FUNCTIONAL AAV CAPSIDS FOR SYSTEMIC ADMINISTRATION

Disclosed herein are engineered AAV VP capsid polypeptides with the ability to assemble into virus particles and having improved tissue tropism to, for example, CNS tissues or muscle tissues. The capsids are engineered using the high throughput discovery system described herein. In certain embodimen...

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Hauptverfasser: BRIGGS, Adrian, Wrangham, STEIN, Kevin, Christopher, JIANG, Yue, HAUSE, JR., Ronald, James, PACKARD, Thomas, VIGNEAULT, Francois, HUSS, David, Jeffrey
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creator BRIGGS, Adrian, Wrangham
STEIN, Kevin, Christopher
JIANG, Yue
HAUSE, JR., Ronald, James
PACKARD, Thomas
VIGNEAULT, Francois
HUSS, David, Jeffrey
description Disclosed herein are engineered AAV VP capsid polypeptides with the ability to assemble into virus particles and having improved tissue tropism to, for example, CNS tissues or muscle tissues. The capsids are engineered using the high throughput discovery system described herein. In certain embodiments, provided herein are recombinant adeno-associated virus (AAV) VP capsid polypeptides having at least one mutation in a 581-589 region of the VP capsid polypeptide, corresponding to residues 581 to residue 589 of a VP1 polypeptide of SEQ ID NO: 1.
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language eng ; fre ; ger
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subjects BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
ORGANIC CHEMISTRY
PEPTIDES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
SPIRITS
VINEGAR
WINE
title FUNCTIONAL AAV CAPSIDS FOR SYSTEMIC ADMINISTRATION
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