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AssigneeUniversity of Pittsburgh—Of the Commonwealth System of Higher Education
InventorsLeah Caroline THOMAS BYRNE, Molly E. JOHNSON, William Richard STAUFFER, Bilge Esin OZTURK
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This document relates to AAV vectors (e.g., AAV2 vectors). For example, AAV vectors (e.g., AAV2 vectors) containing an AAV capsid polypeptide that includes an amino acid sequence set forth in any one or more of Tables 1A-1L (or a variant thereof) or according to Formula A based on such a set forth sequence, such AAV capsid polypeptides, nucleic acid molecules encoding such vectors, nucleic acid molecules encoding such AAV capsid polypeptides, host cells containing and/or expressing such nucleic acid molecules, and methods and materials for making or using such vectors and/or AAV capsid polypeptides are provided.
An adeno-associated virus (AAV) vector comprising an AAV capsid polypeptide, wherein said capsid polypeptide comprises the amino acid sequence of any one of SEQ ID NOs:2-1103.
An AAV capsid polypeptide comprising the amino acid sequence of any one of SEQ ID NOs:2-1103.
A method for delivering an exogenous nucleic acid sequence to a retinal cell within a mammal, wherein said method comprises contacting said retinal cell with an AAV vector comprising an AAV capsid polypeptide and said exogenous nucleic acid sequence, wherein said capsid polypeptide comprises the amino acid sequence of any one of SEQ ID NOs:2-1103, wherein said AAV vector infects said retinal cell, thereby delivering said exogenous nucleic acid sequence to said retinal cell.
The method of 23, wherein said capsid polypeptide comprises the amino acid sequence of SEQ ID NO:1 or SEQ ID NO:2206 except that the amino acids from position 585 to 590 of SEQ ID NO:1 or SEQ ID NO:2206 are replaced with said amino acid sequence of any one of SEQ ID NOs:2-1103.
A method for treating a retinal condition in a mammal in need thereof, wherein said method comprises contacting retinal cells of a mammal having said retinal condition with AAV vectors comprising an AAV capsid polypeptide and an exogenous nucleic acid sequence, wherein said capsid polypeptide comprises the amino acid sequence of any one of SEQ ID NOs:2-1103, wherein said AAV vectors infect said retinal cells and drive expression of said exogenous nucleic acid sequence within said retinal cells, thereby treating said retinal condition.
A non-naturally occurring AAV capsid polypeptide, wherein said capsid polypeptide comprises the amino acid sequence of SEQ ID NO:1 or SEQ ID NO:2206 comprising an amino acid sequence insert of Formula A located between amino acid positions 587 and 588 of SEQ ID NO:1 or SEQ ID NO:2206, wherein said Formula A is:
The capsid polypeptide of any one of claims 57-59-111, wherein said X2 is selected from the group of amino acid residues consisting of A, V, I, and L.
A method of treating a retinal disorder in a patient in need thereof, comprising administering to the patient's eye an effective amount of an AAV vector, wherein the AAV vector comprises an AAV capsid polypeptide and an exogenous nucleic acid sequence, wherein the AAV capsid polypeptide is represented by Formula A.
Dependencies are inferred from the claim text.