首页|Advances in the Application of CRISPR/Cas9 System in the Study of Hematologic Diseases and Cancer
Advances in the Application of CRISPR/Cas9 System in the Study of Hematologic Diseases and Cancer
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NETL
Clustered regularly interspaced short palindromic repeats (CRISPR) / CRISPR-associated nuclease 9 (Cas9), as a powerful gene editing tool, makes remarkable development in genomics. Because it has several advantages such as easy-to-operate, accurate targeting and efficient editing, CRISPR/Cas9 has been widely studied in fundamental biomedicine and preclinical experiments. CRISPR/Cas9 system, designed from a prokaryotic CRISPR immune system, uses single-guided RNA (sgRNA)/Cas9 nuclease complex to bind target DNA for gene editing. Cas9 proteins cause double strand DNA breaks (DSBs), and DSBs are repaired by non-homologous end joining (NHEJ) and homology directed repair (HDR). Currently, it has showed some success in treating human diseases such as monogenic diseases. Herein, we review mechanisms of CRISPR/Cas9 system and the findings of CRISPR/Cas9 system for the therapy of hematologic diseases (including sickle cell disease, leukemia as well as anemia) and cancer, in order to provide some references and ideas for these two diseases both in basic research and clinical therapy as far as possible.
NETL
