University of California

The Scripps Research Institute

Ethan Bier

Annabel Guichard

Shaila Srinivasan

Kweon Yu

Robert M Krug

Victor Nizet

Michael Oldstone

Stephen Hedrick

John R Teijaro

Meghana Malur

Valentino Gantz

Margery Smelkinson

Described herein are methods and compositions for autocatalytic genome editing and neutralizing autocatalytic genome editing. The autocatalytic genome editing may be based on genomic integration of a construct containing multiple elements or on a trans-complementation approach, in which genetic elements can be propagated separately. The disclosure provides a method for autocatalytic genome editing based on the CRISPR/CAS9 system, and methods of use thereof, in animals, humans, and plants for eliminating pathogens, targeting suppression of crop pests, strategies to combat virus (e.g., HIV) and other diseases (e.g., cancer) caused by retrovirus, as well as to generate homozygous mutations that are transmitted to nearly all offspring.

Methods for autocatalytic genome editing and neutralizing autocatalytic genome editing and compositions thereof

Methods and pharmaceutical compositions for treating diseases characterized by compromised epithelial or endothelial barrier integrity are provided. The pharmaceutical compositions include an effective amount a Epac/Rap1 signaling inhibitor. The methods include administering to a patient suffering from a disease characterized by compromised epithelial or endothelial barrier integrity a therapeutically effective amount of a Epac/Rap1 signaling inhibitor, such as ESI-09, SecinH3, Slit, or AG1024.

Pharmacological restoration of epithelial or endothelial barrier integrity by agents blocking EPAC/RAP1 signaling

Method and composition for protection and treatment against influenza viral infection by modulating the activity of at least one essential host signaling pathways, including the Hedgehog (Hh), the Bone Morphogenetic Protein (BMP), the Notch (N) signaling pathways or combinations thereof, which activity is altered by the viral protein NS1. The method and composition comprise use of a compound that modulates at least one of these essential host signaling pathways for preventing or controlling symptoms associated with influenza viral infection. The method or composition can also be used either alone or in combination with other therapeutic agents for treating or protecting against influenza infection, and associated syndromes.

Protection and treatment against influenza infection

Methods for generating and using novel overexpression activity alleles of a gene in any organism, especially, are provided. Such alleles may be utilized in screening assays, and used to generate dominant-negative forms of bacterial toxins.

Method for generating overexpression of alleles in genes of unknown function

A potent peptide inhibitor (SuperSog) of TGF-&#x3b2; family growth factor signaling, peptide variants thereof and nucleotide coding sequences therefore are provided by the invention. The Super-Sog peptide comprises a fragment of theshort gastrulation (Sog) gene which includes the CR-1 cysteine-rich repeat of Sog. Methods and compositions for use of Super-Sog in therapeutic and diagnostic applications are also provided.

Peptide inhibitor of TGF-&#x3b2; growth factors

A potent peptide inhibitor (SuperSog) of TGF-&bgr; family growth factor signalling, peptide variants thereof and nucleotide coding sequences therefor are provided by the invention. The Super-Sog peptide comprises a fragment of the Drosophilia short gastrulation (Sog) gene which includes the CR-1 cysteine-rich repeat of Sog. Methods and compositions for use of Super-Sog in therapeutic and diagnostic applications are also provided.

Growing community of inventors

San Diego, CA, United States of America

Ethan Bier