University of California

Sylvia Evans

Ju Chen

Chenleng Cai

Kenneth R Chien

Michael G Rosenfeld

Alessandra Moretti

Wenlai Zhou

Stephanie Cherqui

Eric D Adler

Karl Laugwitz

Lizhu Lin

Karl-Ludwig Laugwitz

Ken Chien

Yibin Wang

Yibing Qyang

Provided herein are methods for treating a lysosomal transmembrane protein disease or disorder through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. Also provided are vectors containing the nucleic acid molecule.

Methods of treating lysosomal disorders

The present invention relates to a small molecule high-throughput screening assay consisting of detectably labeled cardiac progenitor cells. The invention also describes a method of identifying small molecules from the high-throughput assay affecting cardiogenesis and/or modulating cardiac progenitor cell development. Also described are methods of stimulating maturation of cardiac progenitor cells using a GSK-3&#x3b2; inhibitor.

Methods of identifying small molecules for renewals, survival and migration of cardiac progenitors

The invention herein provides a mode of treating a disease associated with the regulation of T-Box and N-Myc gene, which includes cancers and heart disease in human and other subjects by identifying and administering a compound which modulates T-Box or N-Myc function. The invention also provides polynucleotides, polypeptides, vectors, cells, tissues and organisms useful in the identification and treatment of metabolic syndrome. A number of desirable cell proliferation and senescence regulating aspects are achieved by various embodiments of the present invention.

Compositions and methods for treating diseases associated with T-box and N-Myc

The present invention provides in vitro methods of expansion and propagation of undifferentiated progenitor cells and more specifically undifferentiated progenitor cells containing Islet1, a marker apparently unique to proliferating cardiac stem cells. Methods are described for isolation of stem cell populations as well as for provoking expansion and propagation of undifferentiated progenitor cells without differentiation, to provide cardiac repair or improve cardiac function, for example.

Use of islet 1 as a marker for isolating or generating stem cells

The invention herein provides a mode of treating a disease associated with the regulation of T-Box and N-Myc gene which includes cancers and heart disease in human and other subjects by identifying and administering a compound which modulates T-Box or N-Myc function. The invention also provides polynucleotides, polypeptides, vectors, cells, tissues and organisms useful in the identification and treatment of metabolic syndrome. A number of desirable cell proliferation and senescence regulating aspects are achieved by various embodiments of the present invention.

The present invention relates to a human type-5 recombinant adenovirus vector for achieving cardiac restricted transcription involving utilization of the cardiomyocyte-restricted cardiac ankyrin repeat protein (CARP) promoter with inclusion of the inverted terminal repeat sequences from human adeno-associated virus (AAV). Using green fluorescent protein (GFP) as a marker gene, the recombinant adenovirus vector (Ad/CG/ITR) is shown to direct transgene expression to myocardial tissue in vivo and in vitro in mouse models.

Growing community of inventors

Del Mar, CA, United States of America

Sylvia Evans