90results about "Muscle proteins" patented technology

Described herein are muscle-specific targeting moieties and compositions including the muscle specific targeting motifs. Also described herein are uses of the muscle-specific targeting motifs and compositions including the muscle specific targeting moieties. In some embodiments, the muscle-specific targeting moieties and compositions including the muscle specific targeting moieties can be used to direct delivery of a cargo to a muscle cell.

Recombinant myotropic AAV rh.74 capsids are described herein. In addition, plasmids, viral vectors, dual vector systems, cells, and compositions comprising or encoding such recombinant capsids are further described. Such recombinant polynucleotides, plasmids, viral vectors, dual vector systems, cells, and compositions may be used to treat muscle diseases or disorders, including muscular dystrophies and cardiomyopathies.

The present invention provides antigen-binding molecules, specifically domain-engineered antibodies in which at least one constant region domain, CH1 / CL, is replaced with Titin T chain / Obscurin-O chain.

Recombinant myotropic AAV rh.74 capsids are described herein. In addition, plasmids, viral vectors, dual vector systems, cells, and compositions comprising or encoding such recombinant capsids are further described. Such recombinant polynucleotides, plasmids, viral vectors, dual vector systems, cells, and compositions may be used to treat muscle diseases or disorders, including muscular dystrophies and cardiomyopathies.

Nucleotide sequences including a micro-dystrophin gene are provided. The micro-dystrophin genes may be operatively linked to a regulatory cassette. Methods of treating a subject having, or at risk of developing, muscular dystrophy, sarcopenia, heart disease, or cachexia are also provided. The methods may include administering a pharmaceutical composition including the micro-dystrophin gene and a delivery vehicle to a subject. Further, the methods may include administering the pharmaceutical composition a subject having Duchenne muscular dystrophy or Becker muscular dystrophy.

In some embodiments, the present disclosure relates to nucleic acids, compositions and methods for delivering therapeutic genes to a subject. In some embodiments, the therapeutic genes are delivered via the use of viral vectors. In some embodiments, the viral vector is an adeno-associated virus. In some embodiments, the therapeutic genes are delivered to treat heart disease, injury or other disorders.

In several embodiments, the present disclosure relates to nucleic acids, compositions, and methods for the delivery of a therapeutic gene to a subject. In several embodiments, the therapeutic gene is through the use of a viral vector. In several embodiments, the viral vector is an adeno-associated virus. In several embodiments, the therapeutic gene is delivered to treat a cardiac disease, injury or other disorder.

The present invention discloses an umami peptide derived from Portunus trituberculatus and an application thereof, and belongs to the technical field of bioactive peptides. An amino acid sequence of the umami peptide derived from the Portunus trituberculatus is SGSFK, as shown in SEQ ID NO. 4. The umami peptide derived from the Portunus trituberculatus is used as or in preparation of an umami agent. The present invention integrates virtual enzymatic hydrolysis, screening and docking technologies to extract the umami peptide from the Portunus trituberculatus. Through sensory evaluation and electronic tongue analysis, it has been verified that the umami peptide SGSFK in the present invention is more umami than monosodium glutamate at a same concentration, and has an umami threshold of 0.125 mg / ml. The present invention provides a new raw material basis for development of umami agents and condiments, and has good application prospects and important application value.

A protein polymer and a production process therefor. The protein polymer is obtained by means of stimulating MSCs, lysing same, and separating and purifying same. The protein polymer has a strong capability to repair oxidative damage, and thus can reduce the range of cerebral infarction, improve neural functions, inhibit the level of neuroinflammation, and increase the number of surviving neurons.

The present disclosure provides a novel muscle-specific enhancer and promoter combination useful for achieving high and sustained expression in muscle tissue or muscle cells. The muscle-specific promoter element is derived from the desmin promoter. The muscle-specific enhancer element is derived from the desmin promoter and the muscle creatine kinase enhancer.

Provided is an application of a protein polymer in the preparation of a drug for treating autism. Research has found that protein polymers derived from mesenchymal stem cells stimulated under stress conditions, such as ultraviolet irradiation, have a certain therapeutic effect on autism, providing a possibility for the treatment of autism.

This invention describes a novel gene regulatory sequence containing the promoter and enhancer sequences of the human cardiac troponin T gene (TNNT2) that selectively induces expression in cardiomyocytes. This novel TNNT2 promoter / enhancer composition can be used to induce adeno-associated virus gene expression, construct cell-type-specific expression vectors, or perform cardiac-specific transgenesis. The use of this novel promoter / enhancer composition is demonstrated by the expression of mAKAP shRNA and a mAKAP-derived anchor-disrupting peptide useful for the treatment of heart failure.

The present invention relates to methods and materials for treating Limb Girdle Muscular Dystrophy R9 (LGMDR9) and Congenital Muscular Dystrophy 1C (MDC1C) using a bicistronic recombinant adeno-associated virus encoding fukutin related protein (FKRP) and follistatin (FST). This therapy is unique in that it can rebuild lost muscle strength at the same time that it prevents subsequent muscle disease from occurring.

Described herein are methods of treating muscular dystrophy comprising administering a recombinant AAV (rAAV) scAAVrh74.MHCK7.hSGCB vector, methods of expressing beta-sarcoglycan gene in a patient, pharmaceutical compositions comprising the rAAV, and methods of generating the rAAV.

The invention relates to methods and compositions for increasing introduction (e.g., transduction) efficiency. In particular, the invention relates to methods of increasing introduction (e.g., transduction) efficiency by contacting a host cell with a heterologous agent in the presence of a cell membrane fusion protein or a functional fragment or derivative thereof. The invention further relates to compositions that include a heterologous agent and a cell membrane fusion protein or a functional fragment or derivative thereof.

Several embodiments of the present disclosure related to selection of Kozak sequences that result in enhanced expression of a transgene of interest. Some embodiments relate to a Kozak sequence that is non-native to either the gene to be expressed and / or to a promoter driving expression of the gene. Some embodiments relate to synthetic Kozak sequences designed to yield enhanced expression of the gene in a tissue of interest, such as cardiac tissue.

Provided is a TRIM72 truncated protein, comprising the Coiled-PRYSPRY domain or PRYSPRY domain or its functional fragment of a TRIM72 protein. Further provided is the use of the TRIM72 truncated protein.

The present disclosure provides nucleic acids (including AAV expression cassettes), AAV vectors, and compositions for use in methods for treating and / or delaying the onset of diseases associated with mutations in genes such as PKP2, arrhythmogenic cardiomyopathy. Also provided herein are methods for treating and / or delaying the onset of arrhythmogenic cardiomyopathy.

The invention provides gene therapy vectors, such as adeno-associated virus (AAV) vectors, expressing a miniaturized human micro-dystrophin gene and method of using these vectors to express micro-dystrophin in skeletal muscle s including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and / or prevent fibrosis in subjects suffering from muscular dystrophy.

Disclosed are compositions and methods for treating diseases of the mammalian eye, and in particular, complications of the retina associated with Usher syndrome 1B (USH1B). Further disclosed are compositions and methods for treating diseases of the mammalian inner ear, and in particular, complications of ear hair cells associated with Usher syndrome 1B (USH1B). The disclosure provides improved AAV-based, dual vector systems that facilitate the expression of full-length proteins whose coding sequences exceed that of the polynucleotide packaging capacity of an individual AAV vector. Described herein are modified hybrid dual vector systems that shift the coding sequence for the MYO7A tail domain from the front-half vector to the back-half vector by altering the split point (e.g., from between exons 23 and 24, to between exons 21 and 22), in order to eliminate the production of truncated MYO7A protein. Further described herein are improved, codon-modified hybrid and overlap vector systems in which putative stop codons and residual sequences in non-coding sequences are removed.

Nucleotide sequences including a micro-dystrophin gene are provided. The micro-dystrophin genes may be operatively linked to a regulatory cassette. Methods of treating a subject having, or at risk of developing, muscular dystrophy, sarcopenia, heart disease, or cachexia are also provided. The methods may include administering a pharmaceutical composition including the micro-dystrophin gene and a delivery vehicle to a subject. Further, the methods may include administering the pharmaceutical composition a subject having Duchenne muscular dystrophy or Becker muscular dystrophy.

The invention relates to the technical field of immune globulin, in particular to an anti-human SMMHC monoclonal antibody and an antigen binding fragment, a preparation method and application thereof, the anti-human SMMHC monoclonal antibody comprises a light chain variable region VL and a heavy chain variable region VH, and amino acid sequences of LCDR1, LCDR2 and LCDR3 of the VL are respectively shown as SEQ ID NO.3-5; the amino acid sequences of the HCDR1, the HCDR2 and the HCDR3 of the VH are as shown in SEQ ID NO.7 to SEQ ID NO.9. The anti-human SMMHC rabbit monoclonal antibody disclosed by the invention has good affinity, can be specifically combined with SMMHC protein, remarkably improves the specificity, sensitivity, accuracy and reliability of SMMHC protein immunodetection, is mainly used for identifying and marking smooth muscle cells, can be suitable for marking breast muscle epithelium, and has the advantages of high sensitivity and high sensitivity. The kit can be used for differential diagnosis of breast duct internal cancer and invasive cancer, and can also be used for diagnosis of smooth muscle-derived tumors.

Provided herein are synthetic RNA molecules for reconstitution of RNA molecules, including compositions and methods of using these molecules. For example, such molecules can be used to deliver a protein coding sequence over two or more viral vectors (such as AAVs), resulting in reconstitution of the full-length protein in a cell. Such methods can be used to deliver a therapeutic protein, for example to treat a genetic disease or cancer.

Described herein are methods and culture medium, useful for inducing polarization in multipotent stem cells. Additionally, described herein are multipotent cells produced by the methods and culture medium of this disclosure that are useful therapeutic agents. Also described herein are extracellular vesicles and factors secreted by multipotent cells that are produced by the methods and culture medium of this disclosure that are useful as therapeutic agents.

The application belongs to the technical field of medicines, and discloses a polypeptide TCP-6 and application of the polypeptide TCP-6 in preparation of a medicine for treating acute kidney injury. The amino acid sequence of the polypeptide TCP-6 is shown as SEQ ID NO:1. Experiments show that the polypeptide TCP-6 can effectively relieve tubular epithelial cell injury and apoptosis occurring when AKI, and protect kidney function, and no obvious toxic side effects are found, so that the polypeptide TCP-6 can be applied as a medicine for treating acute kidney injury.

The invention relates to siRNA of a targeted skin scar promoting gene and application of the siRNA, and belongs to the technical field of biology. The invention provides siRNA (small interfering Ribonucleic Acid) targeting a skin scar promoting gene, and an antisense strand of the siRNA can be specifically combined with the skin scar promoting gene so as to trigger the degradation of the skin scar promoting gene; the skin scar promoting gene is a gene for coding a transforming growth factor 1; a positive-sense strand of the siRNA comprises a nucleic acid molecule with a nucleotide sequence as shown in SEQ ID NO.1, and an antisense strand of the siRNA comprises a nucleic acid molecule with a nucleotide sequence as shown in SEQ ID NO.11. Researches show that the siRNA can target the skin scar promoting gene, is high in interference efficiency, has high inhibitory activity on the skin scar promoting gene, and provides a good theoretical and practical basis for skin scar treatment.

The present invention provides a use of a polypeptide complex on the basis of Titin-Telethonin beta-pleated sheet structure as a polypeptide or protein drug carrier, a method, and a fusion protein complex thereof. The polypeptide complex is capable of maintaining the activity of polypeptide or protein drugs and prolonging the half-life period simultaneously.

The present invention relates to an improved variant monoclonal antibody binding to cardiac troponin T and having a better KD than the parent monoclonal antibody 11-7.

A protein polymer and a use thereof in the treatment of Alzheimer's disease. The protein polymer is obtained by stimulating MSCs, and then lysing the MSCs and performing separation and purification. The protein polymer has a strong oxidative damage repair ability, can improve Alzheimer's disease-related biomarker abnormalities, and has a significant treatment effect on Alzheimer's disease.

The present disclosure relates to compositions and methods for treating cardiomyopathy.Some embodiments provided herein relate to virus-mediated gene transfer into host cells to induce the host to express encoded polypeptide, protein, or other product to improve one or more symptoms of cardiomyopathy.In some embodiments, the method and composition relate to recombinant adeno-associated virus particles encoding human TNNT2 for treating cardiomyopathy, including dilated cardiomyopathy or hypertrophic cardiomyopathy.