150results 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.

Provided herein are gene therapy compositions and methods for treating reduced levels of functional cardiac myosin binding protein C in a subject suffering from hypertrophic cardiomyopathy.

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.

Aspects of the disclosure relate to compositions and methods useful for delivering minigenes to a subject. Accordingly, the disclosure is based, in part, on isolated nucleic acids and gene therapy vectors, such as viral (e.g., rAAV) vectors, comprising one or more gene fragments encoding a therapeutic gene product, such as a protein or peptide (e.g. a minigene). In some embodiments, the disclosure relates to gene therapy vectors encoding a USH1B protein (e.g. the gene product of USH1B, also referred to as MY07A) or a portion thereof. In some embodiments, compositions described by the disclosure are useful for treating diseases associated with mutations in the USH1B (MY07A) gene, for example Usher Syndrome.

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.

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 invention belongs to the technical field of genetic engineering, and particularly relates to truncated tropomyosin, a nucleic acid molecule and application of the truncated tropomyosin. The truncated tropomyosin is based on an alpha-helical structure of tropomyosin, and is obtained by taking glutamic acid (E26) at the 26th site, glutamine (Q51) at the 51st site or glutamic acid (E73) at the 73rd site as a truncation site. By reasonably designing a truncation site and further replacing the secretion signal peptide, the expression quantity of the protein in a recombinant expression system is remarkably improved, the immunogenicity of the protein is effectively reserved, and the protein can be used for precise diagnosis of allergy and development of anti-allergic vaccines.

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 present invention relates to the field of biomedicine technology, and in particular to an α-myosin mutant and its application. Lysine (K) at position 1897 in the wild-type Myh6 protein is mutated to arginine (R). The nucleotide sequence corresponding to the wild-type Myh6 protein is shown in the bases in SEQ ID NO.1. Application of α-myosin mutants as Ang II-induced heart failure pathology models. The present invention obtains α-myosin mutants through the gene editing method of the CRISPR / Cas9 system, confirms that the 1897 site in α-myosin participates in and regulates the degree of interaction between α-MHC and Titin, thereby reflecting the degree of heart failure. The obtained α-myosin mutant is used as a pathology model leading to heart failure, which can lay the foundation for the study of organic heart failure, especially heart failure caused by abnormal myofilament structure.

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.

Nucleic acid molecules comprising at least one transcription regulatory element operably linked to an open reading frame, wherein the open reading frame encodes a single RNA transcript encoding GCaMP7f, a ribosomal skipping peptide, and a fusion protein of a nuclear protein and photoactivatable red fluorescent protein are provided. Expression vectors and cells comprising the nucleic acid molecules are also provided, as are methods of using the nucleic acid molecules for simultaneous labeling and measuring calcium and analyzing a target cell.

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.

Disclosures herein are directed to compositions comprising single guide RNA (sgRNA) and fusion proteins comprising a Cas9 nickase and deaminase designed for a CRISPR-Cas9 system and method of using thereof for preventing, ameliorating or treating one or more cardiomyopathies.

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.

The invention belongs to the technical field of medicines, and particularly relates to a milk acylation modified Myh6 protein and application thereof. The antigen polypeptide for identifying the heart failure marker has an amino acid sequence as shown in SEQ ID NO: 1, lysine located at the 9th site is subjected to milk acylation modification, and the heart failure marker is milk acylation modified Myh6 protein; the milk acylation modified Myh6 protein is a lysine milk acylation modified protein (Myh6-K1897) which is located at the 1897 site of a wild type Myh6 protein. The Myh6-K1897 can be used as a marker for identifying heart failure as a target spot, is a set of novel and perfect protein milk acylation molecular regulation and control system, can judge the interaction between Myh6 and Tin, and is used for treating the core and key of heart failure. The research provides a new and powerful treatment strategy for the treatment of human heart failure diseases.

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 invention provides intrathecal compositions comprising AAV particles and their use for treating monogenic muscle disorders such as dystrophinopathies, including Duchenne muscular dystrophy.

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.

High-throughput screening (HTS) assays described herein rely on structural alterations triggered by cTnI phosphorylation during beta-adrenergic stimulation. These HTS assays may be used to pinpointsmall molecules that emulate phosphorylation effects, amplifying lusitropy by accelerating calcium dissociation from thin filaments. With a focus on addressing diastolic dysfunction, especially in conditions like HCM, the aim is to determine compounds tailored to modulate diastolic performance.

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 disclosure provides polynucleotides containing regions of the Myosin 15 (Myo15) promoter, as well as vectors containing the same, that can be used to promote expression of a transgene specifically in hair cells. The polynucleotides described herein may be operably linked to a transgene, such as a transgene encoding a therapeutic protein, so as to promote hair cell-specific expression of the transgene. The polynucleotides described herein may be operably linked to a therapeutic transgene and used for the treatment of subjects having or at risk of developing hearing loss or vestibular dysfunction.

The disclosure provides polynucleotides containing regions of the Myosin 15 (Myo15) promoter, as well as vectors containing the same, that can be used to promote expression of a transgene specifically in hair cells. The polynucleotides described herein may be operably linked to a transgene, such as a transgene encoding a therapeutic protein, so as to promote hair cell-specific expression of the transgene. The polynucleotides described herein may be operably linked to a therapeutic transgene and used for the treatment of subjects having or at risk of developing hearing loss or vestibular dysfunction.

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.

The present invention provides compositions and methods for treating a neuromuscular disorder. In certain embodiments, the invention provides compositions and methods for assessing weaning readiness and continuation of weaning off of a mechanical ventilator in a subject with X-linked myotubular myopathy (XLMTM).

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.