Base Editing in Progeria

The Hutchinson–Gilford progeria syndrome is an autosomal dominant disease caused by inheritance of a single point mutation in LMNA , which encodes laminin. Recent experiments have shown that life span in a mouse model of the disease was augmented through DNA base editing.

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Bibliographische Detailangaben
Veröffentlicht in:	The New England journal of medicine 2021-04, Vol.384 (14), p.1364-1366
Hauptverfasser:	Kim, Jin-Soo, Eriksson, Maria
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals CRISPR Deoxyribonucleic acid Disease Disease Models, Animal DNA DNA - metabolism Enzymes Fibroblasts Gene Editing - methods Gene expression Humans Investigations Lamin Type A - genetics Life span Liver Longevity Mice Mice, Transgenic Mutation Point Mutation Progeria Progeria - genetics Progeria - therapy Proteins Rodents
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Beschreibung
Zusammenfassung:	The Hutchinson–Gilford progeria syndrome is an autosomal dominant disease caused by inheritance of a single point mutation in LMNA , which encodes laminin. Recent experiments have shown that life span in a mouse model of the disease was augmented through DNA base editing.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMcibr2100661