California Institute of Technology

City of Hope

Si-ping Han

William A Goddard, Iii

John J Rossi

Lisa Scherer

Robert D Barish

Hareem T Maune

Guido Marcucci

Ya-Huei Kuo

Paul W K Rothemund

Erik Winfree

Marc William Bockrath

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

The invention provides compositions and methods for signal activated RNA interference (saRNAi), preferably in vivo. The invention provides polynucleotides that switches between an inactive form and an active form upon covalent or non-covalent interaction with one or more specific chemical signals, such as disease-specific mRNA, miRNA, or other cellular RNA products with sequences that characterize diseased states of the cell. The interaction between the subject polynucleotides and the signals is preferably mediated by hybridization, which exposes, facilitates the formation, and/or allows the formation of a substrate that can be processed by proteins of the RNAi pathway (such as Dicer). The input and output of multiple different polynucleotides of the invention can form an in vivo signaling network. In addition, the multiple input signals can be integrated to modulate the activity of the subject polynucleotides.

Signal activated RNA interference

A linker polynucleotide for attaching a nanomaterial to a polynucleotidic platform and related nanoassemblies, arrangements, structures, methods and systems.

Polynucleotides and related nanoassemblies, structures, arrangements, 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.

Exonuclease resistant polynucleotide and related duplex polynucleotides, constructs, compositions, methods and systems

The disclosure relates to nanotube composite structures and related methods and systems. In particular, structures, methods and systems are provided herein to allow for precise, tunable separation between nanomaterials such as carbon nanotubes.

Growing community of inventors

Yorba Linda, CA, United States of America

Si-ping Han