General Electric Company

Children's Medical Center Corporation

Momentive Performance Materials Gmbh

Cincinnati Children's Hospital Medical Center

Bluebird Bio, Inc.

David A Williams

Shayne Joseph Landon

Vikram Kumar

Indumathi Ramakrishnan

Yi Zheng

Edward Joseph Nesakumar

Thomas L Evans

Edward J Nesakumar

Richard Gregory

Michael Milsom

Edward M Jeram

Teresa Grocela-Rocha

August O Liermann

Gerardo Rocha-Galicia

Bruce Boudreau

Daniel Joseph Brunelle

Thomas G Shannon

Brian J Ward

Paul R Willey

Sachin Askok Shelukar

Serena De Vita

James Anthony Cella

Niles R Rosenquist

Eric Raymond Pohl

Michael Alan Vallance

Anantharaman Dhanabalan

Michael Douglas Arnett

Daniel Anthony Nowak

Kevin R Stewart

Wieslaw Adam Mazur

William Hilakos

Thomas Michael Moors

Elbridge A O'Neil, Jr

Dwight J Patterson

Carlos Alberto Collado

Emilio Fernandez

Gabor Veres

Kenneth Lorenzo Parks

Zhiqing Lin

Gilbert Joseph Dean

Hui Kuang

Benjamin Erick Baker

Patrick Mohr

Reuben Correia

Indumothi Ramakrishnan

Huzoor Akbar

Scott Pregont

Rachel Elizabeth Kyano

Swaroopa Guda

Gary Morgan Lucas

Sachin A Shelukar

Edward Matthew Jeram

Gerardo Galicia Rocha

Embodiments herein provide specially designed synthetic BCL11A-targeting microRNAs for RNA polymerase II expression, and methods of use to treat hemoglobinopathies, such as sickle cell disease or thalassemia, by increasing the expression levels of fetal hemoglobin levels.

BCL11A microRNAs for treating hemoglobinopathies

Embodiments herein provide specially designed synthetic BCL11A-targeting microRNAs for RNA polymerase II expression, and methods of use to treat hemoglobinopathies such as sickle cell disease or thalassemia by increasing the expression levels of fetal hemoglobin levels.

Compositions and methods to treating hemoglobinopathies

This disclosure relates to certain compounds that are effective in the treatment of a Rac-GTPase mediated disorder (e.g., acute lymphoblastic or chronic myelogenous leukemia), as well as methods for the manufacture of and the use of these compounds (e.g., for treating a Rac-GTPase mediated disorder).

Compounds for treating Rac-GTPase mediated disorder

This disclosure relates to compositions including certain compounds identified by a quantitative, high throughput assay to be effective in the treatment of a Rac-GTPase mediated disorder (e.g., acute lymphoblastic or chronic myelogenous leukemia), as well as methods for the manufacture of and the use of these compounds for treating a Rac-GTPase mediated disorder.

The invention provides improved gene therapy vectors, compositions, and methods.

Vectors and compositions for treating hemoglobinopathies

Compounds for treating rac-GTPase mediated disorder

Embodiment herein provide specially designed synthetic BCL11A-targeting microRNAs for RNA polymerase II expression, and methods of use to treat hemoglobinopathies such as sickle cell disease or thalassemia by increasing the expression levels of fetal hemoglobin levels. In particular illustrative embodiment, the present invention provides, in part, improved compositions and methods for achieving gene therapy in hematopoietic cells and hematopoietic precursor cells, including erythrocytes, erythroid progenitors, and embryonic stem cells. The invention further provides improved gene therapy methods for treating hematopoietic-related disorders.

Chimeric peptides or fusion proteins are disclosed that include a RhoGAP activity domain and at least one specificity domain that targets a specific Rho protein. The fusion proteins can be used to inhibit any GTPase activity within a cell. The fusion proteins are particularly advantageous for the treatment of cancer. The present invention generally relates to chimeric peptides capable of regulating GTPases, and more particularly, to methods of targeting individual GTPases by using GTPase-activating proteins. Such proteins may be used for the treatment of cancers and other GTPase-related diseases. This invention relates to nucleic acid molecules and the encoded GTPase activating proteins, and variants thereof, and to the use of these molecules in the characterization, diagnosis, prevention, and treatment of cell signaling, immune, and cell proliferative disorders, particularly cancer. Disclosed herein are compounds and methods for regulating transcription of a selected gene.

Chimeric peptides for the regulation of GTPases

There is provided herein a composition comprising: (A) a silanol and/orhydrocarbon-terminated polyorganosiloxane in an amount of from about 30 weight percent to about 90 weight percent; and, (B) a moisture curable silylated polyurethane resin in an amount of from about 10 weight percent to about 70 weight percent. There is also provided herein in another embodiment a two part sealant composition wherein the first part comprises: (A) a silanol and/or hydrocarbon-terminated polyorganosiloxane in an amount of from about 15 weight percent to about 60 weight percent, (B) a moisture curable silylated polyurethane resin in an amount of from about 2 weight percent to about 32 weight percent, and (C) a filler, said weight percents being based on the weight of the first part. In addition, there is provided herein a fumed silica composition comprising: (A) a silanol and/or hydrocarbon-terminated polyorganosiloxane in an amount of from about 20 weight percent to about 62 weight percent; (B) a moisture curable silylated polyurethane resin in an amount of from about 8 weight percent to about 38 weight percent; and, (C) a treated fumed silica in an amount of from about 5 to about 10 weight percent.

Silylated polyurethane/polyorganosiloxane blend and sealant composition and fumed silica composition containing same

The invention relates to an insulated glass unit having an increased service life. An outer glass pane and inner glass pane are sealed to a spacer to provide an improved gas impermeable space. The glass unit includes a curable two-part sealer composition which, upon curing, exhibits unexpectedly high adhesion to substrates.

Insulated glass unit possessing room temperature-cured siloxane sealant composition of reduced gas permeability

The invention relates to a high thermal efficiency, insulated glass unit structure sealed with a cured composition containing, inter alia, diorganopolysiloxane(s) and organic nanoclay(s), the cured composition exhibiting low permeability to gas(es).

Insulated glass unit possessing room temperature-curable siloxane-containing composition of reduced gas permeability

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Ganesvoort, NY, United States of America

David A Williams