University of Florida Research Foundation, Incorporated

Chen Ling

Arun Srivastava

Sergei Zolotukhin

Mavis Agbandje-McKenna

George Vladimirovich Aslanidi

Kim M Van Vliet

Li Zhong

Yuan Lu

The present invention provides novel mRNA elements capable of forming hairpin, double-stranded RNA structures independent of other non-coding RNAs. These mRNAs are stably expressed, lack polyadenylation tails, and allow minimal protein translation except when in the presence of specific proteins. Also provided, are compositions and kits comprising the mRNA element, as well as methods for its use in the regulation of protein translation. Advantageously, the disclosed elements represent a novel tool useful in regulating the expression of a wide variety of proteins of interest.

Hairpin MRNA elements and methods for the regulation of protein translation

Disclosed are next-generation multi-mutated capsid protein-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations that include them. Also disclosed are methods of preparing and using these high transduction efficiency vector constructs in a variety of therapeutic applications including, inter alia, as delivery agents for the treatment or amelioration of one or more diseases or abnormal conditions in an affected mammal using in vivo and/or ex situ viral vector-based gene therapy protocols. Also disclosed are large-scale production methods for the multi-mutated, capsid-modified rAAV expression vectors, viral particles, and infectious virions, as well as use of the disclosed compositions in the manufacture of medicaments for use in a variety of in vitro and/or in vivo therapeutic methodologies.

Capsid-modified, RAAV3 vector compositions and uses in gene therapy of human liver cancer

Provided herein are recombinant adeno-associated virus (rAAV) nucleic acid vectors that comprise one or more modifications within at least one inverted terminal repeat (ITR) region. Exemplary modifications include ITR sequences comprising a glucocorticoid responsive element and/or a transcription factor binding site. Also provided are plasmids, libraries, rAAV particles, compositions, kits, and methods related to such vectors.

Genome-modified recombinant adeno-associated virus vectors

Hairpin mRNA elements and methods for the regulation of protein translation

Growing community of inventors

Gainesville, FL, United States of America

Chen Ling