California Institute of Technology

University of California

Massachusetts Institute of Technology

Leland Stanford Junior University

The General Hospital Corporation

Whitehead Institute for Biomedical Research

David Baltimore

Carlos Lois-Caballe

Lili Yang

Xiao-Feng Qin

Pin Wang

Irvin S Y Chen

Dong Sung An

Michael Thomas Bethune

Konstantin Taganov

Ryan Michael O'Connell

Mark Boldin

Kenan Christopher Garcia

Juan Luis Mendoza

Alejandro Benjamin Balazs

Michael Edward Birnbaum

Elizabeth J Hong

Shirley Pease

Irvin Sy Chen

Antoni Ribas

Marie B Chow

James Economou

Howard Y Chang

Xiaolu Yang

Matthew Porteus

Parameswaran Ramakrishnan

Alan J Fischman

Robert H Rubin

Irvin S Chen

Jonathan Tsai

Devdoot Majumdar

Roya Khosravi-Far

Marjorie A Oettinger

David G Schatz

Vincent R Racaniello

Jocelyn T Kim

Guideng Li

Stephanie Wong

Kenneth Yu

Alok V Joglekar

Michael T Leonard

Marvin H Gee

Anirudh Mathukumilli

Lilli Yang

Aadel Chaudhuri

Alex Steven So

Xin Luo

Luk Van Parijs

Joel L Pomerantz

Irvin SY Chen

Jonathan Michael Tsai

Described herein are compositions and methods for signaling and antigen-presenting bifunctional receptors (SABRs) comprising one or more antigen presenting domains; and one or more signal transduction domains, wherein the one or more antigen presenting domains comprise a binding fragment of a major histocompatibility complex (MHC) molecule. Various immunological functions of the SABRs are also described.

Signaling and antigen-presenting bifunctional receptors (SABR)

Disclosed herein are trogocytosis based TCR ligand discovery platforms and methods of using the same, trogocytosis based TCR discovery platforms and methods of using the same, and trogocytosis based ligand discovery platforms and methods of using the same. Also disclosed herein are isolated cells, nucleotides, sequences, and sequence databases produced using trogocytosis based discovery platforms.

Trogocytosis mediated epitope discovery methods

Compositions and methods are provided for the identification of peptide sequences that are ligands for a T cell receptor (TCR) of interest, in a given MHC context.

Ligand discovery for T cell receptors

The invention provides methods and compositions for the expression of small RNA molecules within a cell using a lentiviral vector. The methods can be used to express doubles stranded RNA complexes. Small interfering RNA (siRNA) can be expressed using the methods of the invention within a cell, which is capable of down regulating the expression of a target gene through RNA interference. A variety of cells can be treated according to the methods of the invention including embryos, embryogenic stem cells, allowing for the generation of transgenic animals or animals constituted partly by the transduced cells that have a specific gene or a group of genes down regulated.

Method for expression of small RNA molecules within a cell

In one aspect, the invention provides methods and compositions for the expression of small RNA molecules within a cell using a retroviral vector (FIG.A). Small interfering RNA (siRNA) can be expressed using the methods of the invention within a cell. In a further aspect, the invention provides methods for producing siRNA encoding lentivirus where the siRNA activity may interfere with the lentiviral life cycle. In yet a further aspect, the invention provides methods for expression of a small RNA molecule within a cell, such as an siRNA capable of downregulating CCR5, wherein expression of the small RNA molecule is relatively non-cytotoxic to the cell. The invention also includes small RNA molecules, such as an siRNA capable of downregulating CCR5, that are relatively non-cytotoxic to cells.

Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell

Provided herein are method and kits for elucidation of immunological repertoires (comprising functional pairs of immune cell receptors and antigens). In some embodiments, an immune cell receptor and an antigen that it binds to are isolated, and sequence information is obtained about the immune cell receptor and antigen. In some embodiments, an antigen expressed on a first cell and an immune cell receptor expressed on a second cell can form a synapse, mRNAs can be isolated from the resulting doublet of cells, and sequences of the functional immune cell receptor and its antigen can be obtained from the mRNAs.

Functional characterization of immune repertoires

Methods and compositions are provided for delivery of a polynucleotide encoding a gene of interest, typically an antigen, to a dendritic cell (DC). The virus envelope comprises a DC-SIGN specific targeting molecule. The methods and related compositions can be used to treat patients suffering from a wide range of conditions, including infection, such as HIV/AIDS, and various types of cancers.

Packaging cell for making a pseudotyped lentivirus

The present disclosure relates to the finding that microRNA-155 plays a role in inflammation, hematopoiesis and myeloproliferation, and that dysregulation of microRNA-155 expression is associated with particular myeloproliferative disorders. Disclosed herein are methods and compositions for diagnosing an treating disorders, including inflammation and myeloproliferation, modulating the levels of expression of one or more genes selected from the group consisting of Cutl1, Arntl, Picalm, Jarid2, PU.1, Csf1r, HIF1&#x3b1;, Sla, Cepb&#x3b2;, and Bach1, and the like.

MicroRNA inhibition for the treatment of inflammation and myeloproliferative disorders

Methods of delivering a pseudotyped lentivirus

Gene targeting is a technique to introduce genetic change into one or more specific locations in the genome of a cell. For example, gene targeting can introduce genetic change by modifying, repairing, attenuating or inactivating a target gene or other chromosomal DNA. In one aspect, this disclosure relates to methods and compositions for gene targeting with high efficiency in a cell. This disclosure also relates to methods of treating or preventing a genetic disease in an individual in need thereof. Further disclosed are chimeric nucleases and vectors encoding chimeric nucleases.

Use of chimeric nucleases to stimulate gene targeting

Methods and compositions are provided for delivering a polynucleotide encoding a gene of interest to a target cell using a virus. The virus envelope comprises a cell-specific binding determinant that recognizes and binds to a component on the target cell surface, leading to endocytosis of the virus. A separate fusogenic molecule is also present on the envelope and facilitates delivery of the polynucleotide across the membrane and into the cytosol of the target cell. The methods and related compositions can be used for treating patients having suffering from a wide range of conditions, including infection, such as HIV; cancers, such as non-Hodgkin's lymphoma and breast cancer; and hematological disorders, such as severe combined immunodeficiency.

Method of targeting gene delivery using viral vectors

Growing community of inventors

Pasadena, CA, United States of America

David Baltimore