Children's Memorial Hospital

Ann & Robert Lurie Children's Hospital of Chicago

Martha C Bohn

Lixin Jiang

Elio F Vanin

Neva C West

Mohan K Sapru

The present invention is directed to small interfering RNAs that down regulate expression of a synuclein gene and methods of using the small interfering RNAs.

siRNA-mediated gene silencing of synuclein

A vector and a method are provided for delivering a nucleic acid to a nervous system cell. The vector includes a first nucleic acid, a second nucleic acid, inverted terminal repeats of adeno-associated virus, and a tetracycline-off regulatable promoter system that includes a first promoter operably linked to the first nucleic acid and a second promoter operably linked to the second nucleic acid. The promoters drive expression in opposite directions and away from the inverted terminal repeats. The method includes providing a recombinant adeno-associated viral (rAAV) vector and administering the vector to a nervous system cell. Expression of a product from the first nucleic acid is regulatable by the promoter system.

Growing community of inventors

Chicago, IL, United States of America

Martha C Bohn