Parameters affecting successful stem cell collections for genetic therapies in sickle cell disease

David G Justus; John P Manis

doi:10.1016/j.transci.2021.103059

Parameters affecting successful stem cell collections for genetic therapies in sickle cell disease

Transfus Apher Sci. 2021 Feb;60(1):103059. doi: 10.1016/j.transci.2021.103059. Epub 2021 Jan 10.

Authors

David G Justus¹, John P Manis²

Affiliations

¹ Department of Laboratory Medicine, Boston Children's Hospital, 300 Longwood Avenue, Boston, MA 02115, United States. Electronic address: david.justus@childrens.harvard.edu.
² Department of Laboratory Medicine, Boston Children's Hospital, 300 Longwood Avenue, Boston, MA 02115, United States. Electronic address: manis@enders.tch.harvard.edu.

PMID: 33541761
DOI: 10.1016/j.transci.2021.103059

Abstract

Emerging cellular therapies require the collection of peripheral blood hematopoietic stem cells (HSC) by apheresis for in vitro manipulation to accomplish gene addition or gene editing. These therapies require relatively large numbers of HSCs within a short time frame to generate an efficacious therapeutic product. This review focuses on the principal factors that affect collection outcomes, especially relevant to gene therapy for sickle cell disease.

Keywords: Gene editing; Gene therapy; Sickle cell disease; Stem cells.

Publication types

Review

MeSH terms

Anemia, Sickle Cell / pathology
Anemia, Sickle Cell / therapy*
Gene Editing / methods*
Genetic Therapy / methods*
Hematopoietic Stem Cell Transplantation / methods*
Hematopoietic Stem Cells / metabolism*
Humans
Transplantation Conditioning / methods*