US 2013/0171124 A1 · Compositions And Methods Of Treating Head And Neck Cancer

Compositions And Methods Of Treating Head And Neck Cancer

AssigneesThe Broad Institute, Inc., Massachusetts Institute of Technology, President and Fellows of Harvard College, Massachusetts Eye and Ear Infirmary, Dana-Farber Cancer Institute, Inc., University of Pittsburgh of the Commonwealth of Higher Education, Instituto Carlos Slim De La Salud, A.C.

InventorsLe Cong, Ann Marie Egloff, Levi A. Garraway, Jennifer Rubin Grandis, Eric S. Lander, Nicholas Stransky, Aaron D. Tward, Feng Zhang

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Abstract

The present invention provides methods related to the treatment of head and neck squamous cell carcinoma (HNSCC) and its associated premalignant lesions. In particular, the invention features methods which may specifically target HNSCC-associated genes and alter gene expression to treat or alleviate a symptom of HNSCC, or its related premalignant lesions. These methods may involve decreasing the function of an HNSCC-associated gene with aberrant gain-of-function; or increasing the function of an HNSCC-associated gene with aberrant loss-of-function.

Claims32

Dependent claims

A method of treating or alleviating a symptom of head and neck squamous cell carcinoma (HNSCC) or its related premalignant lesions, comprising administering to a subject in need thereof

a) a compound that decreases the function of an HNSCC-associated gene with aberrant gain-of-function;
or
b) a compound that increases the function of an HNSCC-associated gene with aberrant loss-of-function
wherein the HNSCC gene is selected from FIG. 22.

1 more element

A method of altering expression of a genomic locus of interest in a mammalian cell, comprising contacting the genomic locus with a non-naturally occurring or engineered composition comprising a deoxyribonucleic acid (DNA) binding polypeptide comprising:

(a) a N-terminal capping region
(b) a DNA binding domain comprising at least five or more TALE monomers and at least one or more half-monomers specifically ordered to target the genomic locus of interest, and
(c) a C-terminal capping region
wherein (a), (b) and (c) are arranged in a predetermined N-terminus to C-terminus orientation,
wherein the polypeptide includes at least one or more effector domains, and
wherein the polypeptide is encoded by and translated from a codon optimized nucleic acid molecule so that the polypeptide preferentially binds to DNA of the genomic locus;
and
wherein the genomic locus of interest is associated with any gene set forth in FIG. 22.

5 more elements

Dependencies are inferred from the claim text.

Facts

Application

13/557,988Filed 25 Jul 2012

Priority

27 Jul 2011

Published

4 Jul 2013

Term ends

Not estimatedThe source records don’t support an estimate.

Classification20 codes

A61K 38/164
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof: from bacteria
A61K 38/465
Hydrolases: acting on ester bonds , e.g. lipases, ribonucleases
A61K 45/06
Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00: mixtures of active ingredients without…
A61P 35/00
Antineoplastic agents