University of California

Howard Hughes Medical Institute

Bum-Yeol Hwang

David V Schaffer

Loren Lee Looger

James T Koerber

Joshua Dudman

Alla Karpova

Dougal Gowanlock Robinson Tervo

Melissa A Kotterman

Adam Hantman

Kimberly Ritola

Sarada Viswanathan

The present disclosure provides AAV variants that exhibit a preference for retrograde movement in neurons and methods of using such variants.

Variant adeno-associated viruses and methods of using

The present disclosure provides infectious recombinant adeno-associated virus (rAAV) virions that comprise a variant capsid protein and a heterologous nucleic acid. The present disclosure further provides the variant adeno-associated virus (AAV) capsid proteins (and/or a nucleic acid encoding the variant AAV capsid proteins), which confer to an infectious rAAV virion an increased resistance to human AAV neutralizing antibodies. The present disclosure further provides host cells comprising an infectious rAAV virion and/or a nucleic acid encoding a subject variant AAV capsid protein. The present disclosure further provides methods of delivering a heterologous nucleic acid to a target cell where the target cell is contacted with a subject infectious rAAV virion. The present disclosure further provides methods of delivering a gene product to an individual, the methods generally involving administering an effective amount of a subject rAAV virion to an individual in need thereof.

Compositions and methods of treatment

Adeno-associated virus variants and methods of use thereof

Growing community of inventors

Moraga, CA, United States of America

Bum-Yeol Hwang