Journal
CURRENT GENE THERAPY
Volume 22, Issue 3, Pages 185-190Publisher
BENTHAM SCIENCE PUBL LTD
DOI: 10.2174/1566523221666210922155413
Keywords
Adeno-associated virus; aromatic L-amino acid decarboxylase deficiency; eladocagene exuparvovec; immunogenicity; vectors; rare disease
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Funding
- PTC Therapeutics
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This article illustrates 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.
Pre existing immunity to adeno-associated virus (AAV) poses a concern in AAV vector-mediated gene therapy. Localized administration of low doses of carefully chosen AAV sero-types 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.
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