Patent classifications
C12N2750/14333
Chimeric adeno-associated virus/ bocavirus parvovirus vector
The invention provides an isolated chimeric virus comprising bocavirus capsid protein, e.g., an isolated chimeric virus comprising human bocavirus capsid protein, and a recombinant adeno-associated viral (AAV) genome, an isolated recombinant bocavirus (rBoV) comprising human bocavirus capsid protein and a recombinant Boy genome, and uses therefor, for example, in gene therapy for diseases in a mammal including diseases with aberrant expression of an endogenous gene product.
Recombinant adeno-associated virus delivery of exon 2-targeted U7SNRNA polynucleotide constructs
The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy.
Recombinant Adeno-Associated Virus Delivery of Exon 2-Targeted U7SNRNA Polynucleotide Constructs
The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy.
Chimeric adeno-associated virus/ bocavirus parvovirus vector
The invention provides an isolated chimeric virus comprising bocavirus capsid protein and a recombinant adeno-associated viral (AAV) genome, an isolated rBoV comprising human bocavirus capsid protein and a recombinant BoV genome, and uses therefor. For example, the chimeric virus may be employed to deliver transgenes, such as those encoding therapeutic or prophylactic gene products, to mammalian cells.
Recombinant Adeno-Associated Virus Delivery of Exon 2-Targeted U7SNRNA Polynucleotide Constructs
The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy.
CHIMERIC ADENO-ASSOCIATED VIRUS/ BOCAVIRUS PARVOVIRUS VECTOR
The invention provides an isolated chimeric virus comprising bocavirus capsid protein and a recombinant adeno-associated viral (AAV) genome, an isolated rBoV comprising human bocavirus capsid protein and a recombinant BoV genome, and uses therefor.
Recombinant adeno-associated virus delivery of exon 2-targeted U7SNRNA polynucleotide constructs
The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy.