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Completed Grants and Sponsored Research

Project Date: July 1, 2015 - June 30, 2016
Sponsoring Organization: COOLEY'S ANEMIA FOUNDATION, INC

Genome Editing of Beta-Globin Gene Cluster Repressive Elements for Beta-Thalassemia

Description of Major Goals
Specific Aim 1. Utilize a custom pooled CRISPR guide RNA library to perform high-resolution mutagenesis to determine minimal non-coding sequences at the B-globin gene cluster necessary for HbF repression. Specific Aim 2. Determine efficiency and specificity of genome editing human HSOCs by measuring rate of on-and off-target editing and impact on HbF level and henatopolesis.