2024 Volume 41 Issue 4 Pages 625-628
Adeno–associated virus (AAV) vectors have the potential for long–term expression in non–dividing neurons. AAV9 has become a mainstay in AAV gene therapy for neurological diseases. An intravenous drug with the AAV9 vector carrying the SMN gene for spinal muscular atrophy has become the first insurance–covered treatment for neurological diseases, yielding positive outcomes.
We performed gene therapy for aromatic L–amino acid decarboxylase deficiency caused by DDC gene mutations. All patients improved their motor function. Now, we are conducting Dr.–led clinical trials.
Gene therapy using AAV vectors is only suitable for certain diseases, which include disorders with clear single–gene candidates, functional disorders, diseases where gene overexpression poses no issues and functional recovery is expected with gene introduction into specific cells, and diseases with model animals for assessing therapeutic effects.
Considering the continued advances in the development of AAV vectors and administration methods, an increase in the number of target diseases for gene therapy can be expected soon.
