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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).

Novel Gene Delivery Approach for Controlled and Sustained Transfection of Cells

UCLA researchers in the Department of Chemical and Biomolecular Engineering have developed a novel hydrogel scaffold-mediated gene delivery approach that can achieve enhanced and sustained transfection of endogenous cells in vivo.

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.

Methods of Monitoring and Manipulating the Fate of Transplanted Cells

Tumor initiation and progression into metastasis are accompanied by complex structural changes in the extracellular matrix and cellular architecture that alters the stiffness in the microenvironment of the cell.

Spinal Subpial AAV-mediated Gene or Antisense Oligonucleotide Delivery System

Currently used approaches to delivery vectors or ASO into the spinal parenchyma use one or two techniques, each having a substantial limitation as compared to this new delivery system. First, intrathecal delivery is used when vectors or ASO is injected into spinal intrathecal space (i.e. outside of pial membrane). Using this approach, no deep parenchymal transgene expression is seen after AAV9 delivery. Intrathecal delivery of ASO leads to a  good penetration of ASO into spinal parenchyma but is seen in the whole spinal cord (i.e. from cervical to sacral segments). No segment restricted distribution of ASO can be achieved by intrathecal delivery. A second technique, also in use currently, is a direct spinal parenchymal injection. By using this approach a segment specific transgene expression or ASO distribution can be achieved in spinal parenchyma. A major limitation of this technique is its invasive nature because a direct spinal parenchymal needle penetration is required.


This invention establishes a new approach to treating liver fibrosis using gene therapy.

Stabilizer Cells to Treat Cardiac Arrhythmias

UCLA researchers in the Department of Cardiology have developed a novel gene therapy for cardiac arrhythmias.

Robust Genome Engineering in Primary Human T Cells using CRISPR/Cas9 Ribonucleoproteins

This invention enables highly effective experimental and therapeutic genomic engineering of primary human T cells and other hematopoietic cells with CRISPR/Cas9 ribonucleoprotein (RNP) technology.  

MicroRNA-214 as a Diagnostic and Prognostic Biomarker for Ulcerative Colitis and Colitis-Associated Colon Cancer Patients

Dr. Dimitrios Iliopoulos in UCLA Department of Medicine has identified a novel biomarker, microRNA-214 (miR-214), that predicts, at near 100% specificity, an ulcerative colitis patient’s risk for developing colon cancer.

Diagnostic, Prognostic and Therapeutic Uses of Non-Coding RNAs in Leukemia

The Rao group at UCLA has developed a method of using lincRNA expression levels as a diagnostic and prognostic tool for B acute lymphoblastic leukemia. Furthermore, regulation of certain leukemia-associated lincRNA may hold therapeutic potential.

Dendritic Peptide Bolaamphiphiles for siRNA Delivery

Novel dendritic peptide bolaamphiphiles that are safe and efficient for siRNA delivery.

Defense Signatures of Glioblastoma Stem Cells: Predicting Prognosis and Treatment Targets for Brain Cancer

Dr. Cho-Lea Tso from the Department of Surgery/Surgical-Oncology at UCLA has identified ribosomal protein RPS11 as a defense signature and survival factor for treatment-resistant glioblastoma stem cell clones (TRGC). In collaboration with Dr. William Yong from Pathology and Lab Medicine, they found that RPS11 can serve as a biomarker that predicts poor prognostic outcome and short survival when overexpressed in patients with newly diagnosed glioblastoma. Since RPS11 is a stress-response associated gene and a survival factor for TRGC, it can potentially serve as a new therapeutic target for novel brain cancer therapy and be used to prevent tumor regeneration.

Biosynthetic Genes That Produce Acetaminophen

Acetaminophen (also known as Paracetamol or APAP) is a pain reliever and fever reducer.  Acetaminophen is found in many prescription medicines and is one of the most widely used medicines in the U.S.  However, current methods of production involve hydrogenation of a nitroaryl precursor to aminophenol, followed by acetylation.  Unfortunately, the step of hydrogenation brings with it explosion risks and requires infrastructural safety protections which can be costly.  UC Berkeley researchers have developed an innovative technology that produces acetaminophen though biosynthesis using a recombinant host cell without the problems associated with existing production methods. 

Novel Chitosan Derivative as a Systemic Drug Delivery Agent and an Antibiotic Treatment

Researchers at the University of California, Irvine have developed a novel chitosan derivative that may be used simultaneously as a systemic drug delivery agent and a systemic antibiotic treatment.

Cost-effective Method to Quickly Produce and Purify Large Quantities of Biologically Active ncRNAs

Researchers at the University of California, Davis have developed a novel method to biosynthesize large quantities of biologically active ncRNA agents (e.g., miRNAs and siRNAs) for functional and therapeutic uses. 

Read-Through Compound Prodrugs Suppressing Premature Nonsense Mutations

UCLA researchers in the Department of Neurology have identified a novel prodrug to enhance the aqueous solubility of RTC13 for the treatment of Duchenne Muscular Dystrophy and other genetic disorders caused by nonsense mutations.

Dendronized Polymer Vectors For siRNA Delivery

Researchers at the University of California, Irvine have developed a new medium for delivery of siRNA genetic materials into cells. This medium is a vector, and its architecture allows for optimal siRNA binding. RNAi has tremendous potential for therapeutic treatment, and this vector allows for safe and efficient intracellular delivery of siRNA.

Novel Method of Using Modified and Optimized Bacterial-derived Genetic CRISPR System for Imaging, Regulating and Editing Mammalian Genomic Elements

This invention is a novel method using optimized small guide RNAs (sgRNAs) to enable dynamic imaging, editing and regulation of specific genomic elements in living mammalian cells via the CRISPR system.

Autologous Adipose-Derived Mesenchymal Stem-Cell Therapy for Cats with Chronic Mucosal Inflammatory Disease

Chronic mucosal inflammatory diseases (for example, chronic gingivostomatitis) are poorly understood diseases in cats characterized by severe inflammation of the gums and oral cavity.  Cats with Chronic mucosal inflammatory disease have painful, debilitating lesions in their mouth that affect their ability to eat or be a suitable companion animal.  Unfortunately, , there is no truly effective medical treatment available, until now. Recently, researchers at the University of California, Davis have developed an innovative and fully effective treatment for this disease in cats by administering autologous Adipose-Derived Mesenchymal Stem-Cells (adMSC).


Microinjection represents the “gold standard” for cellular manipulation, due to its precision, safety, and applicability to a wide variety of cell types and molecules.  However, the reliance of current instrumentation on skilled operators and serialized injection methodologies limits availability and throughput (~3 cells/min), thus hampering progress in many areas including ex-vivo cell therapies. Automation efforts have shown promise for improving success rates, but the expense of instrument complexity and limited gains in throughput (≤35 cells/min) have held back its universal adoption.    

Novel and Effective Gene Therapy for Critical Limb Ischemia

Critical limb Ischemia (CLI) represents a significant unmet medical need since there are currently no effective pharmaceuticals or biologic therapies for treatment of patients with occluded vessels. Researchers at the University of California, Davis have designed a Mesenchymal Stem/Stromal Cell (MSC) which secretes supraphysiological amounts of human Vascular Endothelial Growth Factor (VEGF) for revascularization of blood vessels and the treatment of peripheral artery diseases such as CLI.

Expression, Purification, And Isolation Of The Full Length Human Breast Cancer Susceptibility Gene 2 (Brca2) Protein

Method for expression, purification, and isolation of the full length human breast cancer susceptibility gene 2 (BRCA2) protein.

Cyclic Amp-Incompetent Adenylyl Cyclase Gene Transfer For Heart Failure

Heart failure is the most common cause of non-elective admission to the hospital in subjects 65 years and older. Despite optimal drug and device therapy, prognosis in heart failure is dismal. Many clinical trials of drugs that increase heart function (“inotropes”) have failed, possibly due to the deleterious effects of agents that increase cAMP. An alternative strategy is to alter myocardial calcium handling or myofilament response to calcium using agents that do not affect cAMP. Expression of a catalytically impaired adenylate cyclase type 6 mutant molecule (AC6mut), one that markedly reduces cAMP production, is associated with normal cardiac function in response to β-adrenergic receptor stimulation. The mechanism is through enhanced effects of AC6mut on Ca2+ handling - effects that do not require cAMP. These data are important in clinical settings for two reasons: 1) the results provide additional insight regarding the interplay between Ca2+ handling and βAR signaling vis-à-vis LV function; and 2) AC6mut may provide inotropic support free from the potentially deleterious effects of increased cAMP.

Treating Type 2 Diabetes by Targeting CAP Protein in the Macrophage

CAP (Cbl associated protein) is an adapter protein that is ubiquitously expressed. CAP acts in concert with Cbl to stimulate glucose uptake in skeletal muscle and adipose tissue as well as to induce the proliferation and migration of macrophages. Whole body CAP gene deletion in mice results in a protection from insulin resistance induced by high fat diet. However, exercise capacity is severely blunted in these mice.

Novel Pseudotyped Lentiviruses for Targeted Gene Therapy

UCLA researchers have developed a novel lentivirus pseudotype to specifically infect EphrinB2 expressing primary cells including difficult to transduce cell types like stem cells and neurons. This virus also has the ability to improve targeted gene therapy methods as it is the only known lentivirus not trapped in the liver following systemic delivery.

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