Genome editing of HBG1 and HBG2 to induce fetal hemoglobin

Induction of fetal hemoglobin (HbF) via clustered regularly interspaced short palindromic repeats/Cas9–mediated disruption of DNA regulatory elements that repress γ-globin gene (HBG1 and HBG2) expression is a promising therapeutic strategy for sickle cell disease (SCD) and β-thalassemia, although th...

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Detalhes bibliográficos
Publicado no:Blood Adv
Main Authors: Métais, Jean-Yves, Doerfler, Phillip A., Mayuranathan, Thiyagaraj, Bauer, Daniel E., Fowler, Stephanie C., Hsieh, Matthew M., Katta, Varun, Keriwala, Sagar, Lazzarotto, Cicera R., Luk, Kevin, Neel, Michael D., Perry, S. Scott, Peters, Samuel T., Porter, Shaina N., Ryu, Byoung Y., Sharma, Akshay, Shea, Devlin, Tisdale, John F., Uchida, Naoya, Wolfe, Scot A., Woodard, Kaitly J., Wu, Yuxuan, Yao, Yu, Zeng, Jing, Pruett-Miller, Shondra, Tsai, Shengdar Q., Weiss, Mitchell J.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2019
Assuntos:
Gene Therapy
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6855127/
https://ncbi.nlm.nih.gov/pubmed/31698466
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2019000820
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