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Browse Category: Medical > Disease: Genetic Diseases and Dysmorphic Syndromes


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Polyrotaxane Nanoparticles for Delivery of Large Plasmid DNA in Duchenne Muscular Dystrophy

UCLA researchers have designed, synthesized, and validated a polyrotaxane nanocarrier for targeted delivery of large plasmids for gene therapy applications for treatment of Duchenne muscular dystrophy and cancer.

High-Throughput Microfluidic Gene-Editing via Cell Deformability within Microchannels

UCLA researchers in the Departments of Pediatrics and Chemistry & Biochemistry have developed a microfluidic device for delivery of biomolecules into living cells using mechanical deformation, without the fouling issues in current systems.

AGPAT5 as a Molecular Mediator of Insulin Resistance

UCLA researchers in the Departments of Medicine and Cardiology have identified a novel gene and pathway in the regulation of insulin sensitivity and discovered an inhibitor of this gene useful for treating AGPAT5-related diseases.

Non-Invasive Preimplantation Genetic Screening using Free DNA from Spent Embryo Media

A UCLA physician has developed a novel method to screen for embryonic genetic competency during the in vitro fertilization process by using the free DNA released from a candidate embryo.

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. 

Methods and Compounds for Treating Mitochondrial Diseases

Researchers at the University of California, Davis have developed a treatment for mitochondrial disease through a repurposing approach whereby a library of FDA-approved drugs was screened for previously unknown therapeutic effectiveness in these diseases.

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.

New Therapeutic Leads for Cachexia and Anorexia

The agouti-related protein (AgRP) produced in the brain is a potent appetite stimulant. AgRP binds with high affinity to melanocortin (MC) receptors and plays a central role in energy balance by stimulating feeding and decreasing energy expenditure. This modified AgRP invention provides unexpectedly superior results when used as an appetite stimulant. AgRP animal models have successfully demonstrated enhanced feeding for an extended period, longer acting than any other known hormone or drug used to treat diseases such as cachexia or anorexia.

Peripheral Biomarkers For The Assessment Of Autism

Researchers in the Department of Neurology and the Autism Center in the Semel Institute at UCLA have identified genetic factors which are associated with autism.

Gene Therapy For Usher Syndrome Type 1B

Brief description not available

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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