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Updated: Feb 16, 2026
Adeno-associated Virus-mediated Transgene Expression in Genetically Defined Neurons of the Spinal Cord
Published on: May 12, 2018
AAV gene delivery to the spinal cord: serotypes, methods, candidate diseases, and clinical trials
Nathan Hardcastle1, Nicholas M Boulis1, Thais Federici1
1a Department of Neurosurgery , Emory University , Atlanta , GA , USA.
Introduction:
Adeno-associated viral (AAV) vector-mediated gene delivery to the spinal cord has finally entered the pathway towards regulatory approval. Phase 1 clinical trials using AAV gene therapy for pediatric disorders - spinal muscular atrophy (SMA) and giant axonal neuropathy (GAN) - are now underway.
Areas Covered:
This review addresses the latest progress in the field of AAV gene delivery to the spinal cord, particularly focusing on the most prominent AAV serotypes and delivery methodologies to the spinal cord. Candidate diseases and scaling up experiments in large animals are also discussed.
Expert Opinion:
Intravenous (IV) and intrathecal (IT) deliveries seem to undoubtedly be the preferred routes of administration for diffuse spinal cord delivery of therapeutic AAV vectors that can cross the blood-brain barrier (BBB) and correct inherited genetic disorders. Conversely, intraparenchymal delivery is still an undervalued but very viable approach for segmental therapy in afflictions such as ALS or Pompe Disease as a means to prevent respiratory dysfunction.
