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Akademický článek
Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy
Autor: Christian Brendel, Olivier Negre, Michael Rothe, Swaroopa Guda, Geoff Parsons, Chad Harris, Meaghan McGuinness, Daniela Abriss, Alla Tsytsykova, Denise Klatt, Martin Bentler, Danilo Pellin, Lauryn Christiansen, Axel Schambach, John Manis, Helene Trebeden-Negre, Melissa Bonner, Erica Esrick, Gabor Veres, Myriam Armant, David A. Williams
Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 589-600 (2020)
In this work we provide preclinical data to support initiation of a first-in-human trial for sickle cell disease (SCD) using an approach that relies on reversal of the developmental fetal-to-adult hemoglobin switch. Erythroid-specific knockdown of BC
Externí odkaz: https://doaj.org/article/7ebf28bc3d664f328d3cea76508fe211
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2
Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy
Autor: Olivier Negre, Swaroopa Guda, Christian Brendel, Chad E. Harris, Martin Bentler, Myriam Armant, Melissa Bonner, Erica B. Esrick, John P. Manis, Helene Trebeden-Negre, Axel Schambach, Alla V. Tsytsykova, Danilo Pellin, Michael Rothe, Lauryn Christiansen, Denise Klatt, David A. Williams, Meaghan McGuinness, Daniela Abriss, Geoff Parsons, Gabor Istvan Veres
Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 17, Iss, Pp 589-600 (2020)
Molecular Therapy. Methods & Clinical Development
In this work we provide preclinical data to support initiation of a first-in-human trial for sickle cell disease (SCD) using an approach that relies on reversal of the developmental fetal-to-adult hemoglobin switch. Erythroid-specific knockdown of BC
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::fb8fc0356964dd1fbe99f38977e8a66a
http://www.sciencedirect.com/science/article/pii/S2329050120300450
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Plný text Recenzováno Digitální knihovna AV ČR
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