The Children's Hospital of Philadelphia

Mustafa N Yazicioglu

Katherine A High

Xavier Anguela

Federico Mingozzi

Fayaz R Khazi

Kirk Chu

Luca Monaldini

Samuel Murphy

Compositions and methods for AAV mediated gene therapy are disclosed. AAV vectors comprise protein capsid variants comprising therapeutically beneficial transgenes. AAV variants are provided which exhibit increased transduction efficiency when compared to AAV serotypes (e.g., AAV1, AAV2, AAV8, AAV-rh74), which lack the modifications disclosed herein. Such improved vectors are useful for transduction of a variety of tissues.

Composition and methods for highly efficient gene transfer using AAV capsid variants

The invention relates to adeno-associated virus (AAV) serotype AAV-Rh74 and related AAV vectors, and AAV-Rh74 and related AAV vector mediated gene transfer methods and uses. In particular, AAV-Rh74 and related AAV vectors target polynucleotides to cells, tissues or organs for expression (transcription) of genes encoding therapeutic proteins and peptides, and polynucleotides that function as or are transcribed into inhibitory nucleic acid sequences.

Variant AAV and compositions, methods and uses for gene transfer to cells, organs and tissues

Compositions and methods for detecting PRRG4- and PRRG2 associated molecules are provided as are methods of use thereof for the detection and treatment of cancer.

PRRG4-associated compositions and methods of use thereof in methods of tumor diagnosis

Growing community of inventors

Broomall, PA, United States of America

Mustafa N Yazicioglu