CRISPR to fix bad blood: a new tool in basic and clinical hematology.

ABSTRACT: Advances in genome engineering in the last decade, particularly in the development of programmable nucleases, have made it possible to edit the genomes of most cell types precisely and efficiently. Chief among these advances, the clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system is a novel, versatile and easy-to-use tool to edit genomes irrespective of their complexity, with multiple and broad applications in biomedicine. In this review, we focus on the use of CRISPR/Cas9 genome editing in the context of hematologic diseases and appraise the major achievements and challenges in this rapidly moving field to gain a clearer perspective on the potential of this technology to move from the laboratory to the clinic. Accordingly, we discuss data from studies editing h

SUBMITTER: Gonzalez-Romero E 

PROVIDER: S-EPMC6518885 | biostudies-literature | 2019 May

REPOSITORIES: biostudies-literature