Zobrazeno 1 - 2
of 2
pro vyhledávání: '"Anne Ramezi"'
Autor:
Samuel Lessard, Pauline Rimmelé, Hui Ling, Kevin Moran, Benjamin Vieira, Yi-Dong Lin, Gaurav Manohar Rajani, Vu Hong, Andreas Reik, Richard Boismenu, Ben Hsu, Michael Chen, Bettina M. Cockroft, Naoya Uchida, John Tisdale, Asif Alavi, Lakshmanan Krishnamurti, Mehrdad Abedi, Isobelle Galeon, David Reiner, Lin Wang, Anne Ramezi, Pablo Rendo, Mark C. Walters, Dana Levasseur, Robert Peters, Timothy Harris, Alexandra Hicks
Publikováno v:
Scientific Reports, Vol 14, Iss 1, Pp 1-18 (2024)
Abstract BIVV003 is a gene-edited autologous cell therapy in clinical development for the potential treatment of sickle cell disease (SCD). Hematopoietic stem cells (HSC) are genetically modified with mRNA encoding zinc finger nucleases (ZFN) that ta
Externí odkaz:
https://doaj.org/article/eeb0c50036ba4eed80ba42d7f6cae1af
Autor:
Asif Alavi, Lakshmanan Krishnamurti, Mehrdad Abedi, Isobelle Galeon, David Reiner, Sharon E Smith, Lin Wang, Anne Ramezi, Pablo Rendo, Mark C. Walters
Publikováno v:
Blood. 138:2930-2930
Introduction Sickle cell disease (SCD) is caused by pathologic variants in both alleles of the β-globin gene, affecting ~100,000 patients in the US (Strouse. Handb Clin Neurol 2016;138:311-24). Elevated fetal hemoglobin (HbF) levels ameliorate sympt