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.

The present invention provides myosin derived peptides, variants and derivative compounds that are anti-coagulant. Also provided in the invention are antibodies that specifically target the peptides or epitopes presented thereon. Further provided in the invention are methods directed to using the anti-coagulant compounds described herein in therapeutic applications, e.g., inhibiting myosin-supported prothrombin activation to reduce the risk of acute trauma coagulopathy in post-trauma patients or inhibiting cardiac myosin-supported prothrombin activation to reduce the risk of coronary thrombosis.

The present disclosure describes the fusogenic activity of the Myomaker protein. This polypeptide, when expressed in non-muscle cells, is able to drive fusion of the cell with a muscle cell, but not with other non-muscle cells. The use of this protein and cell expressing it in the delivery of exogenous genetic material to muscle cells also is described.

The present disclosure relates to a new polypeptide complex. Specifically, the present disclosure relates to a domain engineered antibody. At least one of the constant region domains CH1 and/or CL of the antibody is replaced, and the domain CH1/CL is replaced by a Titin T chain/Obscurin-O chain or by a Titin T chain/Obscurin-like-O chain.

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.

The present invention relates to the combination of dietary peptides, composition comprising such combinations including nutritional supplements and methods for inducing satiation and satiety, for preventing or reducing the incidence of metabolic syndrome comprising overweight and obesity, cardiovascular diseases, atherosclerosis, hypertension, hepatosteatosis, diabetes and/or cancer.

The present application provides materials and methods for treating a patient with a titin-based myopathy, particularly a titin-based cardiomyopathy, and/or other titinopathy. In addition, the present application provides materials and methods for editing the titin gene in a cell by genome editing.

The present invention relates to the field of genetic engineering, preferably the expression of recombinant proteins (RP). In particular, the invention relates to a promoter and variants thereof having an equal function and more than 90% sequence identity. The promoter comprises a fragment of 1147 base pairs (bp) of a first promoter, promoter of the β-actin gene of the Cricetulus griseus genome, enriched in cytosine-guanine dinucleotides (RegCG). The first promoter can be upstream of a second promoter, cytomegalovirus (CMV) promoter. The invention also relates to vectors, transfected cellular lines and a method for producing RP in mammal cells that have been transfected with vectors containing said promoter or variants thereof.

Disclosed are compositions and methods for treating diseases of the mammalian eye, and in particular, complications of the retina associated with Usher syndrome IB (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 IB (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 MY07A 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 MY07A 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.

This disclosure provides methods and materials for producing a transgenic plant expressing a myoglobin gene, producing myoglobin protein in the transgenic plant, and isolating the myoglobin protein from the transgenic plants.