Zobrazeno 1 - 2
of 2
pro vyhledávání: '"Julia Parzych"'
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:
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