The disclosure relates to pharmaceutical compositions and methods of using pharmaceutical compositions comprising effective dosages of an adenoviral-based biological delivery and expression system for use in the treatment or prevention of osteoarthritis in human or mammalian joints by long-term inducible gene expression of human or mammalian interleukin-1 receptor antagonist (IL-1Ra) in synovial cells, comprising a helper-dependent adenoviral vector containing a nucleic acid sequence encoding for human or mammalian interleukin-1 receptor antagonist (IL-1Ra), left and right inverted terminal repeats (L ITR and R ITR), the adenoviral packaging signal and non-viral, non-coding stuffer nucleic acid sequences, wherein the expression of the human or mammalian interleukin-1 receptor antagonist (IL-1Ra) gene within synovial cells is regulated by an inflammation-sensitive promoter.

The present invention provides a method for preventing or treating liver cancer in a subject comprising administrating at least one selected from the group consisting of an Ssu72 peptide, a polynucleotide encoding the Ssu72 peptide and an expression vector containing the polynucleotide to the subject.

The present disclosure belongs to the technical field of biomedicine, and provides an engineered circular RNA circmiR-29b and use thereof in preparation of a medicine for treating muscle atrophy. The present disclosure provides a circular RNA circmiR-29b including an effective sequence and a random sequence, wherein 6-13 repetitions of the effective sequence are connected in series, the random sequence is inserted between the effective sequences, and the nucleotide sequence of the effective sequence is shown in SEQ ID NO: 1. The present disclosure delivers circmiR-29b to skeletal muscle by AAV8, enabling stable expression of circmiR-29b in the skeletal muscle, thereby effectively inhibiting various types of muscle atrophy. Therefore, the present disclosure also provides a gene therapy based on the AAV8 virus vector delivering the engineered circular RNA circmiR-29b to achieve the objective of treating muscle atrophy.

The present disclosure provides methods for treating, preventing, or inhibiting diseases in patients having one or more mutations in complement factor H (CFH), complement component 3 (C3), and complement factor B (CFB) by administering to the patients a recombinant adeno-associated virus (rAAV) vector encoding a CFH polypeptide or biologically active fragment and/or variant thereof.

A vector system for expressing a transgene in a cell, the vector system comprising a first vector and a second vector, wherein: (a) the first vector comprises in a 5′ to 3′ direction: a promoter; an intron; a 5′ end portion of the transgene coding sequence (CDS); a splice donor sequence; and a first recombinogenic region; (b) the second vector comprises in a 5′ to 3′ direction: a second recombinogenic region; a splice acceptor sequence; and a 3′ end portion of the transgene CDS; wherein the 5′ end portion and the 3′ end portion together constitute the transgene CDS, and wherein the intron is not capable of homologous recombination with the splice donor sequence to excise the 5′ end portion of the transgene CDS.

The present invention provides compositions and methods for treating cartilage disorders using expression vectors including a nucleic acid encoding a suicide gene, and a fibroblast growth factor 18 (FGF-18) polypeptide or a functional fragment thereof. The present invention relates to expression vectors, and methods of use thereof for promoting the proliferation of cells responsible for the production and maintenance of tissues, such as chondrocytes, cardiomyocytes, and synoviocytes.

The invention provides compositions and methods for the preparation, manufacture, formulation and therapeutic use of adeno-associated virus (AAV) particles for the prevention and/or treatment of diseases.

The present invention concerns an expression system for systemic administration comprising a sequence encoding a FKRP protein, and: —a promoter sequence allowing the expression at a therapeutically acceptable level of FKRP in the skeletal muscles and a target sequence of an miRNA expressed in the heart; or—a promoter sequence allowing the expression at a therapeutically acceptable level of FKRP in the skeletal muscles and presenting a promoter activity at a toxically acceptable level in the heart; and its use for the treatment of various diseases linked to FKRP deficiencies.

The present disclosure relates generally to methods and adeno-associated virus (AAV) vectors for in vivo transduction. The disclosure is directed to methods for producing modified AAV capsid polypeptides and AAV vectors that are suitable for transduction of cells in vivo, and methods for screening AAV vectors. The disclosure also relates to adeno-associated virus (AAV) capsid polypeptides and encoding nucleic acid molecules, AAV vectors comprising the capsid polypeptides, and nucleic acid vectors comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.

The present invention relates to methods and materials useful for systemically delivering polynucleotides across the blood brain barrier using adeno-associated virus as a vector. For example, the present invention relates to methods and materials useful for systemically delivering α-N-acetylglucosamidinase polynucleotides to the central and peripheral nervous systems, as well as the somatic system. Use of these methods and materials is indicated, for example, for treatment of the lysosomal storage disorder mucopolysaccharidosis IIIB. As another example, the present invention relates to methods and materials useful for systemically delivering N-sulphoglucosamine sulfphohydrolase polynucleotides to the central and peripheral nervous systems, as well as the somatic system. Use of this second type of methods and materials is indicated, for example, for treatment of the lysosomal storage disorder mucopolysaccharidosis IIIA.