Novel Lung Tropic Adeno-Associated Virus Capsids for Therapeutic Gene Delivery

Efforts to identify mutations that underlie inherited genetic diseases combined with strides in the development of gene therapy vectors over the last three decades have culminated in the approval of several adeno-associated virus (AAV)-based gene therapies. Genetic diseases that manifest in the lung...

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Veröffentlicht in:	Human gene therapy 2020-09, Vol.31 (17-18), p.996-1009
Hauptverfasser:	Carneiro, Ana, Lee, Hyuncheol, Lin, Li, van Haasteren, Joost, Schaffer, David V
Format:	Artikel
Sprache:	eng
Schlagworte:	Barriers Bioinformatics Biology Capsids Clinical trials Cystic fibrosis Expression vectors Gene therapy Gene transfer Genetic disorders Immunology Lungs Molecular biology Molecular modelling Mucus Mutation Viruses
Online-Zugang:	Volltext
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