Exploiting epigenetics for the treatment of inborn errors of metabolism

Gene therapy is currently considered as the optimal treatment for inborn errors of metabolism (IEMs), as it aims to permanently compensate for the primary genetic defect. However, emerging gene editing approaches such as CRISPR‐Cas9, in which the DNA of the host organism is edited at a precise locat...

Ausführliche Beschreibung

Bibliographische Detailangaben

Veröffentlicht in:	Journal of inherited metabolic disease 2020-01, Vol.43 (1), p.63-70
Hauptverfasser:	Rutten, Martijn G. S., Rots, Marianne G., Oosterveer, Maaike H.
Format:	Artikel
Sprache:	eng
Schlagworte:	(epi)genome editing CRISPR DNA methylation Epigenetics gene correction Gene expression Gene therapy Genome editing histone modifications Inborn errors of metabolism inherited metabolic disease Metabolism Phenotypes Review therapy development
Online-Zugang:	Volltext
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!