Patent classifications
A01K2207/20
GENE THERAPIES FOR LYSOSOMAL DISORDERS
The disclosure relates to compositions and methods for treatment of diseases associated with aberrant lysosomal function, such as Parkinson's disease and Gaucher disease. The disclosure provides expression constructs comprising a transgene encoding beta-glucocerebrosidase, an inhibitory RNA targeting alpha-Synuclein, or a combination of the foregoing. The disclosure further provides methods of treating Gaucher disease, Parkinson's disease or other synucleinopathies by administering such expression constructs to a subject in need thereof.
GENE THERAPIES FOR LYSOSOMAL DISORDERS
The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease (PD) and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof alone or in combination with one or more PD-associated genes. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.
METHOD FOR IMPROVING IMMUNITY IN SHRIMPS
The invention discloses a method for improving immunity in shrimps, by administering an extract of cocoa rind to a shrimp body to improve immunity of the shrimp body. The extract of cocoa rind is obtained by extracting a dried sample of cocoa rind by an aqueous ethanol solution with a concentration of ethanol being 90-98%. The dried sample of cocoa rind has a water content of 2-5%.
GENE THERAPIES FOR LYSOSOMAL DISORDERS
The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease (PD) and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof alone or in combination with one or more PD-associated genes. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.
COMPOSITION FOR PREVENTING OR TREATING LIVER FIBROSIS, CONTAINING EXOSOME OR EXOSOME-DERIVED RIBONUCLEIC ACID
The present invention relates to a composition or cell therapeutic agent for preventing or treating liver fibrosis, containing an exosome or exosome-derived ribonucleic acid. The exosome or ribonucleic acid derived therefrom, of the present invention, has effects of inhibiting activities of hepatic stellate cells and Kupffer cells and reducing the expression of -SMA and inhibits the progression of liver fibrosis by inhibiting the deposition of collagen, thereby being effectively usable as a cell therapeutic agent for the prevention or treatment of liver fibrosis.
Small Molecule-Induced Method for Directly Reprogramming Human Fibroblasts into Liver Cells
This disclosure relates to a method for direct reprogramming (transdifferentiation) of human fibroblasts into hepatocytes by small molecules. A method and a small molecule composition for direct reprogramming (transdifferentiation) of human fibroblasts into hepatocytes based on a mechanism of chemically induced cell direct reprogramming are disclosed. The small molecule composition can be developed into drug or prodrugs for the treatment of hepatic fibrosis (Cirrhosis), pulmonary fibrosis and fibrosis diseases of other organ or tissue in human. This disclosure also discloses a cell transdifferentiation medium and a reagent prepared by the small molecule composition.
GENE THERAPIES FOR LYSOSOMAL DISORDERS
The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease (PD) and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof alone or in combination with one or more PD-associated genes. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.
Compositions for Treating Pathological Calcification Conditions, and Methods Using Same
The present invention includes compositions and methods for treating diseases or disorders associated with pathological calcification or pathological ossification. In certain embodiments, the diseases or disorders are selected from the group consisting of Generalized Arterial Calcification of Infancy (GACI), Idiopathic Infantile Arterial Calcification (IIAC), Ossification of the Posterior Longitudinal Ligament (OPLL), hypophosphatemic rickets, osteoarthritis, calcification of atherosclerotic plaques, PXE, hereditary and non-hereditary forms of osteoarthritis, ankylosing spondylitis, hardening of the arteries occurring with aging, calciphylaxis resulting from end stage renal disease and progeria.
Methods of agent delivery into eggs and embryos of egg-producing aquatic animals for drug screening, agent toxicity assay and production of infertile fish
Methods are provided for delivery of at least one agent into egg(s) from an egg-producing aquatic animal including contacting fertilized or unfertilized egg(s) from said egg-producing aquatic animal with the at least one agent in the presence of a guanidine-containing compound capable of enhancing the permeability of the chorion of the egg(s). Methods are provided for the drug screening and compound toxicity assays. Methods are also provided for the production of reproductively sterile fish and aquatic animals for aquaculture, the aquarium trade, and control of invasive species are described. The methods include disruption of gonadal development through the administration of agents that lead to the failure of fertile gonadal development. Agents may be delivered to the eggs directly before the fertilization or post fertilization by contacting eggs in an immersion medium including the agent of interest.
INHIBITORS OF DIHYDROOROTATE DEHYDROGENASE
Embodiments of the present invention are directed to methods for treatment of melanoma using an inhibitor of dihydroorotate dehydrogenase (DHODH) and to combination therapies that involve administering to a subject an inhibitor of oncogenic BRAF (e.g. BRAF(V600E)), as well as an inhibitor of dihydroorotate dehydrogenase (DHODH). Assays for identifying compounds useful for the treatment of melanoma are also provided. The methods comprise screening for compounds or agents that inhibit neural crest progenitor formation in a zebra fish model of melanoma.