Adeno-associated virus variant capsids and use for inhibiting angiogenesis
Assignee4D Molecular Therapeutics Inc.
InventorsDavid H. Kirn, Melissa Kotterman, David Schaffer, Paul Szymanski, Peter Francis
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Assignee4D Molecular Therapeutics Inc.
InventorsDavid H. Kirn, Melissa Kotterman, David Schaffer, Paul Szymanski, Peter Francis
Estimating the term…
Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina for the treatment of retinal disorders and diseases.
A nucleic acid encoding an inhibitor of VEGF-A, said nucleic acid comprising one or more of:
Dependencies are inferred from the claim text.