City of Hope

University of Massachusetts

California Institute of Technology

General Electric Company

University of California

Applied Biosystems, LLC

National and Kapodistrian University of Athens

Life Technologies Corporation

Leica Microsystems Cms Gmbh

Jun Xie

David A Horne

Guangping Gao

Yu-Chong Tai

Sangkil Nam

Richard Jove

Terence Flotte

Phillip D Zamore

John C Williams

Li Zhu

Jason Shih

Wei-Cheng Tian

Erin Jean Finehout

Judith L Campbell

Binghui Shen

Li Zheng

Hongzhi Li

Kenneth Karanja

Kashan Ali Shaikh

Larry Eugene Overman

Krzysztof Bzymek

Shashi Thutupalli

Robert John Filkins

Alex David Corwin

Debjyoti Banerjee

Umberto Ulmanella

Cindy Zer

Yuelong Ma

Joshua Michael Donaldson

Kendra Nicole Avery

Terry D Lee

Konrad Faulstich

Heng Wei Chang

Qing He

Chi-Yuan Shih

Marcus Baumann

Claudia Kowolik

Wendong Huang

Po-Jui Chen

John E Shively

Damien Craig Rodger

Changlin Pang

Rongzhen Xu

Anthony John Murray

Matthieu Liger

Aldrich Lau

Nannan Chen

Angela L Perkins Harki

Darron K Young

Junfeng Li

Jung Min Kim

Zijie Sun

Dan Wang

Shubbir Ahmed

Christopher Michael Puleo

Aldrich N K Lau

Stacey Joy Kennerly

Christopher Lincoln

Oliver Charles Boomhower

Christopher James Sevinsky

Alexios Leandros Skaltsounis

Nicolas Gaboriaud-Kolar

Sachin Chaugule

Jae-Hyuck Shim

Yeon-Suk Yang

Binfeng Zhang

Senlin Xu

Marina Kritsanida

Hansong Zeng

Yunan Miao

John Williams

Krzysztof P Bzymek

David Horne

Plillip D Zamore

In some aspects the disclosure relates to recombinant adeno-associated virus (rAAV) vectors and rAAVs (e.g., viral particles) engineered to express a transgene comprising an inhibitory nucleic acid (e.g., an artificial miRNA, amiRNA) having a pri-miRNA scaffold and a guide strand that targets a human target gene.

Modified AAV constructs and uses thereof

In some aspects, the disclosure relates to methods for improving titer and yield of viral vector production. In some embodiments, the methods comprise transient silencing of transgene expression during packaging of a viral vector.

Viral vector production

In some aspects, the disclosure relates to compositions and methods for modulating (e.g., increasing and/or decreasing) bone mass in a subject. In some aspects, the disclosure provides isolated nucleic acids, and vectors such as rAAV vectors, configured to express transgenes that promote (e.g., increase) or inhibit (e.g., decrease) activity, differentiation, or function of certain types of bone cells, for example osteoblasts, osteoclasts, osteocytes, etc. In some embodiments, the isolated nucleic acids and vectors described by the disclosure are useful for treating disorders and conditions associated with increased bone mass (e.g., osteopetrosis) or decreased bone mass (e.g., osteoporosis).

Gene therapeutics for treating bone disorders

Provided herein are, inter alia, methods of treating cancer using compounds of the invention.

Berbamine derivatives and methods of use thereof

The present disclosure relates to the field of rAAV delivery of transgenes. In some aspects, the disclosure relates to RNAi. Provided herein are recombinant adeno-associated virus (rAAV) vectors comprising modified ITRs. In some embodiments, the modified ITRs comprise a sequence encoding a shRNA, miRNA, or AmiRNA.

Disclosed herein are compositions and methods for treating cancer, FLT3-AML, and CML.

5-Bromo-indirubins

Provided herein are functionalized monoclonal antibodies (mAbs) including antibody fragments, including those where a Fab-binding molecule (Fab binding moiety) linked to a steric hindering molecule (steric hindering chemical moiety) is mechanically interlocked (e.g., through noncovalent conjugation) with the antibody or antibody fragment. Also provided are compositions that form highly stable and versatile drug delivery and diagnostic compositions.

Mechanically interlocking complexes

The invention in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the invention relates to gene transfer methods using the recombinant adeno-associate viruses. In some aspects, the invention relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

AAV's and uses thereof

Disclosed herein, inter alia, are compositions and methods for inhibiting DNA2.

DNA2 inhibitors for cancer treatment

In some aspects, the disclosure relates to compositions and methods for treating fibrodysplasia ossificans progressiva (FOP) in a subject. In some aspects, the disclosure provides isolated nucleic acids, and vectors such as rAAV vectors, configured to express transgenes that inhibit (e.g., decrease) expression of mutated AVCR1 gene in muscle cells or connective tissues.

Gene therapeutics for fibrodysplasia ossificans progressiva

Provided herein are methods for treating metastatic prostate cancer using anti-androgen compounds and radionuclide-labeled androgens.

Methods of treating advanced prostate cancer

The disclosure in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the disclosure relates to gene transfer methods using the recombinant adeno-associated viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

Recombinant AAV variants and uses thereof

The invention in some aspects relates to methods and compositions for assessing the effectiveness of miRNA inhibitors. In other aspects of the invention, methods and compositions for treating cholesterol related disorders are provided. In one aspect of the invention, miRNA inhibitors against miR-122 and rAAV-based compositions comprising the same are provided.

AAV-based treatment of cholesterol-related disorders

Disclosed herein inter alia are compositions and methods for treating cancer using 5-Br-indirubin derivatives.

5-bromo-indirubins

Growing community of inventors

Niskayuna, NY, United States of America

Jun Xie