The University of North Carolina at Chapel Hill

University of Florida Research Foundation, Incorporated

Duke University

University of Pittsburgh

Indiana University

Aravind Asokan

Richard Jude Samulski

Mavis Agbandje-McKenna

Brittney Gurda

Long Ping Victor Tse

Trevor Gonzalez

Giridhar Murlidharan

Erin Borchardt

Lawrence Patrick Havlik

Martin F Jarrold

Dwight D Koeberl

Shen Shen

Mario Mietzsch

Alexander Deiters

Mohamad Mikati

James B Hemphill

Ram Puranam

Boris Kantor

Arsen Hunanyan

Rita Meganck

William F Marzluff

Elizabeth Pierson

Blake Albright

James K Smith

David Keifer

Patrick Havlik

Nagesh Pulicherla

Zhijian Wu

The present disclosure provides AAV capsid proteins comprising a modification in the amino acid sequence and virus vectors comprising the modified AAV capsid protein. The disclosure also provides methods of administering the virus vectors and virus capsids of the disclosure to a cell or to a subject in vivo. This disclosure also relates to recombinant adeno associated virus (rAAV) capsid proteins comprising one or more amino acid substitutions in the 5-fold region of the rAAV capsid that result in decreased reactivity to neutralizing antibodies, and their use as gene delivery vehicles.

Methods and compositions for antibody-evading virus vectors

The present disclosure provides adeno-associated virus (AAV) vectors, comprising coevolved capsid variant proteins, pharmaceutical compositions, methods of making, and methods for delivering such to a subject.

Cross-species compatible adeno-associated virus compositions and methods of use thereof

The present invention provides methods and compositions comprising an adeno-associated virus (AAV) capsid protein, comprising one or more amino acids substitutions, wherein the substitutions introduce a new glycan binding site into the AAV capsid protein.

Methods and compositions for dual glycan binding AAV vectors

The present disclosure provides AAV capsid proteins comprising a modification in the amino acid sequence and virus capsids and virus vectors comprising the modified AAV capsid protein. The disclosure also provides methods of administering the virus vectors and virus capsids of the disclosure to a cell or to a subject in vivo.

Methods and compositions for gene transfer across the vasculature

The present invention provides AAV capsid proteins comprising a modification in the amino acid sequence and virus vectors comprising the modified AAV capsid protein. The invention also provides methods of administering the virus vectors and virus capsids of the invention to a cell or to a subject in vivo.

The present disclosure provides nucleic acid expression cassettes, vectors, compositions and methods for the treatment of ATPase-mediated diseases in a subject.

Compositions comprising nucleic acid molecules and methods of treating ATPase-mediated diseases

This invention is directed to AAV compositions for circular RNA expression and methods of expressing covalently closed, circular RNA.

Methods and compositions for circular RNA molecules

Disclosed herein is a genetically encoded light- or chemically-activated Cas9 engineered through the site-specific installation of an activatable lysine amino acid. Such activatable Cas9 proteins can be used in CRISPR/Cas9 systems to control gene expression temporally, spatially, or both. Systems, methods, kits, and compositions for manipulation of sequences and/or activities of target sequences are provided. Also provided are methods of directing CRISPR complex formation in cells and methods for selecting specific cells by introducing precise mutations utilizing the CRISPR/Cas9 system.

Activatable CRISPR/CAS9 for spatial and temporal control of genome editing

The present invention provides AAV capsid proteins comprising a modification in the amino acid sequence and virus capsids and virus vectors comprising the modified AAV capsid protein. The invention also provides methods of administering the virus vectors and virus capsids of the invention to a cell or to a subject in vivo.

Methods and compositions for targeted gene transfer

The present invention provides a method of identifying components present in a preparation of virus particles, comprising: a) analyzing the preparation of virus particles with single molecule mass spectrometry to obtain a mass histogram; and b) interpreting the mass histogram of (a) to identify different components present in the preparation.

Methods and compositions for resolving components of a virus preparation

Growing community of inventors

Chapel Hill, NC, United States of America

Aravind Asokan