Abstract: A polynucleotide comprising a nucleotide sequence encoding the retinitis pigmentosa GTPase regulator ORF15 isoform (RPGRORF15) wherein the RPGRORF15 encoding nucleotide sequence has been codon optimised to increase fidelity of replication and wherein the sequence comprises one or more nucleotides selected from: C at the position corresponding to position 1968 of SEQ ID NO: 2, A at the position corresponding to position 2088 of SEQ ID NO: 2, and C at the position corresponding to position 2205 of SEQ ID NO: 2.

Abstract: Disclosed are recombinant adeno-associated viral (rAAV) vector compositions that are expressed in selected mammalian cells, such as pancreatic islets cells, and that encode one or more mammalian serpin or cytokine polypeptides having therapeutic efficacy in the amelioration, treatment and/or prevention of interleukin deficiencies, such as for example diabetes, and related diseases of the pancreas. Also disclosed are methods and compositions for preventing diabetes in a mammal, reducing the rate of disease progression, and ameliorating the symptoms of diabetes in humans at risk for developing such conditions.

Abstract: Vectors that encode Adeno-Associated Virus (AAV) Rep and Cap proteins of different serotypes and Adenovirus transcription products that provide helper functions were used to produce pseudotyped recombinant AAV (rAAV) virions. Purification methods generated pseudotyped rAAV virion stocks that were 99% pure with titers of 1×1012?1×1013 vector genomes/ml.

Abstract: Disclosed are recombinant adeno-associated viral (rAAV) vector compositions that are expressed in selected mammalian cells, such as pancreatic islets cells, and that encode one or more mammalian serpin or cytokine polypeptides having therapeutic efficacy in the amelioration, treatment and/or prevention of interleukin deficiencies, such as for example diabetes, and related diseases of the pancreas. Also disclosed are methods and compositions for preventing diabetes in a mammal, reducing the rate of disease progression, and ameliorating the symptoms of diabetes in humans at risk for developing such conditions.

Abstract: Disclosed are methods for the use of therapeutic polypeptide-encoding polynucleotides in the creation of transformed host cells and transgenic animals. In particular, the use of recombinant adeno-associated viral (rAAV) vector compositions that specifically target mammalian cells, such as pancreatic islets cells, that express low-density lipoprotein receptors on their cell surface. The disclosed vectors comprise one or more polynucleotide sequences that express one or more mammalian polypeptides having therapeutic efficacy in the amelioration, treatment and/or prevention of AAT- or cytokine polypeptide deficiencies, such as for example in diabetes and related diseases, as well as a variety of autoimmune disorders including, for example, lupus and rheumatoid arthritis.

Abstract: Vectors that encode Adeno-Associated Virus (AAV) Rep and Cap proteins of different serotypes and Adenovirus transcription products that provide helper functions were used to produce pseudotyped recombinant AAV (rAAV) virions. Purification methods generated pseudotyped rAAV virion stocks that were 99% pure with titers of 1×1012–1×1013 vector genomes/ml.

Abstract: Vectors that encode Adeno-Associated Virus (AAV) Rep and Cap proteins of different serotypes and Adenovirus transcription products that provide helper functions were used to produce pseudotyped recombinant AAV (rAAV) virions. Purification methods generated pseudotyped rAAV virion stocks that were 99% pure with titers of 1×1012-1×1013 vector genomes/ml.

Abstract: Recombinant adeno-associated virus (rAAV) virions having a serotype 1 capsid protein are effective for transferring heterologous nucleic acids into DC.