Key result
Distinct routes of administration for adeno-associated viruses, including intravenous, intraparenchymal, and cerebrospinal fluid delivery, affect the efficiency and cellular tropism of gene therapies.
Why the study?
Distinct routes of administration affect the efficiency and safety of AAV-delivered gene therapies, requiring careful consideration when designing delivery strategies for CNS diseases.
This review provides updated knowledge on AAV administration routes to facilitate optimal delivery strategies for CNS gene therapies in preclinical models.
Route optimization may improve AAV CNS gene therapy efficacy; extends preclinical data but leaves clinical translation open.
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 efficiency of transduction, their immunotoxicity, loading capacity, the choice of serotype, and peripheral off-target effects should be carefully considered when designing an optimal AAV delivery strategy for a specific disease. In addition, distinct routes of administration may affect the efficiency and safety of AAV-delivered gene therapies. In this review, we summarize different administration routes of gene therapies delivered by AAVs to the brain in mice and rats. Updated knowledge regarding AAV-delivered gene therapies may facilitate the selection from various administration routes for specific disease models in future research.
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Zhou et al. (2022) conducted a review in Central nervous system diseases. Adeno-associated viruses (AAVs) administration routes was evaluated. Distinct routes of administration for adeno-associated viruses, including intravenous, intraparenchymal, and cerebrospinal fluid delivery, affect the efficiency and cellular tropism of gene therapies.
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