Base Editing of Human Hematopoietic Stem Cells

Jing Zeng; Gabriele Casirati; My Anh Nguyen; Pietro Genovese; Daniel E Bauer

doi:10.1007/978-1-0716-2879-9_5

Base Editing of Human Hematopoietic Stem Cells

Methods Mol Biol. 2023:2606:43-62. doi: 10.1007/978-1-0716-2879-9_5.

Authors

Jing Zeng^{1

2

3}, Gabriele Casirati^{1

2

3}, My Anh Nguyen^{1

2

3}, Pietro Genovese^{1

2

3}, Daniel E Bauer^{4

5

6}

Affiliations

¹ Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA, USA.
² Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA, USA.
³ Department of Pediatrics, Harvard Stem Cell Institute, Broad Institute, Harvard Medical School, Boston, MA, USA.
⁴ Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA, USA. [email protected].
⁵ Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA, USA. [email protected].
⁶ Department of Pediatrics, Harvard Stem Cell Institute, Broad Institute, Harvard Medical School, Boston, MA, USA. [email protected].

Abstract

Base editing by nucleotide deaminases linked to programmable DNA-binding proteins represents a promising approach to remedy blood disorders. Here we describe the ex vivo base editing of human CD34+ hematopoietic stem and progenitor cells (HSPCs) by electroporation of base editor mRNA or protein.

Keywords: Base editing; CD34+ hematopoietic stem and progenitor cells; Electroporation.

Publication types

Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

MeSH terms

Antigens, CD34 / metabolism
Gene Editing*
Hematopoietic Stem Cell Transplantation*
Hematopoietic Stem Cells / metabolism
Humans

Substances

Antigens, CD34

Abstract

Publication types

MeSH terms

Substances

Grants and funding