12 results about "Progeria" patented technology

The application discloses application of a novel peptide UHP in preparation of a medicine for treating and / or preventing delayed aging, and belongs to the technical field of biological medicines.The novel peptide UHP is composed of 37 L-type amino acids including a transmembrane peptide HIV-TAT.It is proved by cell experiments that the novel peptide UHP significantly improves the aging phenotype of a HGPS cell model simulated by overexpression of progerin and a replicative senescent human normal fibroblast cell, reduces the SA-beta-gal positive cell proportion and the expression of SASP factors, and increases the expression levels of cell proliferation markers Ki67, Lamin B1 and Cyclin A2.It is proved by animal experiments that the novel peptide UHP can improve the progeria phenotype, body weight and exercise capacity of a progeria mouse, relieve fibrosis in multiple tissues, improve the skin aging phenotype, and prolong the survival period of the progeria mouse, thereby providing a new medicine selection for clinical treatment of aging-related diseases.

The present disclosure provides methods and compositions for treating progeria or neurodegenerative diseases, particularly neurodegenerative diseases associated with amyloid deposition and neuronal death, such as Alzheimer's disease. Accordingly, aspects of the present disclosure relate to methods for treating progeria in a subject in need thereof, comprising administering to the subject a TERT-activating therapeutic agent. Other aspects relate to methods for treating a neurodegenerative disease in a subject, comprising administering to the subject a TERT-activating therapeutic agent.

The application discloses a method for purifying and chromatographing and mass spectrometrically identifying deoxyguanosine or 3-hydroxybenzoic acid and application. After metabolites are extracted by a methanol method, UPLC-MS / MS-ultra high performance liquid chromatography and ABSCIEX QTRAP 5500 triple quadrupole tandem linear ion trap mass spectrometry are used to distinguish the metabolites by using liquid chromatography and mass spectrometry. The MS technology provides the ability to detect and quantify a large number of metabolites. The method optimizes the purification and mass spectrometric identification of the progeria markers in blood, identifies the structures of the differential markers deoxyguanosine and 3-hydroxybenzoic acid in blood by using the mass spectrometric method, and distinguishes the difficult-to-distinguish differential markers. The method for purifying and mass spectrometrically identifying the progeria differential markers in blood is simple and easy to operate, has small experimental dosage, good stability, is fast and accurate, and can make up for the deficiency in searching for the blood differential markers.

The present invention relates to a method for identifying the aging degree of a subject or a tissue or organ thereof, and for evaluating the effect of anti-aging interventions. Specifically, provided is a method for identifying the aging degree of a subject or a cell, tissue or organ thereof, or for evaluating the biological age of the subject or diagnosing premature aging disorders, which method predicts the aging status of a tissue or organ of a subject on the basis of the levels of DNA methylation, gene transcripts, proteins or metabolites. The present invention further relates to the use of metformin in the preparation of a drug for treating, preventing or delaying aging-related diseases or conditions in primates, preferably humans.

The invention belongs to the field of biological medicine, and relates to a preparation method of a premature senility animal model. The invention provides an RNA (Ribonucleic Acid), wherein the RNA is selected from any sequence shown in SEQ ID NO: 1-3. The RNA is sgRNA, the sgRNA can specifically target the Lmna c.1827 site, and the Lmna c.1827 site is edited by combining a base editing protein, so that the premature senility animal model is successfully obtained. And a powerful model animal model tool is provided for deeply researching the pathogenesis of the premature senility and finding a potential treatment method.

Disclosed herein are products, methods, and uses for treating, ameliorating, delaying the progression of, and / or preventing a disease or disorder associated with expression of an aberrant lamin A (LMNA) gene or progerin gene. Such disease or disorder includes, but is not limited to, a laminopathy, progeroid syndrome, progeria, or aging disorder resulting from the aberrant expression of LMNA or progerin. In some instances, the progeria is Hutchinson-Gilford progeria syndrome (HGPS). In some instances, the disease or disorder associated with the expression of progerin is premature aging or natural aging including, but not limited to, atherosclerosis, alopecia, osteoporosis, cardiovascular disease, skin abnormalities, fat storage, stroke, myocardial infarction, stroke, heart failure, muscle wasting, muscle weakness, myotonia, skeletal muscle problems, abnormalities of the retina, hip weakness, abdominal muscle weakness, joint and spinal abnormalities, lower leg weakness, shoulder weakness, hearing loss, and / or tissue inflammation. More particularly, disclosed herein are RNA interference-based products, methods, and uses for inhibiting or downregulating the expression of progerin. Even more particularly, the disclosure provides guide RNA and a CRISPR endonuclease for inhibiting or downregulating the expression of progerin and methods of using said guide RNA and a CRISPR endonuclease to correct a mutation in the LMNA gene and inhibit or downregulate progerin expression in cells and / or in cells of a subject having a condition resulting from the expression of progerin including, but not limited to, HGPS or progeria, an HGPS-like condition affecting LMNA mutations that affect exon 11 splicing, or a condition resulting from the expression of progerin.

Methods for the treatment and prevention of laminopathies and diseases characterised by hyperlipidemia through LING complex inhibition are disclosed. In particular, LING complex disruption by expression of dominant-negative LING complex proteins alleviates pathophysiology in Lmna mutation-associated muscular dystrophy, progeria, and dilated cardiomyopathy. In addition, LING complex disruption by expression of dominant-negative LING complex proteins also alleviates pathophysiology in mouse models of atherosclerosis and familial hypercholesterolemia.

The application discloses a gene editing system for constructing a progeria model pig nuclear transfer donor cell with a ZMPSTE24 gene mutation and application thereof. The application provides a kit comprising ZMPSTE24-gRNA2 shown in SEQ ID NO: 16, ZMPSTE24-gRNA4 shown in SEQ ID NO: 17 and NCN protein. The application also provides a method for preparing a recombinant pig cell: co-transfecting ZMPSTE24-gRNA2, ZMPSTE24-gRNA4 and NCN protein into a pig cell to obtain a recombinant pig cell. The recombinant pig cell is a recombinant cell with a ZMPSTE24 gene mutation. The kit is used for: preparing a recombinant pig cell; preparing a progeria model pig; preparing a progeria cell model or a progeria tissue model or a progeria organ model. The application has great application value for the research and development of progeria drugs and the revelation of the pathogenesis of the disease.

Disclosed herein are products, methods, and uses for treating, ameliorating, delaying the progression of, and / or preventing a disease or disorder associated with expression of an aberrant lamin A (LMNA) gene or progerin gene. Such disease or disorder includes, but is not limited to, a laminopathy, progeroid syndrome, progeria, or aging disorder resulting from the aberrant expression of LMNA or progerin. In some instances, the progeria is Hutchinson-Gilford progeria syndrome (HGPS). In some instances, the disease or disorder associated with the expression of progerin is premature aging or natural aging including, but not limited to, atherosclerosis, alopecia, osteoporosis, cardiovascular disease, skin abnormalities, fat storage, stroke, myocardial infarction, stroke, heart failure, muscle wasting, muscle weakness, myotonia, skeletal muscle problems, abnormalities of the retina, hip weakness, abdominal muscle weakness, joint and spinal abnormalities, lower leg weakness, shoulder weakness, hearing loss, and / or tissue inflammation. More particularly, disclosed herein are RNA interference-based products, methods, and uses for inhibiting or downregulating the expression of progerin. Even more particularly, the disclosure provides guide RNA and a CRISPR endonuclease for inhibiting or downregulating the expression of progerin and methods of using said guide RNA and a CRISPR endonuclease to correct a mutation in the LMNA gene and inhibit or downregulate progerin expression in cells and / or in cells of a subject having a condition resulting from the expression of progerin including, but not limited to, HGPS or progeria, an HGPS-like condition affecting LMNA mutations that affect exon 11 splicing, or a condition resulting from the expression of progerin.

The present disclosure relates to vectors, compositions, pharmaceutical compositions and kits providing brain cell-specific expression of reprogramming genes, such as Yamanaka factors Oct4, Sox2, Klf4 and c-Myc (OSKM). Methods and uses including the same for treating Alzheimer's disease and progeria by brain cell-specific expression of reprogramming genes, such as OSKM, are also provided.

The application discloses a Caenorhabditis elegans disease model and a construction method and application thereof, and particularly relates to a Caenorhabditis elegans disease model for neurodegenerative diseases, which comprises Caenorhabditis elegans, wherein a fragment of NOTCH2NLC GGC is overexpressed in the Caenorhabditis elegans, the sequence of the fragment is shown as SEQ ID NO. 1, and the neurodegenerative diseases include NIID. The application overcomes the difficulty of cloning GGC repeat sequences, successfully overexpresses 100 GGC repeat sequences of a human NOTCH2NLC gene in Caenorhabditis elegans, and shows similar behavior disorders and premature aging symptoms to human NIID diseases. The model is a good animal model for studying the pathogenic mechanism of GGC repeat sequences of the NOTCH2NLC gene and drug screening.

The application discloses a polypeptide UPCP and application thereof in preparation of anti-aging products. The polypeptide UPCP provided by the application has an amino acid sequence of sequence 4 in the sequence list. The polypeptide is applied in preparation of products with any one of the following functions: 1) treating children's premature aging; 2) delaying physiological aging of mammals or cells thereof; 3) improving premature aging of mammals; the improvement of premature aging of mammals is embodied in delaying premature aging of mammals; 4) improving body weight of premature aging animals; the improvement of body weight of premature aging animals is embodied in increasing body weight of premature aging animals; 5) relieving motor dysfunction of premature aging animals; 6) prolonging survival period of premature aging animals, etc. The application develops a specific peptide (UPCP) which can inhibit HGPS cell aging, improve aging characteristics of HGPS premature aging mice and prolong survival period, and provides a new strategy for treatment of HGPS.