Gene Therapy for Hemophilia and Duchenne Muscular Dystrophy in China

Gene therapy is a new technology that provides potential for curing monogenic diseases caused by mutations in a single gene. Hemophilia and Duchenne muscular dystrophy (DMD) are ideal target diseases of gene therapy. Important advances have been made in clinical trials, including studies of adeno-as...

Ausführliche Beschreibung

Gespeichert in:

Bibliographische Detailangaben
Veröffentlicht in:	Human gene therapy 2018-02, Vol.29 (2), p.146-150
Hauptverfasser:	Liu, Xionghao, Liu, Mujun, Wu, Lingqian, Liang, Desheng
Format:	Artikel
Sprache:	eng
Schlagworte:	Antisense oligonucleotides Clinical trials Dependovirus - genetics Duchenne's muscular dystrophy Dystrophy Gene Editing - trends Gene therapy Genetic Therapy - trends Genetic Vectors - therapeutic use Genomes Hemophilia Hemophilia A - genetics Hemophilia A - therapy Humans Medical research Muscular dystrophy Muscular Dystrophy, Duchenne - genetics Muscular Dystrophy, Duchenne - therapy Mutation New technology Nuclease Oligonucleotides Oligonucleotides, Antisense - drug effects Oligonucleotides, Antisense - genetics Oligonucleotides, Antisense - therapeutic use Viruses
Online-Zugang:	Volltext
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!

Schreiben Sie den ersten Kommentar!