University of Massachusetts

Terence Flotte

Guangping Gao

Jun Xie

Christian Mueller

Phillip D Zamore

Allison Keeler-Klunk

Qiushi Tang

Qin Su

Phillip David Zamore

The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency).

RAAV-based compositions and methods

In some aspects, the disclosure relates to compositions and methods for production of recombinant adeno-associated virus (rAAV) particles. The disclosure is based, in part, on isolated nucleic acids and systems that include only two vectors and are suitable for production of multiple different single gene viral vectors in a multiple small-scale campaign mode (e.g., 10to 10viral particles.)

Campaign-ready series of recombinant adeno-associated virus (rAAV) complementing plasmids

The invention in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the invention relates to gene transfer methods using the recombinant adeno-associate viruses. In some aspects, the invention relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

AAV's and uses thereof

The disclosure in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the disclosure relates to gene transfer methods using the recombinant adeno-associated viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

Recombinant AAV variants and uses thereof

rAAV-based compositions and methods

RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies

Growing community of inventors

Holden, MA, United States of America

Terence Flotte