Preprint Review Version 1 Preserved in Portico This version is not peer-reviewed

Correction of the Sickle Cell Mutation Through Base and Prime Editing in Hematopoietic Stem Cells

Version 1 : Received: 18 September 2020 / Approved: 21 September 2020 / Online: 21 September 2020 (04:23:21 CEST)

How to cite: Ji, K. Correction of the Sickle Cell Mutation Through Base and Prime Editing in Hematopoietic Stem Cells. Preprints 2020, 2020090490 (doi: 10.20944/preprints202009.0490.v1). Ji, K. Correction of the Sickle Cell Mutation Through Base and Prime Editing in Hematopoietic Stem Cells. Preprints 2020, 2020090490 (doi: 10.20944/preprints202009.0490.v1).

Abstract

Sickle cell disease is characterized by stiff, “sickled” red blood cells that have difficulty moving through the bloodstream and do not efficiently carry oxygen. It is an inherited disease with severely limited treatment options, and is caused by a point mutation. Its prevalence in black and brown communities makes the already limited treatment options even less accessible. Base editing and prime editing are two relatively recent discoveries in the field of genome editing and were developed after the groundbreaking discovery of the CRISPR Cas9 system. While not fully tested, they hold a lot of promise in providing alternative treatment options for sickle cell disease. Both editing systems are able to install individual point mutations in the beta globin gene, which is where the sickle cell mutation occurs, and can thus cure sickle cell disease (in theory). In this paper we outline the mechanisms of CRISPR-Cas9 systems and base and prime editing, and provide insight into how to apply them to treat SCD. Further investigation should be done on specific editing systems and designs to use to ensure optimal treatment of SCD.

Subject Areas

base editing; prime editing; ABE; SCD; sickle cell disease; sickle cell anemia; CRISPR; Cas9

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