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

• Main Authors: Kaitly J. Woodard (Author), Phillip A. Doerfler (Author), Kalin D. Mayberry (Author), Akshay Sharma (Author), Rachel Levine (Author), Jonathan Yen (Author), Virginia Valentine (Author), Lance E. Palmer (Author), Marc Valentine (Author), Mitchell J. Weiss (Author)
• Format: Book
• Published: The Company of Biologists, 2022-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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,...