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Adenylyl Cyclase Catalytic Domain Gene Transfer for Heart Failure

Heart failure (HF) is a disease of epidemic portions in the United States affecting over 6 million patients with heart failure in the US, with 400,000 new cases per year. It is the most common cause of non-elective admission to the hospital in subjects 65 yrs and older. The introduction of new drugs over the last 30 years that target pathways critical to progression of HF, along with implantable cardiac defibrillators and resynchronization devices have shown some successes, however, both the morbidity and mortality associated with heart failure remains at unacceptable levels, with as many as 30-40% of affected individuals dying within 5 years of diagnosis. Recently, preclinical and clinical trials have tested gene transfer to increase left ventricular (LV) function, especially in heart failure with reduced ejection fraction.

Use of Gene Therapy to Treat Joint Disease and Synovial Tumors

The National Center for Advancing Translational Sciences and Genetic and Rare Diseases Information Center characterizes Pigmented villonodular synovitis (PVNS) as a rare disease estimated to occur in ~ 5-6 people out of 100,000. This locally invasive tumor most often occurs in younger adults and causes severe damage to joints. The first line of treatment is surgery but at least 50% of patients require multiple surgeries over many years due to re-growth of the tumor.

Improvement To Retroviral Vectors Containing The Human Ubiquitin C Promoter

UCLA researchers in the Department of Molecular Biology have developed a lentiviral vector, “pCCLc-roUBC”, containing the cellular promoter from the human ubiquitin C gene (UBC), to improve transgene expression in retroviral vectors.

Augmentations to Lentiviral Vectors to Increase Expression

UCLA researchers in the Department of Microbiology, Immunology and Molecular Genetics have developed a novel method to produce short lentiviral vectors with tissue-specific expression, with a primary focus on lentiviral vectors for treating sickle cell disease and other disorders of hemoglobin.

Optimized Lentiviral Vector for Stem Cell Gene Therapy of Hemoglobinopathies

UCLA researchers in the Department of Microbiology, Immunology and Molecular Genetics have developed a novel method to produce short lentiviral vectors with tissue-specific expression, with a primary focus on lentiviral vectors for treating sickle cell disease and other disorders of hemoglobin.

Technologies that can be Used to Selectively Bind Messenger RNA and Enhance Protein Translation

Control of gene expression is a general approach to treat diseases where there is too much or too little of a gene product. However, while there are many methods which are available to downregulate the expression of messenger RNA transcripts, very few strategies can upregulate the endogenous gene product. The vast majority of gene regulatory drugs which are commercially available or being developed are designed to knockdown gene expression (i.e. siRNAs, miRNAs, anti-sense, etc.). There exist some methods to enhance gene expression, such as the delivery of messenger RNAs; although, therapeutic delivery of such large and charged RNA molecules is technically challenging, inefficient, and may not be practical. There are also classical gene therapy approaches where a gene product is delivered as viral-encoded products (AAV or lentivirus-packaged). However, these methods suffer from not being able to accurately reproduce the correct alternatively spliced isoforms in the right ratios in cells.  

Generation Of Minimal Enhancer Elements Using Massively Parallel Reporter Assays

UCLA researchers in the Department of Microbiology, Immunology and Molecular Genetics have developed a novel method to produce short lentiviral vectors with tissue-specific expression, with a primary focus on lentiviral vectors for treating sickle cell disease and other disorders of hemoglobin.

A Method to Prevent the Myelin Abnormalities Associated with Arginase Deficiency

UCLA researchers in the Department of Surgery have developed a gene therapy to prevent dysmyelination (and other CNS abnormalities) as a result of arginase deficiency.

Treatment for Restoring Ureagenesis in Carbamoyl Phosphate Synthetase 1 Deficiency

UCLA researchers in the Department of Surgery have developed a gene therapy to treat carbamoyl phosphate synthetase 1 deficiency.

Method To Implement A Crispr-Cas9 Copycat Gene Drive In Rodents

Currently, alleles at multiple loci in the mouse genome must be combined by Mendelian genetics in crosses of animals to one another to produce a desired compound mutant genotype. For example, to combine homozygous mutations at two loci, animals that are heterozygous for each gene must be produced by breeding, and these are subsequently crossed to one another. Since the frequency of homozygosity for each allele is 1:4 the frequency of homozygosity for both genes is 1:16. Since the average litter of mice is approximately 10 pups, and the generation time from conception to reproductive age is about 3 months, this requires a substantial number of animals and time. With the addition of each new locus (three, four, etc), the cost measured in animals, time, and money increases exponentially. These factors increase substantially more if two or more loci are genetically linked, which requires rare recombination events to combine engineered alleles on the same chromosome. The CRISPR-Cas9 gene drive system stands to revolutionize rodent breeding. If each desired allele is encoded as a gene drive element that contains an sgRNA designed to target the same genomic location in the wild type homologous chromosome, each locus will be “driven” to homozygosity in the presence of Cas9. Therefore, in order to combine three alleles, for example, a mouse with one gene drive element (A) would be crossed to a mouse that encodes Cas9. Offspring of this cross would then be crossed to mice carrying gene drive element B, and these offspring would be crossed to mice carrying gene drive element C. In the presence of Cas9 at each generation, these gene drive elements at three distinct loci will be converted to homozygosity such that 50% of offspring, those that inherit Cas9, will be triple homozygous after three generations, even if they are genetically linked loci. A CRISPR-Cas9 mediated gene drive leverages the native cellular mechanism of homology directed repair to copy a desired allele from one chromosome to another. This process can convert a heterozygous genotype to homozygosity in a single generation. While CRISPR-Cas9 gene drives have been implemented in two species of insects, flies and mosquitos, it has not been reported in any non-insect animal species. 

Biodegradable Polymeric Vectors For Delivery Of Various RNAs

Current methods for ribonucleic acid (RNA) delivery are inefficient and toxic. UCI researchers have synthesized a new delivery system that is not only efficient and non-toxic but also allows the delivery of RNAs of multiple shapes and sizes.

Biotinylated Ligand-Directed Targeting Lentiviral Vectors

UCLA researchers in the Department of Medicine have developed a novel method to conjugate targeting ligands on lentiviral vectors.  The method allows for selective transduction of mammalian cells types avoiding non-target organs.

Transcription Factor Treatment for Schizophrenia and Bipolar Disorder

Current time to diagnosis for Schizophrenia and Bipolar Disorder is extremely lengthy (~6 months), and a delay in treatment greatly increases risk for suicidal thoughts. Once diagnosis has occurred, therapeutic options for both mental illnesses are greatly varied and have numerous side effects. To address both issues, UCI researchers have developed a novel way to diagnose and treat Schizophrenia and Bipolar Disorder by targeting specific transcription factors.

Novel CRISPR Gene Therapy for Haploinsufficiency

This technology presents a way to treat human genetic disease caused by haploinsufficiency and reduced protein production. The method employs the use of adeno-associated viral (AAV) vectors for the in vivo delivery of a CRISPR-based gene expression activator (CRISPRa) that boosts transcription from the existing functional copy of the affected gene.

Targeting Lentiviral Vectors To Specific Cells And Tissues

Researchers in the UCLA Departments of Medicine and Microbiology, Immunology and Molecular Genetics have developed retroviral vectors pseudotyped with a modified Sinbus virus envelope that exhibit reduced tropism and can be used for the targeted transduction of heterologous genes into cells.

Human-Derived Reporter Gene for Positron Emission Tomography Imaging

UCLA researchers from the Department of Microbiology, Immunology, & Molecular Genetics have developed a novel reporter gene for positron emission tomography imaging of transplanted cells.

Lateral Cavity Acoustic Transducer (LCAT) for Shear-Induced Cell Transfection

Inventors at UC Irvine have developed a new technique for inserting materials, such as genes, into cells that uses a lateral cavity acoustic transducer (LCAT).  This technology is efficient, does not result in lasting damage to cells, and the device is portable.

Ocular Therapeutics Using Stem Cell Microvesicles

Researchers in the UCLA Department of Ophthalmology have invented a method of using human embryonic stem cell microvesicles (hESMVs) to induce the regenerative capacity of several tissues, in particular, the ability to induce reconstruction of diseased retinas.

Novel Tau Aggregation Inhibitor Peptide

UCLA researchers from the Department of Chemistry and Biochemistry have developed a novel approach to inhibit the aggregation of tau proteins in the brain, which is linked to over 20 dementias including Alzheimer’s Disease and Chronic Traumatic Encephalopathy.

Method to Direct the Reciprocal Interactions Between the Ureteric Bud and the Metanephric Mesenchyme

Researchers at UCLA have developed an approach to construct an embryonic kidney in vitro for the treatment of end stage renal disease.

Gene Repair For Hemophilia A

Researchers at UCLA from the Departments of Medicine and Urology have developed a treatment for hemophilia A that utilizes non-viral gene editing technologies for ex vivo repair of the mutation in the gene F8.

A Codon-Optimized Lentiviral Vector For Stem Cell Reprogramming

UCLA researchers in the Department of Medicine and the Department of Surgery have developed a novel lentiviral vector that expresses a codon-optimized sequence of a T cell receptor (TCR) specific for the cancer-testis antigen NY-ESO-1 as well as a positron emission tomography (PET) reporter and suicide gene HSV1-sr39tk for use in adoptive T cell therapy for cancer treatment.

Transient Expression Of BCL-2 To Ameliorate Cytotoxicity Of Gene Modification Reagents In Stem Cells

Researchers at the UCLA Department of Microbiology, Immunology and Molecular Genetics have developed methods for efficient gene editing in stem cells by increasing the level of apoptosis regulator BCL-2.

Ex Vivo Maintenance and Expansion Of Hematopoietic Stem Cells

Hematopoietic stem cell (HSC) transplants are used to treat patients with a broad spectrum of hematological malignancies, immune disorders and genetic blood diseases. Unfortunately, even after decades of use and research, there is a significant shortage of histocompatible HSCs available for transplants. Transplanting larger numbers of HSCs increases the likelihood and speed of successful engraftment, which can reduce the risk of complications such as anemia and infection, and more effectively treat underlying disease. The inability to efficiently maintain adult HSCs ex vivo is also a significant barrier for the wider development and implementation of gene therapies for diverse blood diseases and a major obstacle for engineering HSC derived cellular products for immunotherapy. One approach to overcome this challenge is to develop a means to maintain and expand HSCs in culture. Unfortunately, there is no well-defined reproducible means to maintain or expand HSCs. Even short culture times in optimized conditions are deleterious to HSCs. Ex vivo HSC maintenance and expansion could significantly enhance their clinical utility in a wide range of human diseases, providing a new platform for testing drugs, enabling more efficient gene editing within stem cells, and developing into a widely-used tool for the research community.

Bispecific, Or-Gate Chimeric Antigen Receptor Responsive To CD19 And CD20

UCLA research have developed a CD19‐OR‐CD20 chimeric antigen receptor (CAR) that can trigger T‐cell activation upon detection of CD19 or CD20.

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