173 results about "Human disease" patented technology

The invention belongs to the technical field of biology, and particularly relates to an SLC44A1 gene and / or SLC44A5 gene knockout animal model as well as a construction method and application thereof. The construction method of the animal model comprises the following steps: deleting exon regions from the fourth exon region to the thirteenth exon region of the SLC44A1 gene and / or the second exon region of the SLC44A5 gene in mouse brain oligodendroglia cells in a high-efficiency manner by utilizing gene editing and a Cre-loxP recombinase system, so as to realize the knockout of a target gene. The animal model constructed by the invention can be used for researching the regulation mechanism of the choline transporters (SLC44A1 and SLC44A5) on myelin sheath formation, deeply knowing the biological functions of the choline transporters (SLC44A1 and SLC44A5), simulating the occurrence and development processes of human related diseases, revealing the pathogenesis of the related diseases, providing a new thought for prevention and treatment of brain myelin sheath dysplasia, and providing a new foundation for the prevention and treatment of brain myelin sheath dysplasia. And an effective drug or a treatment mode aiming at the disease is screened by utilizing a gene knockout animal model, so that a new choice is provided for clinical treatment of the disease.

Isolated or recombinant EphA5 or GRP78 targeting antibodies are provided. In some cases, antibodies of the embodiments can be used for the detection, diagnosis and / or therapeutic treatment of human diseases, such as cancer. A method of rapidly identifying antibodies or antibody fragments for the treatment of cancer using a combination of in vitro and in vivo methodologies is also provided.

The invention relates to the field of immunology, and discloses a polypeptide and a kit for distinguishing bovine and sheep brucella natural infection from a vaccine immune antibody. Polypeptides as shown in SEQ ID NO: 1 and SEQ ID NO: 2 are artificially synthesized and used as antigens to coat an elisa plate and a nitrocellulose membrane, and according to response characteristics of antibody level, identification and detection of natural infection and vaccine immunity of cattle and sheep populations are realized. The peptide fragment and the detection kit provided by the invention can make up the blank of identifying bovine and sheep brucella natural infection and immune antibodies. The method solves the problem that the antibody induced by the attenuated live vaccine and the naturally infected antibody are difficult to distinguish, provides a scientific basis for quarantine and purification of Brucella, and provides a technical guarantee for preventing human diseases and animals, advancing the mouth and protecting the body health of people.

This invention provides antibodies or functional antibody fragments or probes thereof targeting a unique set of antigens recognized in cancer. The invention comprises a nucleotide sequence derived from the L2A5 monoclonal antibody. The antibodies or functional antibody fragments or probes thereof include variable heavy chain domains and variable light chain domains having the amino acid sequences provided herein. The DNA / amino acid sequence binding is unique and has never been previously described. The invention further provides antibodies or functional antibody fragments or conjugates or recombinant proteins useful for the detection, treatment, and prevention of human diseases, including cancer.

Disclosed herein are systems for LNP-mediated delivery of circular single-stranded DNA (cssDNA). The systems can be used to facilitate integration of the cssDNA into a cellular genome for a variety of purposes, including treatment of human diseases.

Disclosed herein are methods for improving detection and monitoring of human diseases. The methods can be used to provide spatial and / or developmental localization of the source of each differential mutation within the body. The methods can also be used to generate a mutation map of a subject. And the mutation map can be used to monitoring state(s) of health of one or more tissues of a subject.

The invention discloses a method for establishing a hemophagocytic syndrome in-vitro cell disease model, and belongs to the technical field of biology. Based on human PBMC construction, the reaction of a human immune system can be truly reflected, experimental result deviation caused by species difference in an animal model is avoided, and a more accurate model basis is provided for research of human diseases. In addition to T cell over-activation, the HLH model of the present application has a clear key feature of NK cell reduction, which is HLH, accompanied by up-regulation of cytokines such as interferon-gamma and IL-10. The establishment process of the model is rapid and efficient, the experimental period is remarkably shortened, a large number of samples or drugs can be rapidly screened, the research efficiency is improved, and the conversion process from basic research to clinical application is accelerated. The method can be used for researching various mechanisms related to the HLH, is beneficial to more comprehensive understanding of pathogenesis of the HLH, and provides a theoretical basis for developing preliminary screening of targeted drugs.

The invention discloses a medicinal arc scraping therapy device prepared from a Chinese herbal medicine composition and a preparation method of the medicinal arc scraping therapy device, the medicinal arc scraping therapy device prepared from a plurality of different negative and positive Chinese herbal medicine raw material compositions is used for carrying out physical therapy on different human diseased organs in the scraping therapy process, and the medicinal arc scraping therapy device has the characteristics that the medicinal arc scraping therapy device takes effect immediately, has a remarkable effect and can control the treatment course.

Disclosed herein are engineered LSR sequences, compositions, and uses including in genome-editing systems and therapeutic compositions. The LSRs can facilitate nucleic acid recombination at particular attachment sites, using suitable donor sequences. The disclosed LSRs may be used to site-specifically recombine or integrate nucleic acid sequences for a variety of purposes, including treatment of human diseases.

The present disclosure provides methods of amplifying tissue samples derived from humans for purposes of treating certain diseases. The method comprises: isolating a small amount of associated tissue by biopsy or other tissue acquisition methods; the tissue is amplified in vitro, and meanwhile, natural functions are reserved; and re-implanting the amplified tissue to alleviate human disease. One example includes ex vivo expansion of human pancreatic islet tissue and reimplantation of the expanded cells into an individual with Type 1 diabetes for treatment and long term relief or cure of disease.

Compounds of formula (I) as defined in the present application, or pharmaceutically acceptable salts thereof, are potent modulators of human IL-17 activity, and thus are beneficial for the treatment and / or prevention of various human diseases, including inflammation and autoimmune disorders.

The present invention relates to a method of treating PIK3CA-Related Overgrowth Spectrum (PROS) more particularly, Congenital, Lipomatous, Overgrowth, Vascular Malformations, Epidermal Nevi and Spinal / Skeletal Anomalies and / or Scoliosis (CLOVES) syndrome. To date, there are no specific treatments for patients and no animal models of PROS to better understand the physiopathology of the disorder. Inventors developed a genetic mouse model of PROS that recapitulates the human disease and demonstrated the efficacy of BYL719. Based on these results they treated two patients, one adult and one child, with severe CLOVES syndrome using BYL719. The drug had a robust efficiency on disease in the two patients inducing quick recovery of all affected organs. Thus, the invention relates to a method of treating PROS in a subject in need thereof comprising the step of administrating the subject with a therapeutically effective amount of BYL719.

The present disclosure provides transgenic mice for use as mouse models, and methods related thereto. The transgenic mice described herein comprise a humanized mouse telomerase (hmTert) gene included in a background mouse germline that provides shortened telomeres capable of reproducing humans and various human disease states.

Provided are a novel bispecific antigen-binding antibody, an antigen-binding fragment thereof, and a Trop2 * HER2 bispecific antibody drug conjugate (Trop2 * HER2 Abs ADC) which comprise a Trop2-binding arm and an HER2-binding arm, are capable of simultaneously specifically binding to HER2 and Trop2, have a high binding affinity to HER2 and Trop2, can be endocytosed by cells expressing HER2 and / or Trop2, and can be used as an antigen-binding fragment of a Trop2 * HER2 bispecific antibody drug conjugate. Endocytosis of the two binding arms L has a synergistic effect; the Trop2 * HER2 Abs ADC can inhibit the growth of tumor cells expressing HER2 and / or Trop2 in vitro and in vivo, and the bispecific antibody can be used for preventing and treating human diseases related to HER2 and / or Trop2, such as cancers.

Methods of treating or preventing a disease resulting from a respiratory syncytial virus (RSV) infection in a human subject is disclosed. The methods disclosed include administering to the human subject a compound of Formula (I), or pharmaceutical composition including a compound of Formula (I). In certain embodiments, the compound is celgosivir.

Cyclopropyl N-[(S)-[3-[1-(2,2-difluoropropylcarbamoyl)-3-hydroxy-3-methyl- cyclobutyl]imidazo[1,2-b][1,2,4]triazin-6-yl]-[4- (trifluoromethyl)cyclohexyl]methyl]carbamate, or a pharmaceutically acceptable salt thereof, being a potent modulator of human IL-17 activity, is accordingly of benefit in the treatment and / or prevention of various human ailments, including inflammatory and autoimmune disorders.

Embodiments are directed to novel GPR52 activators. In particular, a series of novel 1-(pyrimidin-4-yl)indoline-4-carboxamide analogs that have been identified as potent and selective GPR52 agonists. The optimized GPR52 agonist that, for example, can be used as a valuable pharmacological tool or a drug candidate for investigating the physiological and therapeutic potential of GPR52 activation for various human diseases.

The present invention relates to a method of treating PIK3CA-Related Overgrowth Spectrum (PROS) more particularly, Congenital, Lipomatous, Overgrowth, Vascular Malformations, Epidermal Nevi and Spinal / Skeletal Anomalies and / or Scoliosis (CLOVES) syndrome. To date, there are no specific treatments for patients and no animal models of PROS to better understand the physiopathology of the disorder. Inventors developed a genetic mouse model of PROS that recapitulates the human disease and demonstrated the efficacy of BYL719. Based on these results they treated two patients, one adult and one child, with severe CLOVES syndrome using BYL719. The drug had a robust efficiency on disease in the two patients inducing quick recovery of all affected organs. Thus, the invention relates to a method of treating PROS in a subject in need thereof comprising the step of administrating the subject with a therapeutically effective amount of BYL719.

N-[(S)-[3-[1-(2,2-difluoropropylcarbamoyl)-3-hydroxy-3-methyl- cyclobutyl]imidazo[1,2-b][1,2,4]triazin-6-yl]-[4-(trifluoromethyl)cyclohexyl]methyl]-4-5 methyl-1,2,5-oxadiazole-3-carboxamide, or a pharmaceutically acceptable salt and / or solvate thereof, being a potent modulator of human IL-17 activity, is accordingly of benefit in the treatment and / or prevention of various human ailments, including inflammatory and autoimmune disorders.

An effective treatment method for a broad variety of diseases in both animals and humans is disclosed. The method includes combining one or more vaccines with a treatment compound to enhance vaccine efficacy. The disclosed treatment compound does not act directly on the pathogen, and thus the organisms cannot readily develop resistance to the treatment. When a compound such as, but not limited to, the disclosed compound is used in conjunction with one or more vaccines, a synergistic effect is realized. The suggested compound is derived from a lipopolysaccharide (LPS) of gram negative bacteria. The treatment compound is combined with one or more appropriate vaccines and is administered early in the life of an animal to achieve a synergistic effect compared with the use of the treatment compound or the vaccine alone.

Pharmaceutical compositions and methods of using these compositions are disclosed that show efficacy in the treatment / amelioration of various human diseases, including cancer. The pharmaceutical composition comprises compounds that modulate the function of fatty-acid binding proteins that are used in combination with immunotherapy treatment that demonstrates synergistic efficacy to target and eliminate disease-associated antigens and / or cells.

Disclosed herein are C-mannoside compounds, compositions thereof and their application as pharmaceuticals for the treatment of human disease. Methods of inhibition of FimH activity in a human subject are also provided for the treatment of diseases such as bacterial infection, Crohn's disease (CD) and Inflammatory Bowel Disease (IBD).

A T cell receptor (TCR) plays a crucial role in adaptive immune response by recognizing specific antigens. In the context of cancer immunotherapy, TCR sequences may be engineered to target specific tumor antigens. In this study, hepatocellular carcinoma (HCC) was simulated in mice by binding to genetic alterations repeatedly observed in human diseases, and HCC-specific TCR was identified using single cell TCR-seq. The TCR convergence is observed in a mouse carrying HCC, indicating that the TCR specific to the HCC antigen initiates the expansion of CD4 + T cells. In order to further clarify the function of such HCC-specific TCRs, TCR expression vectors are designed and constructed, which can be used in a variety of in vitro and in vivo applications, including identification of specific peptide sequences for TCR recognition, manufacture of TCR engineered T cells, study of T cell function, and screening for potential immunotherapy. These findings are of great significance to the development of T cell-based cancer immunotherapies.

Provided herein are methods for the activation and expansion of genetically-modified Tcells, such as tumor infiltrating lymphocytes. In some cases, cells of the embodiments can be used for the therapeutic treatment of human diseases, such as cancer.

The present invention relates to antibodies which specifically bind to human IL1RAP and my also bind to cynomolgus monkey and / or mouse IL1RAP. The present invention also relates to the use of such antibodies to diagnose and treat human disease.

N-[(S)-(4, 4-difluorocyclohexyl) {3-[1-(2, 2-difluoropropylcarbamoyl)-3-hydroxy-3-methylcyclobutyl] imidazo [1, 2-b] [1, 2, 4] triazin-6-yl} methyl]-4-methyl-1, 2, 5-oxadiazole-3-carboxamide, or a pharmaceutically acceptable salt thereof, is a potent modulator of human IL-17 activity, and is therefore of benefit in the treatment and / or prevention of a variety of human diseases, such as, for example, human IL-17. Inflammatory and autoimmune disorders are included.

The present application provides a method for directional transfer of heterologous chromosomes, which comprises the following steps: artificially adding a functional intron containing a loxP sequence into a drug resistance gene, splitting the drug resistance gene into two parts with an overlapping region containing a loxP sequence, inserting the two parts of the drug resistance gene into target chromosomes of a donor species and a recipient species respectively (the two parts of the drug resistance gene undergo Cre / loxP site-specific recombination under the action of a Cre recombinase, and the drug resistance function is recovered), and realizing directional transfer of heterologous chromosomes by means of heterologous cell fusion. The method can be widely applied to gene function research, construction of human disease models, development of therapeutic antibodies, and pharmacological and pharmacodynamic tests.