Zobrazeno 1 - 10
of 12
pro vyhledávání: '"Meghan Blackwood"'
Autor:
Marina Zieger, Florie Borel, Cynthia Greer, Gwladys Gernoux, Meghan Blackwood, Terence R. Flotte, Christian Mueller
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 25, Iss , Pp 425-438 (2022)
α1-antitrypsin deficiency is a rare genetic condition that can cause liver and/or lung disease. There is currently no cure for this disorder, although repeated infusions of plasma-purified protein may slow down emphysema progression. Gene therapy in
Externí odkaz:
https://doaj.org/article/dd33fcbe12cd471e89896bea164e7f7b
Autor:
Motahareh Arjomandnejad, Katelyn Sylvia, Meghan Blackwood, Thomas Nixon, Qiushi Tang, Manish Muhuri, Alisha M. Gruntman, Guangping Gao, Terence R. Flotte, Allison M. Keeler
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 23, Iss , Pp 490-506 (2021)
Immune responses to adeno-associated virus (AAV) capsids limit the therapeutic potential of AAV gene therapy. Herein, we model clinical immune responses by generating AAV capsid-specific chimeric antigen receptor (AAV-CAR) T cells. We then modulate i
Externí odkaz:
https://doaj.org/article/1d3794b4a1154ad0b895efd390f57d2a
Autor:
Angela Lek, Brenda Wong, Allison Keeler, Meghan Blackwood, Kaiyue Ma, Shushu Huang, Katelyn Sylvia, Ana Rita Batista, Rebecca Artinian, Danielle Kokoski, Shestruma Parajuli, Juan Putra, Chrystalle Katte Carreon, Hart Lidov, Keryn Woodman, Sander Pajusalu, Janelle Spinazzola, Thomas Gallagher, Joan LaRovere, Diane Baulderson, Lauren Black, Keith Sutton, Richard Horgan, Monkol Lek, Terence Flotte
An N-of-1 trial was developed to deliver a dCas9-VP64 transgene designed to upregulate the cortical dystrophin as a custom therapy for a Duchenne muscular dystrophy (DMD) patient. After showing signs of mild cardiac dysfunction and pericardial effusi
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::28c0f416fcddad83a5bbc110f034ef08
https://doi.org/10.1101/2023.05.16.23289881
https://doi.org/10.1101/2023.05.16.23289881
Autor:
Terence R. Flotte, Oguz Cataltepe, Ajit Puri, Ana Rita Batista, Richard Moser, Diane McKenna-Yasek, Catherine Douthwright, Gwladys Gernoux, Meghan Blackwood, Christian Mueller, Phillip W. L. Tai, Xuntian Jiang, Scot Bateman, Spiro G. Spanakis, Julia Parzych, Allison M. Keeler, Aly Abayazeed, Saurabh Rohatgi, Laura Gibson, Robert Finberg, Bruce A. Barton, Zeynep Vardar, Mohammed Salman Shazeeb, Matthew Gounis, Cynthia J. Tifft, Florian S. Eichler, Robert H. Brown, Douglas R. Martin, Heather L. Gray-Edwards, Miguel Sena-Esteves
Publikováno v:
Nature Medicine. 28:251-259
Tay-Sachs disease (TSD) is an inherited neurological disorder caused by deficiency of hexosaminidase A (HexA). Here, we describe an adeno-associated virus (AAV) gene therapy expanded-access trial in two patients with infantile TSD (IND 18225) with sa
Autor:
Thomas Nixon, Alisha M. Gruntman, Manish Muhuri, Katelyn Sylvia, Qiushi Tang, Meghan Blackwood, Motahareh Arjomandnejad, Terence R. Flotte, Guangping Gao, Allison M. Keeler
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 23, Iss, Pp 490-506 (2021)
Molecular Therapy: Methods & Clinical Development, Vol 23, Iss, Pp 490-506 (2021)
Immune responses to adeno-associated virus (AAV) capsids limit the therapeutic potential of AAV gene therapy. Herein, we model clinical immune responses by generating AAV capsid-specific chimeric antigen receptor (AAV-CAR) T cells. We then modulate i
Autor:
Nicholas Wightman, Merit Cudkowicz, Margaret A. Owegi, Dario Gelevski, Gwladys Gernoux, Meghan Blackwood, Christian Mueller, James D. Berry, Sarah Luppino, Catherine Douthwright, Robert H. Brown, Terrence R. Flotte, Derek H. Oakley, Diane McKenna-Yasek, Lindsay Pothier, Matthew P. Frosch
Publikováno v:
New England Journal of Medicine. 383:151-158
Two patients with familial amyotrophic lateral sclerosis (ALS) and mutations in the gene encoding superoxide dismutase 1 (SOD1) were treated with a single intrathecal infusion of adeno-associated virus encoding a microRNA targeting SOD1. In Patient 1
Autor:
Gwladys Gernoux, Marina Zieger, Terence R. Flotte, Christian Mueller, Alisha M. Gruntman, Meghan Blackwood
Publikováno v:
Molecular Therapy
With the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) approvals for Zolgensma, Luxturna, and Glybera, recombinant adeno-associated viruses (rAAVs) are considered efficient tools for gene transfer. However, studies in anim
Autor:
Marina, Zieger, Florie, Borel, Cynthia, Greer, Gwladys, Gernoux, Meghan, Blackwood, Terence R, Flotte, Christian, Mueller
Publikováno v:
Molecular therapy. Methodsclinical development. 25
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Autor:
Ana Rita Batista, Mohammed Salman Shazeeb, Richard P. Moser, Robert H. Brown, Florian Eichler, Spiro Spanakis, Miguel Sena-Esteves, Laura Gibson, Matthew J. Gounis, Christian Mueller, Xuntian Jiang, Oguz Cataltepe, Allison M. Keeler, Bruce A. Barton, Catherine Douthwright, Diane McKenna-Yasek, Scot Bateman, Saurabh Rohatgi, Heather L. Gray-Edwards, Z Vardar, Aly Abayazeed, Terence R. Flotte, Julia Parzych, Robert W. Finberg, Meghan Blackwood, Douglas R. Martin, Gwladys Gernoux, Ajit S. Puri
Tay-Sachs Disease (TSD) is an inherited neurological disorder caused by deficiency of hexosaminidase A (HexA). Preclinical work demonstrated safety and efficacy of CNS gene therapy using AAVrh8-HEXA/HEXB. Here we describe an expanded access trial in
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::eb434a3336893bbf1437fc36aa8a7bb0
https://doi.org/10.21203/rs.3.rs-195847/v1
https://doi.org/10.21203/rs.3.rs-195847/v1
Autor:
Ann-Kristin U. Friedrich, Ron Korstanje, Giacomo Basadonna, Mitchell A. Cahan, Natalie Pozzi, Eric W. Dickson, Meghan Blackwood, Nicholas L. Fowler, Dean E. Beyer, Kahraman Tanriverdi, Jahnavi Kakuturu, Jane E. Freedman, Chris Mueller, Jerrold L. Belant, Milka Koupenova, J. Alexander Palesty, Marjory B. Brooks, Alper Kucukural, Amanda Fazzalari
Publikováno v:
The Journal of surgical research. 257
Background Hibernating American black bears have significantly different clotting parameters than their summer active counterparts, affording them protection against venous thromboembolism during prolonged periods of immobility. We sought to evaluate