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Therapeutic Approach To Prevent Or Alleviate Drug-, Noise- And Age-Related Hearing Loss

UCLA researchers in the Department of Head and Neck Surgery have developed a novel therapeutic approach to treating hearing loss using inflammation-resolving molecules.

Lentiviral Vectors Expressing FoxP3 Or IL-10 In Hematopoietic Stem Cells To Treat Immune Deficiencies And Auto-Immune Diseases

The Kohn group at UCLA has created lentiviral vectors expressing FoxP3 or IL-10 for transduction into patient hematopoietic stem cells (HSC). Successful transplantation of autologous HSCs would allow for treatment of a number of auto-immune and auto-inflammatory diseases, while avoiding complications associated with allogenic transplants or autologous T cell therapies.

Method to Develop a Stable Pluripotent Bovine Embryonic Stem Cell Line

Researchers at the University of California, Davis have developed a method to produce stable pluripotent bovine embryonic stem cells.

Scalable Lipid Bilayer Microfluidics for High-Throughput Gene Editing

Researchers led by Paul Weiss from the Department of Chemistry and Pediatrics at UCLA have created a new microfluidic device for high-throughput gene editing of cells.

Gene Editing of Monogenic Disorders in Human Hematopoietic Stem Cells

Researchers at the UCLA Department of Microbiology, Immunology & Molecular Genetics have developed novel methods to achieve efficient, precise gene integration and effective expression of cDNA cassettes to express normal versions of genes in hematopoietic stem cells.

System and Methods for Efficient Collection of Single Cells and Colonies of Cells and Fast Generation of Stable Transfectants

A plate manufactured to enable samples of cells, microorganisms, proteins, DNA, biomolecules, transfectants, and other biological media to be positioned at specific sites. Some or all of the sites are built from removable material so that samples may be isolated.

A Novel Method and Protocol to Induce Pluripotent Stem Cells Toward Astrocyte Differentiation

Rett syndrome (RTT) is a devastating disease that affects 1 in every 10,000 children born in the United States, primarily females. RTT patients undergo apparently normal development until 6-18 months of age, followed by impaired motor function, stagnation and then regression of developmental skills, hypotonia, seizures and a spectrum of autistic behaviors. Rett syndrome is a rare disease that shares certain pathways with major developmental disorders such as autism and schizophrenia, increasing the potential impact. There is no cure for Rett syndrome and the animal model does not entirely recapitulate the human disease. Thus, having the possibility to screen drugs directly in human neurons is a major milestone.

Development of Human-Derived Cerebral Organoids with Network Oscillations

Historically, the understanding of the development and pathophysiology of the human brain has been studied by examination of post-mortem and diseased specimens in conjunction with non-human primates and mouse models. The understanding of complex biological mechanisms is driven by advancement of techniques and new model systems and recent advances in stem technologies have contributed to the advancement of our knowledge of human neural development. Moreover, the reprograming of human somatic cells into induced pluripotent stem cells (iPSCs) which can be redirected to a specific cell fate has led to a breakthrough in neurobiology research. These findings have led to the generation of human brain organoids from IPSCs.

The Use Of Parathyroid Gland Cells And Their Secreted Factors To Promote Islet Beta Cell Engraftments

This invention identifies a novel method to increases survival of transplanted islets in extra-hepatic sites (subcutaneous and intramuscular) through co-transplantation of pancreatic islet beta cells together with parathyroid glands (PTGs). This treatment can successfully be used for patients with type I diabetes.

Inhibition of Pyruvate Oxidation to Promote Hair Growth

UCLA researchers in the departments of Molecular, Cell & Developmental Biology and Biological Chemistry have elucidated a novel mechanism by which pyruvate oxidation can be inhibited in order to promote hair growth.

Methods for Enhancing Cell Populations for Articular Cartilage Repair

Cartilage lesion treatments require expanding cells from healthy donor cartilage which have limited availability and restricted potential to produce cartilage. This invention overcomes these challenges, presenting chemical and physical methods for enhancing cell populations capable of producing neocartilage. According to a 2015 global market report, tissue engineering technologies are expected to reach over 94B USD by 2022.

Methods for Producing Neocartilage with Functional Potential

Cell expansion for cartilage tissue production usually leads to loss of the potential to produce cartilage, which impedes uses for cartilage repair. This invention features methods and systems for producing highly expanded primary cells to construct functional neocartilage and other neotissue. According to a 2015 global market report, tissue engineering technologies are expected to reach over 94B USD by 2022.

Preconditioned Human Mesenchymal Stem Cells (hMSCs) for Musculoskeletal Tissue Repair

Researchers at the University of California, Davis have developed a preconditioning treatment and delivery method for human mesenchymal stem cell (hMSC) for enhanced bone healing.

Soluble Epoxide Hydrolase-Conditioned Stem Cells for Cardiac Cell-Based Therapy

Researchers at the University of California, Davis, have adapted novel soluble epoxide hydrolase (sEHI) inhibitors as adjuvant treatment in cardiac cell-based therapy to improve the survival and engraftment of stem cells by pre-conditioning with the sEHI in vitro.

A Method for Making a Human Alzheimer’s Disease Neuronal Model Using Purified ApoE-HDL

Alzheimer's disease (AD) is a common neurodegenerative disease and the most common cause of dementia. Alzheimer’s disease is defined post-mortem by the increased presence of amyloid plaques and neurofibrillary tangles (NFTs) in the brain. Amyloid plaques are extracellular deposits consisting primarily of amyloid-ß (Aß) peptides, and NFTs are intraneuronal aggregations of hyperphosphorylated tau, a microtubule-associated protein involved in microtubule stabilization. The discovery of new drugs for treating Alzheimer’s disease is currently limited by difficulties in obtaining live neurons from patients and the inability to accurately model Alzheimer’s disease. Animal models of Alzheimer’s disease have been developed, however, these animal models do not completely mimic true human disease, and none of these animal models are neuronal models of the disease. There is a need to develop a human neuronal model that more accurately mimics true human Alzheimer’s disease, and then use such a model for Alzheimer’s disease drug discovery and research.

Intracellular-Ligand-Responsive Cytotoxic Molecules For Selective T-Cell Mediated Cell Killing

UCLA researchers in the Department of Chemical and Biomolecular Engineering have developed a novel immunotherapeutic strategy that uses a selectively-activated cytotoxic molecule to enable tumor-specific T cell-mediated killing.

Inhibition Of Protein Tyrosine Phosphatase - Sigma For Hematopoietic Regeneration

UCLA Researchers have identified a novel pharmacological target for hematopoietic stem cell regeneration. They have developed small molecule inhibitors against the target and shown that the inhibitors cause rapid stem cell regeneration.

Xenobiotic-Free Culture System To Expand Human Limbal Stem Cells

UCLA researchers in the Departments of Opthalmology have developed a xenobiotic-free manufacturing process to produce transplantable human limbal stem cells for use in treating limbal stem cell deficiency.

Microchambers With Solid-State Phosphorescent Sensor For Measuring Single Mitochondrial Respiration

The invention is a miniaturized device that assays the respiration of a single mitochondrion. Through a novel approach for measuring oxygen consumption rate, the device provides information on cell and tissue mitochondrial functional. This data is relevant for understanding human conditions associated with mitochondrial dysfunction, such as Alzheimer’s Disease and cancer.

Induction of Corneal Endothelial Cells

Ocular degenerative diseases including age-related macular degeneration (AMD), retinitis pigmentosa, glaucoma, and corneal endothelial dystrophy (CED) cause irreversible vision loss and affect millions of people worldwide. Currently, there is no effective drug intervention. Grafting healthy eye cells to replenish the diseased tissues such as retina represents a promising therapeutic approach. However, previous attempts at using primary human eye cells have met with limited success due to the limited expansion capacity and differentiation potential of adult progenitors or difficulty of obtaining sufficient human fetal retinal progenitors, and possible ethical concerns. Human pluripotent stem cells (PSCs), including human embryonic stem cells (ESCs), and induced pluripotent stem cells (iPSCs) represent promising renewable donor sources for cell-based replacement therapy. Nevertheless, PSCs themselves are not suitable for direct transplantation in clinical applications due to their tendency to form teratomas and low efficiency in repopulating host tissues with desirable reprogrammed cell types in vivo. While the advancement of clinical trials of hESC-derived RPE transplants for treatment of patients with Stargardt's macular dystrophy and AMD is encouraging to the field, there is a great need for methods of generating unlimited other specialized eye cells effectively in vitro for treating blindness due to the loss of photoreceptors, RGCs and CECs. Therefore, there is a major interest in development of in vitro expandable cell sources for engineering corneal endothelium.

Small Molecule Generation of Multinucleated and Striated Myofibers from Human Pluripotent Stem Cells Equivalent to Adult Skeletal Muscle

Researchers in the UCLA Department of Microbiology, Immunology and Molecular Genetics have developed a novel means of generating adult skeletal muscle-equivalent myofibers from human pluripotent stem cells.

CRISPR/Cas9 Mediated Genome Editing For Duchenne Muscular Dystrophy

UCLA researchers in the Department of Microbiology, Immunology & Molecular Genetics have developed a method to permanently correct the out-of-frame dystrophin gene in patient cell models of Duchenne Muscular Dystrophy (DMD)

Induced Pluripotent Stem Cell-Derived Glial Enriched Progenitor Cells For The Treatment Of White Matter Stroke

UCLA researchers in the Department of Neurology and the Department of Molecular, Cell & Developmental Biology have developed novel therapies for cerebral ischemic injuries, including white matter stroke, using glial-enriched progenitor cells.

Method for Making Electrophysiologically Mature Cardiomyocytes Using a Non-Genetic Approach

Researchers at the University of California, Davis have developed a novel method for inducing maturity in cardiomyocytes by altering cell phenotype by mimicking the pacing environment.

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. 

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