Wuh-Liang Hwu, Shin-Ichi Muramatsu* and Bruria Gidoni-Ben-Zeev
Preexisting immunity to adeno-associated virus (AAV) poses a concern in AAV vector–mediated gene therapy. Localized administration of low doses of carefully chosen AAV serotypes can mitigate the risk of an immune response. This article will illustrate the low risk of immune response to AAV serotype 2 vector–mediated gene therapy to the brain with support from clinical trial data in aromatic L-amino acid decarboxylase deficiency and Parkinson disease.
adeno-associated virus, aromatic L-amino acid decarboxylase deficiency, eladocagene exuparvovec, immunogenicity, vectors, rare disease
Department of Medical Genetics and Pediatrics, 7 Chung-Shan S. Road, National Taiwan University Hospital, Taipei, Division of Neurological Gene Therapy, Center for Open Innovation, Jichi Medical University, 3311-1 Yakushiji, Shimotsuke, Tochigi 329-0498, Department of Pediatric Neurology, Sackler School of Medicine, Tel Aviv University, Tel Aviv-Yafo, 6997801