Zobrazeno 1 - 10
of 24
pro vyhledávání: '"Allison P Dane"'
Autor:
Thibaut Marais, Federico Mingozzi, Elena Barbon, Maria-Grazia Biferi, Sandra Le Quellec, Claude Negrier, Amit C. Nathawani, Nathalie Enjolras, Jean-Claude Bordet, Allison P Dane, Yesim Dargaud
Publikováno v:
Thromb Haemost
Thromb Haemost, 2019, 119, pp.1956-1967. ⟨10.1055/s-0039-1697658⟩
Thromb Haemost, 2019, 119, pp.1956-1967. ⟨10.1055/s-0039-1697658⟩
Gene therapy using recombinant adeno-associated virus (AAV) has induced sustained long-term coagulation human factor IX (hFIX) levels in hemophilia B (HB) patients. However, asymptomatic transient liver toxicity was observed at high vector doses, hig
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::377687d168dd27a1d86f0863c65320ca
https://hal.archives-ouvertes.fr/hal-02880804
https://hal.archives-ouvertes.fr/hal-02880804
Autor:
Venette Fannin, Natalie Northcott, Jaminder Khinder, Benjamin Liou, Elisa Chisari, Amit C. Nathwani, Miriam Canavese, Jalpa Pandya, Rachel Blackwood, Azadeh Kia, Romuald Corbau, Ying Sun, Rose Sheridan, Carlos Henrique Miranda, Allison P Dane
Publikováno v:
Molecular Genetics and Metabolism. 129:S110
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::da31d49f86bc22c8188f159463ddc8b7
https://doi.org/10.1016/j.ymgme.2019.11.283
https://doi.org/10.1016/j.ymgme.2019.11.283
Autor:
Jenny McIntosh, Nathalie Enjolras, Amit C. Nathwani, Allison P Dane, Claude Negrier, Cecilia Rosales, Sandra Le Quellec
Publikováno v:
Haemophilia : the official journal of the World Federation of Hemophilia. 25(1)
Introduction: The variety of treatment for haemophilia B (HB) has recently improved with the emergence of both AAV‐based gene therapy and bioengineered human factor IX (hFIX) molecules with prolonged half‐life due to fusion to either albumin (Alb
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0ef8c49fdfd09d557c49eb103a1deb1a
https://pubmed.ncbi.nlm.nih.gov/30520547
https://pubmed.ncbi.nlm.nih.gov/30520547
Autor:
Andrew M. Davidoff, Nicole K. Paulk, Sean Nygaard, Feijie Zhang, Amit C. Nathwani, Annelise Haft, Bin Li, Katja Pekrun, Marcus B. Valentine, Kirk Chu, Mark A. Kay, Markus Grompe, Erhua Zhu, Jianpeng Xu, Christopher L. Morton, Ian E. Alexander, Allison P Dane, Yue Zhang, Federico Mingozzi, Leszek Lisowski, Christian Leborgne
Publikováno v:
Molecular Therapy
Molecular Therapy, Nature Publishing Group, 2018, 26, pp.289-303. ⟨10.1016/j.ymthe.2017.09.021⟩
Molecular Therapy, Cell Press, 2018, 26, pp.289-303. ⟨10.1016/j.ymthe.2017.09.021⟩
Molecular Therapy, 2018, 26, pp.289-303. ⟨10.1016/j.ymthe.2017.09.021⟩
Molecular Therapy, Nature Publishing Group, 2018, 26, pp.289-303. ⟨10.1016/j.ymthe.2017.09.021⟩
Molecular Therapy, Cell Press, 2018, 26, pp.289-303. ⟨10.1016/j.ymthe.2017.09.021⟩
Molecular Therapy, 2018, 26, pp.289-303. ⟨10.1016/j.ymthe.2017.09.021⟩
International audience; Existing recombinant adeno-associated virus (rAAV) serotypes for delivering in vivo gene therapy treatments for human liver diseases have not yielded combined high-level human hepatocyte transduction and favorable humoral neut
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::83ee65c4d3d4bc6def4182dd8b883784
https://hal.archives-ouvertes.fr/hal-02880809
https://hal.archives-ouvertes.fr/hal-02880809
Publikováno v:
Gene Therapy. 22:917-922
Recombinant vectors based on adeno-associated virus (AAV) are proving to be powerful tools for genetic manipulation of the liver, for both discovery and therapeutic purposes. The system can be used to deliver transgene cassettes for expression or, al
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8297e4bb4fa8a1feb3bf36b26de1b61a
https://doi.org/10.1038/gt.2015.66
https://doi.org/10.1038/gt.2015.66
Autor:
Emilie E. Wilkie, Christine M. Smyth, Grant J Logan, Claus V. Hallwirth, Anais K. Amaya, Neeta Khandekar, Sophia H.Y. Liao, Allison P Dane, Leszek Lisowski, Sharon C. Cunningham, David W. Russell, Patrick P.L. Tam, Marti Cabanes-Creus, Ian E. Alexander, Samantha L. Ginn, Erhua Zhu, Natsuki Sasaki
Publikováno v:
Nature genetics. 49(8)
Vectors based on adeno-associated virus type 2 (AAV2) are powerful tools for gene transfer and genome editing applications. The level of interest in this system has recently surged in response to reports of therapeutic efficacy in human clinical tria
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e89c42d12f92cc091f041a3d030cec81
https://pubmed.ncbi.nlm.nih.gov/28628105
https://pubmed.ncbi.nlm.nih.gov/28628105
Autor:
Olivier D. Christophe, Andreas Schulze, Elisa Chisari, Allison P Dane, Azadeh Kia, Jalpa Pandya, Maria Portillo, Fabrizio Comper, Renee Kober, Segen Negash, Cécile V. Denis, Jonathan H. Foley, Florian Sonntag, Hattie Ollerton, Samantha Correia, Jenny McIntosh, Jaminder Khinder, Rebecca Alade, Jey Jeyakumar, Clement Cocita, Caterina Casari, Romuald Corbau, Dodev Tihomir, Erald Shehu
Publikováno v:
Blood. 134:4638-4638
Background: Several first-generation gene therapies are currently in clinical trials for Haemophilia A. These trials have to date exhibited varying results including issues with large patient to patient variation in FVIII levels and lack of durabilit
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::489ef7d4d8af0a5659b8f2114ee06f39
https://doi.org/10.1182/blood-2019-128490
https://doi.org/10.1182/blood-2019-128490
Autor:
Jalpa Pandya, Jey Jeyakumar, Rose Sheridan, Maria Portillo, Elisa Chisari, Miriam Canavese, Carlos Henrique Miranda, Clement Cocita, Amit C. Nathwani, Romuald Corbau, Jonathan H. Foley, Azadeh Kia, Allison P Dane, Jenny McIntosh
Publikováno v:
Blood. 134:3354-3354
Introduction: Gaucher disease (GD), one of the most common lysosomal storage disorders, is an autosomal recessive condition resulting from mutations in the GBA gene that codes for the b-glucocerebrosidase (GCase) enzyme. Over 90% of patients have typ
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::1ed9d03622a53774c9a13f35e51280bb
https://doi.org/10.1182/blood-2019-124280
https://doi.org/10.1182/blood-2019-124280
Autor:
Mark A. Kay, Markus Grompe, Yue Zhang, Kirk Chu, Ian E. Alexander, Sean Nygaard, Sharon C. Cunningham, Elizabeth M. Wilson, Leszek Lisowski, Allison P Dane
Publikováno v:
Nature
Recombinant adeno-associated viral (rAAV) vectors have shown early promise in clinical trials. The therapeutic transgene cassette can be packaged in different AAV capsid pseudotypes, each having a unique transduction profile. At present, rAAV capsid
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::eb34307a60dedeaa568d76f329a1ca16
http://europepmc.org/articles/PMC3939040
http://europepmc.org/articles/PMC3939040
Autor:
Allison P Dane, Cindy Kok, Philip W. Kuchel, Sharon C. Cunningham, Kevin Carpenter, Grant J Logan, Ian E. Alexander, Susan M. Siew
Publikováno v:
Molecular Therapy. 21:1823-1831
Viral vectors based on adeno-associated virus (AAV) are showing exciting promise in gene therapy trials targeting the adult liver. A major challenge in extending this promise to the pediatric liver is the loss of episomal vector genomes that accompan
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5b6cbc76b35760589755e92a41fabb70
https://doi.org/10.1038/mt.2013.139
https://doi.org/10.1038/mt.2013.139