Zobrazeno 1 - 10
of 21
pro vyhledávání: '"Rose Sheridan"'
Publikováno v:
Human Gene Therapy. 33:879-888
Gene therapy is an exciting therapeutic concept that offers the promise of a cure for an array of inherited and acquired disorders. The liver has always been a key target for gene therapy as it controls essential biological processes including digest
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::abe0b0c1468d167ebed270cd086b470b
https://doi.org/10.1089/hum.2022.169
https://doi.org/10.1089/hum.2022.169
Autor:
Jey M. Jeyakumar, Azadeh Kia, Lawrence C. S. Tam, Jenny McIntosh, Justyna Spiewak, Kevin Mills, Wendy Heywood, Elisa Chisari, Noemi Castaldo, Daniël Verhoef, Paniz Hosseini, Petya Kalcheva, Clement Cocita, Carlos J. Miranda, Miriam Canavese, Jaminder Khinder, Cecilia Rosales, Derralynn Hughes, Rose Sheridan, Romuald Corbau, Amit Nathwani
Publikováno v:
Gene Therapy.
Fabry disease is an X-linked lysosomal storage disorder caused by loss of alpha-galactosidase A (α-Gal A) activity and is characterized by progressive accumulation of glycosphingolipids in multiple cells and tissues. FLT190, an investigational gene
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::3b4f60e3c8e78696b3ad39b88123464b
https://doi.org/10.1038/s41434-022-00381-y
https://doi.org/10.1038/s41434-022-00381-y
Autor:
Jey Jeyakumar, Sophie A. Snow, Olivia Allen, Sujata Ravi, Rose Sheridan, Jonathan H. Foley, Romuald Corbau, Erald Shehu
Publikováno v:
Molecular Genetics and Metabolism. 132:S90-S91
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::d1ea22ee8158a3d4c8ce7fb882a0a45c
https://doi.org/10.1016/j.ymgme.2020.12.217
https://doi.org/10.1016/j.ymgme.2020.12.217
Autor:
Amit C. Nathwani, Romuald Corbau, Petya Kalcheva, Emmaline Stotter, Erald Shehu, Jalpa Pandya, Fabrizio Comper, Clement Cocita, I-Mei Yu, Carlos Henrique Miranda, Natalie Northcott, Elisa Chisari, Rose Sheridan, Samantha Correia
Publikováno v:
Molecular Genetics and Metabolism. 132:S27-S28
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::d8a97aad6f24912b4ed56ffb4911de33
https://doi.org/10.1016/j.ymgme.2020.12.047
https://doi.org/10.1016/j.ymgme.2020.12.047
Autor:
Natalie Northcott, Romuald Corbau, Ying Sun, Fabrizio Comper, Erald Shehu, Amit C. Nathwani, Rose Sheridan, Clement Cocita, Emmaline Stotter, Jalpa Pandya, I-Mei Yu, Jaminder Khinder, Petya Kalcheva, Samantha Correia, Benjamin Liou, Carlos Henrique Miranda, Elisa Chisari
Publikováno v:
Molecular Genetics and Metabolism. 132:S28-S29
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::aae5d07ac5b3f9fbd5e17732b5e3d979
https://doi.org/10.1016/j.ymgme.2020.12.050
https://doi.org/10.1016/j.ymgme.2020.12.050
Autor:
Azadeh Kia, Jey Jeyakumar, Niten Patel, Gerard Short, Romuald Corbau, Derralynn Hughes, Rose Sheridan, Amit C. Nathwani, Leanne Dronfield, Russell Kinch
Publikováno v:
Molecular Genetics and Metabolism. 129:S77-S78
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::f964e563397b7fad1a593ea0d5ed30f5
https://doi.org/10.1016/j.ymgme.2019.11.188
https://doi.org/10.1016/j.ymgme.2019.11.188
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:
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:
Azadeh Kia, Romuald Corbau, Rose Sheridan, Paniz Hosseini, Jenny McIntosh, Doyoung Lee, Amit C. Nathwani, Jonathan H. Foley, Jey Jeyakumar, Carlos J. Miranda, Miriam Canavese
Publikováno v:
Molecular Genetics and Metabolism. 126:S100
Gaucher disease (GD) is characterised by the deposition of glucocerebroside in cells of the macrophage-monocyte system caused by impaired production of the enzyme beta-glucocerebrosidase (GCase). Over the past 20 years, enzymatic replacement therapy
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::e15de76fb57f715dc4ae423c43312cce
https://doi.org/10.1016/j.ymgme.2018.12.251
https://doi.org/10.1016/j.ymgme.2018.12.251
Autor:
Azadeh Kia, Amit C. Nathwani, Jenny McIntosh, Paniz Hosseini, Jey Jeyakumar, Daniël Verhoef, Romuald Corbau, Petya Kalcheva, Rose Sheridan
Publikováno v:
Molecular Genetics and Metabolism. 126:S80
Fabry disease is an X-linked lysosomal storage disorder (LSD) resulting from mutations in the gene encoding for α-galactosidase A (GLA). It is characterised by the abnormal accumulation of neutral glycosphingolipids (GSL), predominantly Globotriaosy
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::2077dea101cdbb8b0c449f0fe9ef92ef
https://doi.org/10.1016/j.ymgme.2018.12.196
https://doi.org/10.1016/j.ymgme.2018.12.196