Identification of novel HPFH-like mutations by CRISPR base editing that elevate the expression of fetal hemoglobin

Naturally occurring point mutations in the HBG promoter switch hemoglobin synthesis from defective adult beta-globin to fetal gamma-globin in sickle cell patients with hereditary persistence of fetal hemoglobin (HPFH) and ameliorate the clinical severity. Inspired by this natural phenomenon, we tile...

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Detalles Bibliográficos
Autores principales: Ravi, Nithin Sam, Wienert, Beeke, Wyman, Stacia K, Bell, Henry William, George, Anila, Mahalingam, Gokulnath, Vu, Jonathan T, Prasad, Kirti, Bandlamudi, Bhanu Prasad, Devaraju, Nivedhitha, Rajendiran, Vignesh, Syedbasha, Nazar, Pai, Aswin Anand, Nakamura, Yukio, Kurita, Ryo, Narayanasamy, Muthuraman, Balasubramanian, Poonkuzhali, Thangavel, Saravanabhavan, Marepally, Srujan, Velayudhan, Shaji R, Srivastava, Alok, DeWitt, Mark A, Crossley, Merlin, Corn, Jacob E, Mohankumar, Kumarasamypet M
Formato: Online Artículo Texto
Lenguaje:English
Publicado: eLife Sciences Publications, Ltd 2022
Materias:
Chromosomes and Gene Expression
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8865852/
https://www.ncbi.nlm.nih.gov/pubmed/35147495
http://dx.doi.org/10.7554/eLife.65421

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8865852/
https://www.ncbi.nlm.nih.gov/pubmed/35147495
http://dx.doi.org/10.7554/eLife.65421

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