The University of North Carolina at Chapel Hill

North Carolina State University

Texas Tech University System

Matthew Louis Hirsch

Richard Jude Samulski

Brian Christopher Gilger

R Bryan Sutton

This invention relates to a truncated dysferlin nucleic acid and protein, vectors (e.g., adeno-associated virus vectors) comprising the nucleic acid and methods of using the same for delivery of dysferlin to a cell or a subject and treating dysferlinopathy.

Truncated dysferlin for treatment of dysferlinopathy

This invention relates to viral vectors for delivery of alpha-L-iduronidase to the cornea of a subject and methods of using the same for treatment and prevention of corneal clouding and blindness in a subject due to mucopolysaccharidosis I.

AAV-IDUA vector for treatment of MPS I-associated blindness

This invention relates to vectors for delivery of human leukocyte antigen G to the eye and/or to cornea explants and methods of using the same for treatment and/or prevention of corneal transplant rejection and other disorders associated with an immune response and/or vascularization.

Vector-mediated immune tolerance in the eye

This invention relates to synthetic adeno-associated virus (AAV) inverted terminal repeats (ITRs) that exhibit altered activities compared to a naturally occurring AAV ITR and methods of using the same for delivery of nucleic acids to a cell or a subject. The synthetic ITRs provide a larger packaging capacity and the ability to manipulate activities such as transduction efficiency, cellular response to transduction, and transcription.

Growing community of inventors

Chapel Hill, NC, United States of America

Matthew Louis Hirsch