Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations

We characterized the human β-like globin transgenes in two mouse models of sickle cell disease (SCD) and tested a genome-editing strategy to induce red blood cell fetal hemoglobin (HbF; α2γ2). Berkeley SCD mice contain four to 22 randomly arranged, fragmented copies of three human transgenes (HBA1,...

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Autori principali:	Kaitly J. Woodard, Phillip A. Doerfler, Kalin D. Mayberry, Akshay Sharma, Rachel Levine, Jonathan Yen, Virginia Valentine, Lance E. Palmer, Marc Valentine, Mitchell J. Weiss
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	The Company of Biologists 2022-06-01
Serie:	Disease Models & Mechanisms
Soggetti:	fetal hemoglobin genome editing sickle cell disease
Accesso online:	http://dmm.biologists.org/content/15/6/dmm049463
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