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Identification Of A Factor That Promotes Human Hematopoietic Stem Cell Self-Renewal

The Mikkola group at UCLA has discovered a novel regulator of hematopoietic stem cell self-renewal. The overexpression of this regulator increases the yield of ex vivo stem cell expansion and could thereby improve the efficiency of stem cell therapies. 

miRNA Therapeutic for Huntington's Disease

Huntington's disease (HD) is a neurodegenerative genetic disorder caused by the mutation of a CAG repeat within the HD gene that causes the accumulation of mHTT species. The biochemical pathways involved in HD are complex and not yet fully uncovered. As a consequence, there are currently no successful disease-modifying therapies for HD. The identification of cellular targets that impact the disease's onset and progression is critical for the development of new treatments. Researchers at UCI and University of Iowa have identified a new target and developed an miRNA therapeutic that reduces accumulation of mHTT in a mouse model of HD.

Chronic Villus Derived Stem Cells for Autologous Prenatal Therapy of Hemophilia A

Researchers at the University of California, Davis have developed a method and composition using chorionic villus-derived stem cells that transgenically express Factor VIII for the treatment and prevention of hemophilia A (HA).

Transposon Vector for Vertebrate & Invertebrate Genetic Manipulation

Background: Therapeutic delivery of genes is a rapidly evolving technique used to treat or prevent a disease at the root of the problem. The global transgenic market is currently $24B, growing at an annual projected rate of 10%. Currently, a variation of this technique is widely used on animals and crops for production of desirable proteins, but this is a heavily infiltrated market. Thus, entering the gene therapy segment is more promising and would enhance the growth of this industry.  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 improve 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.

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.

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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