, Laurence O.W. Wilson, Adrian J. Thrasher, Ian E. Alexander, Leszek Lisowski
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 24, Iss , Pp 88-101 (2022)
Recent clinical successes have intensified interest in using adeno-associated virus (AAV) vectors for therapeutic gene delivery. The liver is a key clinical target, given its critical physiological functions and involvement in a wide range of genetic
Externí odkaz:
https://doaj.org/article/1abf6bd1dabc4da8b922b404440e19d1
Autor:
Marti Cabanes-Creus, Adrian Westhaus, Renina Gale Navarro, Grober Baltazar, Erhua Zhu, Anais K. Amaya, Sophia H.Y. Liao, Suzanne Scott, Erwan Sallard, Kimberley L. Dilworth, Arkadiusz Rybicki, Matthieu Drouyer, Claus V. Hallwirth, Antonette Bennett, Giorgia Santilli, Adrian J. Thrasher, Mavis Agbandje-McKenna, Ian E. Alexander, Leszek Lisowski
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 1139-1154 (2020)
Use of the prototypical adeno-associated virus type 2 (AAV2) capsid delivered unexpectedly modest efficacy in an early liver-targeted gene therapy trial for hemophilia B. This result is consistent with subsequent data generated in chimeric mouse-huma
Externí odkaz:
https://doaj.org/article/192ac1b2202f420f9e1d2c4d0646e77d
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:
Adrian Westhaus, Marti Cabanes-Creus, Kimberley L. Dilworth, Erhua Zhu, David Salas Gómez, Renina G. Navarro, Anais K. Amaya, Suzanne Scott, Magdalena Kwiatek, Alexandra L. McCorkindale, Tara E. Hayman, Silke Frahm, Dany P. Perocheau, Bang Manh Tran, Elizabeth Vincan, Sharon L. Wong, Shafagh A. Waters, Georgina E. Riddiough, Marcos V. Perini, Laurence O.W. Wilson, Julien Baruteau, Sebastian Diecke, Gloria González-Aseguinolaza, Giorgia Santilli, Adrian J. Thrasher, Ian E. Alexander, Leszek Lisowski
The liver is a prime target for in vivo gene therapies using recombinant adeno-associated viral vectors (rAAV). Multiple clinical trials have been undertaken for this target in the past 15 years, however we are still to see market approval of the fir
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::456eba301b4b73f9427328cdc937aa92
http://edoc.mdc-berlin.de/23212/1/23212oa.pdf
http://edoc.mdc-berlin.de/23212/1/23212oa.pdf
Akademický článek
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Autor:
Sophia H.Y. Liao, Suzanne Scott, Erhua Zhu, Loan Hanh Nguyen, Adrian Westhaus, Adrian J. Thrasher, Grober Baltazar, Renina Gale Navarro, Anais K. Amaya, Marti Cabanes-Creus, Leszek Lisowski, Matthieu Drouyer, Laurence O. W. Wilson, Ian E. Alexander, Matthias Hebben
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 24, Iss, Pp 88-101 (2022)
Molecular Therapy: Methods & Clinical Development, Vol 24, Iss, Pp 88-101 (2022)
Recent clinical successes have intensified interest in using adeno-associated virus (AAV) vectors for therapeutic gene delivery. The liver is a key clinical target, given its critical physiological functions and involvement in a wide range of genetic
Autor:
Adrian Westhaus, Marti Cabanes-Creus, Kimberley L. Dilworth, Erhua Zhu, David Salas Gómez, Renina G. Navarro, Anais K. Amaya, Suzanne Scott, Magdalena Kwiatek, Alexandra L. McCorkindale, Tara E. Hayman, Silke Frahm, Dany P. Perocheau, Bang Manh Tran, Elizabeth Vincan, Sharon L. Wong, Shafagh A. Waters, Laurence O. W. Wilson, Julien Baruteau, Sebastian Diecke, Gloria González-Aseguinolaza, Giorgia Santilli, Adrian J. Thrasher, Ian E. Alexander, Leszek Lisowski
The liver is a prime target for in vivo gene therapies using recombinant adeno-associated viral vectors (rAAV). Multiple clinical trials have been undertaken for this target in the past 15 years, however we are still to see market approval of the fir
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::ae8acdce92d8cb8bbee102c1127b55a1
https://doi.org/10.1101/2022.09.28.510021
https://doi.org/10.1101/2022.09.28.510021
Akademický článek
Tento výsledek nelze pro nepřihlášené uživatele zobrazit.
K zobrazení výsledku je třeba se přihlásit.
K zobrazení výsledku je třeba se přihlásit.