University of California

Courtney S Young

April D Pyle

Melissa J Spencer

Huan Meng

Xiangsheng Liu

Ying Ji

Michael Reza Emami

In various embodiments a polyrotaxane carrier for in vivo delivery of a nucleic acid is provided. In certain embodiments the carrier comprises: a multi-arm polyethylene glycol (PEG) backbone comprising at least three arms; at least one cyclic compound having a cavity, where an arm of said multi-arm PEG backbone is threaded into the cavity of said cyclic compound forming an inclusion complex; a bulky moiety capping the terminal of the arm(s) threaded into said cyclic compound where said moiety inhibits dethreading of the cyclodextrin from the arm(s) of said backbone; and where at least one arm of said PEG backbone is free of cyclic compounds; and where said carrier has a net positive charge.

Multi-armed polyrotaxane platform for protected nucleic acid delivery

The present disclosure provides a method of modifying a mutant dystrophin gene in the genome of a cell. The present disclosure further provides compositions and kits for modifying a mutant dystrophin gene in the genome of a cell.

Methods and compositions for modifying a mutant dystrophin gene in a cell's genome

Growing community of inventors

Los Angeles, CA, United States of America

Courtney S Young