Molecular Evidence of Genome Editing in a Mouse Model of Immunodeficiency

Volume: 8, Issue: 1
Published: May 29, 2018
Abstract
Genome editing is the introduction of directed modifications in the genome, a process boosted to therapeutic levels by designer nucleases. Building on the experience of ex vivo gene therapy for severe combined immunodeficiencies, it is likely that genome editing of haematopoietic stem/progenitor cells (HSPC) for correction of inherited blood diseases will be an early clinical application. We show molecular evidence of gene correction in a mouse...
Paper Details
Title
Molecular Evidence of Genome Editing in a Mouse Model of Immunodeficiency
Published Date
May 29, 2018
Volume
8
Issue
1
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