Abstract: A chimeric protein that is a fusion construct of a series of functional domains is used to deliver a therapeutic agent to a human subject suffering from disease. In some embodiments, the chimeric protein includes a therapeutic region, a transportation region, and a cleavage region disposed between the therapeutic region and the transportation region. The transportation region allows the chimeric protein to be moved across a cellular membrane of an affected cell within the subject. Cleavage of the chimeric protein at the cleavage region once within the cell separates the therapeutic region from the transportation region, enabling the therapeutic region to function normally within the cell. The therapeutic region can be effective in the treatment of, for example, muscular dystrophy, diastrophic dysplasia, malignant melanoma, porphyria, alpha-1 antitrypsin deficiency, Aicardi-Goutieres syndrome, cystic fibrosis, progeria, Marfan syndrome, tuberous sclerosis, adrenoleukodystrophy, and the like.

Abstract: The design and generation of a number of chimeric VSV-G (or VSV-G variants) proteins are used as transfer vehicles to enhance delivery of nucleic acids like plasmid DNA, single and double stranded DNA and RNA, and antisense oligonucleotides into human and animal cells. These chimeric VSV-G protein-nucleic acid transfer vehicles have widespread applications to deliver nucleic acids for exon skipping and gene delivery for gene replacement in human and animals.

Abstract: The design and generation of a number of chimeric VSV-G (or VSV-G variants) proteins are used as transfer vehicles to enhance delivery of nucleic acids like plasmid DNA, single and double stranded DNA and RNA, and antisense oligonucleotides into human and animal cells. These chimeric VSV-G protein-nucleic acid transfer vehicles have widespread applications to deliver nucleic acids for exon skipping and gene delivery for gene replacement in human and animals.

Abstract: A chimeric protein that is a fusion construct of a series of functional domains is used to deliver a therapeutic agent to a human subject suffering from disease. In some embodiments, the chimeric protein includes a therapeutic region and a transportation region. The transportation region allows the chimeric protein to be moved across a cellular membrane of an affected cell within the subject. The therapeutic region can be effective in the treatment of, for example, muscular dystrophy, diastrophic dysplasia, malignant melanoma, porphyria, alpha-1 antitrypsin deficiency, Aicardi-Goutieres syndrome, cystic fibrosis, progeria, Marfan syndrome, tuberous sclerosis, adrenoleukodystrophy, and the like.

Abstract: The design and generation of a number of chimeric VSV-G (or VSV-G variants) proteins are used as transfer vehicles to enhance delivery of nucleic acids like plasmid DNA, single and double stranded DNA and RNA, and antisense oligonucleotides into human and animal cells. These chimeric VSV-G protein-nucleic acid transfer vehicles have widespread applications to deliver nucleic acids for exon skipping and gene delivery for gene replacement in human and animals.

Abstract: A chimeric protein that is a fusion construct of a series of functional domains is used to deliver a therapeutic agent to a human subject suffering from disease. In some embodiments, the chimeric protein includes a therapeutic region, a transportation region, and a cleavage region disposed between the therapeutic region and the transportation region. The transportation region allows the chimeric protein to be moved across a cellular membrane of an affected cell within the subject. Cleavage of the chimeric protein at the cleavage region once within the cell separates the therapeutic region from the transportation region, enabling the therapeutic region to function normally within the cell. The therapeutic region can be effective in the treatment of, for example, muscular dystrophy, diastrophic dysplasia, malignant melanoma, porphyria, alpha-1 antitrypsin deficiency, Aicardi-Goutieres syndrome, cystic fibrosis, progeria, Marfan syndrome, tuberous sclerosis, adrenoleukodystrophy, and the like.