Selected adeno-associated virus compositions having preferred brain, spinal cord, and/or heart expression levels
AssigneeCapsida Biotherapeutics, Inc.
InventorsBrandon G. Wheeler, Xiaojing Shi, Nicholas S. Goeden, Nicholas C. Flytzanis, Troy E. Sandberg
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Abstract
Described herein are compositions and kits comprising recombinant adeno-associated viruses (rAAVs) with increased viral transduction enrichment in the brain, spinal cord, and/or heart. The rAAV compositions described herein encapsidate a transgene, such as a therapeutic nucleic acid. Gene therapy using the rAAVs is described. Also described are methods of treating diseases of the brain, spinal cord, and/or heart-related diseases and conditions.
Claims21
An AAV capsid protein comprising a sequence having at least 70% identity with a sequence selected from SEQ ID NOS:
- 976-1949.
-44. (canceled)
Dependencies are inferred from the claim text.