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OwnerScott Loiler, Terence Flotte +2inventors
InventorsScott Loiler, Terence Flotte, Nicholas Muzyczka, Mark Atkinson
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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.
An adeno-associated viral vector comprising a first polynucleotide comprising a first nucleic acid segment that encodes an AAV capsid protein that comprises an exogenous amino acid sequence that binds to a mammalian lipoprotein receptor.
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A recombinant adeno-associated viral expression system comprising:
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