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- Title
Genome Editing: A Tool For Research and Therapy: Targeted genome editing hits the clinic.
- Authors
Lombardo, Angelo; Naldini, Luigi
- Abstract
The article discusses ways to apply targeted genome-editing technologies in engineering disease-associated somatic variation into human disease models and cell lines. It mentions studies on clinical trials of targeted genome editing, such as the HIV trial with autologous T cells in which a gene encoding an HIV co-receptor was disrupted. It also outlines two studies on targeted genome editing in human hematopoietic stem cells (HSCs) in experimental models.
- Subjects
GENOME editing; ANIMAL disease models; CELL lines; CLINICAL trials; HIV infections; T cells; HEMATOPOIETIC stem cells
- Publication
Nature Medicine, 2014, Vol 20, Issue 10, p1101
- ISSN
1078-8956
- Publication type
Article
- DOI
10.1038/nm.3721