California Institute of Technology

City of Hope

The General Hospital Corporation

Clty of Hope

Lisa Scherer

John J Rossi

William A Goddard, Iii

Si-ping Han

Si-Ping Han

Marwa Ben Haj Salah

Guido Marcucci

Ya-Huei Kuo

Ane Miren Salvador Garicano

Julia Derogatis

Sahil Sagar

Robin Hu

Saumya Das

Disclosed herein are conditional siRNAs activatable by pro-hypertrophic RNA sequences and use thereof for treating conditions such as cardiac hypertrophy. The conditional siRNAs target calcineurin or HDAC2.

Conditional-siRNAS and uses thereof in treating cardiac hypertrophy

Disclosed herein are multi-way oligonucleotide junctions for delivering one or more cargo molecules to a biological target and method of making such junctions. The oligonucleotide junctions are formed by two or more oligonucleotides and are stable outside the cell and easily dissociate inside the cell to release the cargo molecule(s). One or more cargo molecules as well as delivery ligand can be loaded to the junctions for targeted delivery. Also disclosed are nanostructures including one or more junctions attached to each other for delivering two or more cargo molecules.

Meta-stable oligonucleotides junctions for delivery of therapeutics

Disclosed herein are programmable, conditionally activated small interfering RNA constructs (Cond-siRNAs) and methods of making and using the same as therapeutic agents. The Cond-siRNA comprises a sensor strand, a core strand, and a guide strand, which crossover to form a sensor duplex and a RNAi duplex attached to each other to form a single structure. Upon binding an input strand to the sensor strand, the Cond-siRNA is activated and releases RNAi targeting a desired gene.

Programmable conditional SIRNAS and uses thereof

Disclosed herein are conditional siRNAs activatable by CBF&#x3b2;-MYH11 oncogenic gene and use thereof for treating conditions such as acute myeloid leukemia (AML). The conditional siRNAs target MCL-1 or HDAC8.

Conditional-siRNAS and uses thereof in treating acute myeloid leukemia

Provided herein are signal activatable molecular constructs for delivery of molecules and related components, compositions, methods, and systems, having a 17 to 30 bp targeting domain duplex RNA, at least one protection strand having a protection segment and linker segment and a sensor strand having a displacement segment and a toehold segment, in which in an inactive conformation the protection segment and the displacement segment form a sensor domain duplex polynucleotide covalently attached to the targeting domain and presenting the toehold segment for binding to a signal molecule. In an active conformation the sensor strand is bound to the signal molecule and is detached from the at least one protection strand and from the targeting domain; and the targeting domain attaches the at least one protection strand in a configuration allowing processing by Dicer and/or an Argonaute enzyme.

Signal activatable constructs and related components compositions methods and systems

Provided herein are signal activatable molecular constructs for enzyme-assisted delivery of molecules and related components, such as a sensor domain, compositions, methods and systems.

Targeting domain and related signal activated molecular delivery

Provided herein are exonuclease resistant polynucleotides and related constructs, compositions, methods and systems.

Growing community of inventors

Monrovia, CA, United States of America

Lisa Scherer