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Routes of administration for adeno-associated viruses carrying gene therapies for brain diseases

26 Oct 2022

Abstract excerpt

Gene therapy is a powerful tool to treat various central nervous system (CNS) diseases ranging from monogenetic diseases to neurodegenerative disorders. Adeno-associated viruses (AAVs) have been widely used as the delivery vehicles for CNS gene therapies due to their safety, CNS tropism, and long-term therapeutic effect. However, several factors, including their ability to cross the blood-brain barrier, the...

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PMID: 36385771
DOI: 10.3389/fnmol.2022.988914

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