Precise and error-prone CRISPR-directed gene editing activity in human CD34+ cells varies widely among patient samples.

ABSTRACT: Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and their associated CRISPR-associated nucleases (Cas) are among the most promising technologies for the treatment of hemoglobinopathies including Sickle Cell Disease (SCD). We are only beginning to identify the molecular variables that influence the specificity and the efficiency of CRISPR- directed gene editing, including the position of the cleavage site and the inherent variability among patient samples selected for CRISPR-directed gene editing. Here, we target the beta globin gene in human CD34+ cells to assess the impact of these two variables and find that both contribute to the global diversity of genetic outcomes. Our study demonstrates a unique genetic profile of indels that is generated based on where along the b

SUBMITTER: Modarai SR 

PROVIDER: S-EPMC7902267 | biostudies-literature | 2021 Feb

REPOSITORIES: biostudies-literature