Adeno-associated virus composition for targeted gene therapy

摘要

Compositions and kits comprising recombinant adeno-associated virus (rAAV) having a directivity showing increased specificity and increased efficiency of viral transduction in target cell types such as brain and lung are described herein. The rAAV compositions described herein also have a directivity showing reduced specificity and reduced efficiency of viral transduction in non-targeted cell types such as the liver. The rAAV compositions described herein encapsulate a transgene, such as a Therapeutic Nucleic Acid, in a capsid. Upon systemic delivery to a subject, rAAV can increase the specificity of the transgene into the target cell type and increase transduction compared to the parent or reference AAV.