Mutant allele knockout with novel CRISPR nuclease promotes myelopoiesis in ELANE neutropenia

Severe congenital neutropenia (SCN) is a life-threatening marrow failure disorder, usually caused by heterozygous mutations in ELANE. Potential genetic treatment strategies include biallelic knockout or gene correction via homology-directed repair (HDR). Such strategies, however, involve the potenti...

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Detalles Bibliográficos
Autores principales: Sabo, Peter, Makaryan, Vahagn, Dicken, Yosef, Povodovski, Lital, Rockah, Liat, Bar, Tzlil, Gabay, Matan, Elinger, Dalia, Segal, Ella, Haimov, Ora, Antoshvili, Maya, Drori, Anat London, Poulsen, Tanoya, Herman, Asael, Emmanuel, Rafi, Dale, David C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9240714/
https://www.ncbi.nlm.nih.gov/pubmed/35795780
http://dx.doi.org/10.1016/j.omtm.2022.06.002

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