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A Transposon Vector From Aedes Aegypti For Use In Vertebrate And Invertebrate Gene Transfer

Background: Therapeutic delivery of genes is a rapidly evolving technique used to treat or prevent a disease at the root of the problem. Another widely used variation of this technique is to insert a transgene into animals and crops for production of desirable proteins. The global transgenic market is currently $24B with annual growth projections of 10%.  Brief Description: UCR Researchers have identified a novel transposon from Aedes aegypti mosquitoes. This mobile DNA sequence can insert itself into various functional genes to either cause or reverse mutations. They have successfully developed a transposon vector system that can be used in both unicellular & multicellular organisms, which can offer notable insight to enhance current transgenic technologies as well as methods of gene therapy.

Stem Cell Therapy for Spina Bifida

Researchers at the University of California, Davis have developed a novel method to treat Spina Bifida or other spinal cord injuries.

Method to Expand and Transduce Cultured Human Small and Large Intestinal Stem Cells

Dr. Martin G. Martin and colleagues in the Department of Pediatric Gastroenterology and Surgery at UCLA have developed a novel method of expanding and differentiating human small intestinal stem cells in culture.

New Chemical Entities for Treatment of Down Syndrome and Related Phenotypes

Down Syndrome (aka Trisomy-21), which affects approximately one of every 700 babies born in the United States each year, is characterized by varying degrees of stunted cognitive and physical ability as well as characteristic facial and body features. The extent to which an individual will be able to integrate into society is determined by the severity of the condition as well as the timing and extent of intervention. Historically, intervention has included screening with subsequent dedicated therapy and training. However, recent work on the etiology of Down Syndrome (see Lu et al., below) has suggested that inhibition of a chromosome 21 (HSA21)-associated, oligodendrocyte transcription factor (OLIG2) may blunt the effects of over-expression of a subset of HSA21 genes that inhibit the growth of neural progenitors, which are necessary for normal brain development. This approach provides new hope for a prevalent disease with a tremendous burden on society and for which no medical treatment exists.

Chordin Compositions

Purified chordin proteins and processes for producing them are disclosed. DNA molecules encoding the chordin proteins are also disclosed. The proteins may be used in the treatment of bone, cartilage, other connective tissue defects and disorders, including tendon, ligament and meniscus, in wound healing and related tissue repair, as well as for treatment of disorders and defects to tissues which include epidermis, nerve, muscle, including cardiac muscle, and other tissues and wounds, and organs such as liver, brain, lung, cardiac, pancreas and kidney tissue. The proteins may also be useful for the induction inhibition of growth and/or differentiation of undifferentiated embryonic and stem cells. The proteins may be complexed with other proteins, particularly members of the transforming growth factor-beta superfamily of proteins.

Protein Markers for Early Lung Cancer Detection

Lung cancer is the leading cause of cancer related death in the United States, claiming more than 160,000 lives annually. The ability to non-invasively screen for lung cancers before they have the opportunity to grow and metastasize would be a significant achievement, and would drastically reduce mortality from this disease.

Gene Therapy For Usher Syndrome Type 1B

Brief description not available

Assessment of Allele-Specific Expression in Cells and Tissue

The success of gene therapy methods such as small fragment homologous recombination and cDNA-based gene therapies is often difficult to quantify. These methods often lack an endogenous selection mechanism that can be used to differentiate and quantify targeted cells. Therefore, it is difficult to monitor and map the success of gene therapy in patients. UCSF investigators have developed methods and compounds enabling the measurement of expression of mutated and non-mutated alleles in the tissue or cells of a human subject. The method, an in situ RT-PCR assay, can be used for diagnosis of allelic variation and the monitoring of gene therapy for a variety of gene-based diseases, especially cystic fibrosis.

Brown Adipose Tissue Cell Lines Derived from Protein-Tyrosine Phosphatase 1B Knockout Mice Reconstituted with Sumoylation Mutant PTP1B K4R

Platform for Testing the Effects of Human PTP1B Inhibition on Insulin Signaling, Adipose Differentiation and Glucose Uptake

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