ADENO-ASSOCIATED VARIANTS, FORMULATIONS AND METHODS FOR PULMONARY DELIVERY

The present disclosure provides a variant AAV capsid protein that confers tropism to lung cells and recombinant adeno-associated viruses comprising the variant AAV and pharmaceutical compositions comprising same and their use in the delivery of heterologous nucleic acids to lung cells for the treatm...

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Hauptverfasser:	GONZALES, Johnny, CROZE, Roxanne, KOTTERMAN, Melissa, SCHMITT, Christopher, FRANCIS, Peter, CALTON, Melissa
Format:	Patent
Sprache:	eng ; fre
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	The present disclosure provides a variant AAV capsid protein that confers tropism to lung cells and recombinant adeno-associated viruses comprising the variant AAV and pharmaceutical compositions comprising same and their use in the delivery of heterologous nucleic acids to lung cells for the treatment of pulmonary disorders. La présente invention concerne un variant de protéine de capside de VAA qui confère un tropisme à des cellules pulmonaires et des virus adéno-associés de recombinaison comprenant le variant de VAA ainsi que des compositions pharmaceutiques les comprenant et leur utilisation dans l'administration d'acides nucléiques hétérologues à des cellules pulmonaires pour le traitement de troubles pulmonaires.