Methods of producing an exogenous protein in a bird egg are provided according to aspects of the present invention which include: providing transfected avian primordial germ cells by forming a complex of Cas protein and a guide nucleotide sequence in the avian primordial germ cells, whereby a donor DNA sequence is inserted into genomic DNA of the avian primordial germ cells under transcriptional control of a regulatory element that directs tissue-specific expression of the exogenous protein; introducing the transfected avian primordial germ cells into a population of recipient bird embryos and incubating the recipient bird embryos, generating germline chimera birds; obtaining a heterozygote and/or homozygote transgenic bird by breeding the germline chimera bird; and isolating the exogenous protein from an egg laid by a female transgenic bird.

The present invention provides for functional chimeric gene regulatory units capable of driving strong and sustained heterologous gene expression.

The present invention provides new adeno-associated virus-derived vectors and pharmaceutical compositions containing the same for the treatment of lysosomal storage disorders and specially, for the treatment of mucopolysaccharidoses Type IIID.

A transgenic avian containing in its genome an exogenous nucleotide sequence which includes a promoter component and a vector with reduced promoter interference wherein the exogenous nucleotide sequence is integrated into the genome and the avian.

Adeno-associated virus rh.20 sequences, vectors containing same, and methods of use are provided.

A transgenic avian containing in its genome an exogenous nucleotide sequence which includes a promoter component and a vector with reduced promoter interference wherein the exogenous nucleotide sequence is integrated into the genome and the avian.

Gene targeting is a technique to introduce genetic change into one or more specific locations in the genome of a cell. For example, gene targeting can introduce genetic change by modifying, repairing, attenuating or inactivating a target gene or other chromosomal DNA. In one aspect, this disclosure relates to methods and compositions for gene targeting with high efficiency in a cell. This disclosure also relates to methods of treating or preventing a genetic disease in an individual in need thereof. Further disclosed are chimeric nucleases and vectors encoding chimeric nucleases.

Proviral plasmids contain a modular gene expression cassette with one or a combination of (i) a wildtype 5 AAV2 ITR sequence flanked by unique restriction sites that permit ready removal or replacement of the ITR; (ii) a promoter flanked by unique restriction sites that permit ready removal or replacement of the entire promoter sequence; (iii) a polylinker sequence that permits insertion of a gene coding sequence without modification thereof, wherein the gene is operatively linked to, and under the regulatory control of, the aforementioned promoter; (iv) a bovine growth hormone polyadenylation sequence flanked by unique restriction sites that permit ready removal or replacement of the polyA sequence; and (v) a wildtype 3 AAV2 ITR sequence flanked by unique restriction sites that permit ready removal or replacement of the 3 ITR. These plasmids enable rapid manipulation of the components of the cassette, such as rapid mutation and/or replacement of any component, and thereby increase the efficiency of recombinant viral vector, such as rAAV, production.

Adeno-associated virus rh.10 sequences, vectors containing same, and methods of use are provided.

Adeno-associated virus rh.20 sequences, vectors containing same, and methods of use are provided.