Bringing gene therapy to where it's needed

Stefan Radtke; Hans-Peter Kiem

doi:10.1016/j.molmed.2022.01.005

Bringing gene therapy to where it's needed

Trends Mol Med. 2022 Mar;28(3):171-172. doi: 10.1016/j.molmed.2022.01.005. Epub 2022 Jan 24.

Authors

Stefan Radtke¹, Hans-Peter Kiem²

Affiliations

¹ Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
² Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA; Department of Medicine, University of Washington School of Medicine, Seattle, WA 98195, USA; Department of Pathology, University of Washington School of Medicine, Seattle, WA 98195, USA. Electronic address: hkiem@fredhutch.org.

PMID: 35086771
DOI: 10.1016/j.molmed.2022.01.005

Abstract

Gene editing allows the precise modification of cells to correct genetic defects or enhance immunotherapies. A limitation is the delivery of this technology to specific cells or organs. Recently, Banskota et al. reported the use of virus-like particles (VLPs) loaded with gene-editing agents for gene therapy delivery directly inside the body.

Keywords: base editors; gene therapy; in vivo delivery; target specificity.

Publication types

Comment

MeSH terms

CRISPR-Cas Systems*
Gene Editing*
Genetic Therapy
Humans