Novel recombinant adeno-associated virus capsids with enhanced human pancreatic tropism
AssigneeThe Board of Trustees of the Leland Stanford Junior University
InventorsKatja Pekrun, Mark A. Kay
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Abstract
The present invention relates to variant AAV capsid polypeptides, wherein the variant AAV capsid polypeptides exhibit increased transduction and/or tropism in human pancreatic tissue or human islets as compared non-variant parent capsid polypeptides.
Claims22
1-22. (canceled)
A method of usinga variant AAV capsid polypeptide to reduce the amount of total nucleic acid administered to a subject, said method comprising administering less total nucleic acid amount to said subject when said nucleic acid is transduced usingthe variant AAV capsid polypeptide as compared to the amount of nucleic acid administered to said subject when said nucleic acid is transduced using a non-variant parent capsid polypeptide in order to obtain a similar therapeutic effect, wherein the variant AAV capsid polypeptide exhibits increased transduction or tropism in human pancreatic tissue or human islets as compared to the non-variant parent capsid polypeptide.
24-38. (canceled)
A method of usinga variant AAV capsid polypeptides to reduce the amount of total AAV vector administered to a subject, said method comprising administering less total AAV vector amount to said subject when said AAV vector is transduced bythe variant AAV capsid polypeptide as compared to the amount of AAV vector administered to said subject when said AAV vector is transduced by a non-variant parent capsid polypeptide in order to obtain a similar therapeutic effect, wherein the variant AAV capsid polypeptide exhibits increased transduction or tropism in human pancreatic tissue or human islets as compared to the non-variant parent capsid polypeptide.
40-66. (canceled)
A method of usinga variant AAV capsid polypeptides to reduce the amount of total AAV vector administered to a subject, said method comprising administering less total AAV vector amount to said subject when said AAV vector is transduced bythe variant AAV capsid polypeptide as compared to the amount of AAV vector administered to said subject when said AAV vector is transduced by a non-variant parent capsid polypeptide in order to obtain a similar therapeutic effect, wherein the variant AAV capsid polypeptide exhibits increased transduction or tropism in human pancreatic tissue or human islets as compared to the non-variant parent capsid polypeptide.
68-79. (canceled)
Dependencies are inferred from the claim text.