Therapeutic gene editing in haematological disorders with  CRISPR /Cas9 | Zendy

Jensen Trine I. | Zendy; Axelgaard Esben | Zendy; Bak Rasmus O. | Zendy

AI Assistant Blog Pricing

Home ZAIA Blog

Premium

Therapeutic gene editing in haematological disorders with CRISPR /Cas9

Author(s) -

Jensen Trine I.,

Axelgaard Esben,

Bak Rasmus O.

Publication year - 2019

Publication title -

british journal of haematology

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 1.907

H-Index - 186

eISSN - 1365-2141

pISSN - 0007-1048

DOI - 10.1111/bjh.15851

Subject(s) - crispr , genome editing , cas9 , chimeric antigen receptor , computational biology , gene , biology , genetic enhancement , bioinformatics , genetics , immunotherapy , cancer

Summary The Clustered Regularly Interspaced Short Palindromic Repeats ( CRISPR )/ CRISPR ‐associated (Cas)9 platform offers an efficient way of making precise genetic changes to the human genome. This can be employed for disruption, addition and correction of genes, thereby enabling a new class of genetic therapies that can be applied to haematological disorders. Here we review recent technological advances in the CRISPR /Cas9 methodology and applications in haematology for curing monogenic genetic disorders and for engineering novel chimeric antigen receptor ( CAR ) T cells to treat haematological malignancies. Furthermore, we discuss current challenges for full clinical implementation of CRISPR /Cas9, and reflect on future trajectories of the technology.

This content is not available in your region!

Continue researching here.

Having issues? You can contact us here

Accelerating Research