Adeno-associated virus vectors for nucleic acid delivery to retinal ganglion cells and/or retinal pigment epithelium cells
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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Abstract
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 Table 1 (or a variant thereof) or Formula A, 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.
Claims92
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:2-5.
A method for delivering an exogenous nucleic acid sequence to a retinal ganglion cell within a mammal, wherein said method comprises contacting said retinal ganglion 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:2-5, wherein said AAV vector infects said photoreceptor cell, thereby delivering said exogenous nucleic acid sequence to said photoreceptor cell.
A method for treating a retinal condition in a mammal in need thereof, wherein said method comprises contacting retinal ganglion cells and/or retinal pigment epithelium 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:2-5, wherein said AAV vectors infect said retinal ganglion cells and/or retinal pigment epithelium cells and drive expression of said exogenous nucleic acid sequence within said retinal ganglion cells and/or retinal pigment epithelium cells, thereby treating said retinal condition.
A non-naturally occurring adeno-associated virus (AAV) vector comprising an AAV capsid polypeptide, wherein said capsid polypeptide comprises the amino acid sequence of SEQ ID NO:1 or SEQ ID NO:10 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:10, wherein said Formula A is:
- -L1-NTEARV(SEQ ID NO:2)-L2-,
- wherein said L1 and said L2 are each independently optional amino acid linkers having one, two, or three amino acids.
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:10 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:10, wherein said Formula A is:
- -L1-
- (SEQ ID NO:
- 2)
- NTEARV
- -L2-,
- wherein said L1 and said L2 are each independently optional amino acid linkers having one, two, or three amino acids.
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.