TY - JOUR
T1 - Reduced Immunogenicity of Intraparenchymal Delivery of Adeno-Associated Virus Serotype 2 Vectors
T2 - Brief Overview
AU - Hwu, Wuh Liang
AU - Muramatsu, Shin Ichi
AU - Gidoni-Ben-zeev, Bruria
N1 - Publisher Copyright:
© 2022 Bentham Science Publishers.
PY - 2022/6
Y1 - 2022/6
N2 - Pre existing immunity to adeno-associated virus (AAV) poses a concern in AAV vec-tor–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.
AB - Pre existing immunity to adeno-associated virus (AAV) poses a concern in AAV vec-tor–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.
KW - Adeno-associated virus
KW - aromatic L-amino acid decarboxylase deficiency
KW - eladocagene exuparvovec
KW - immunogenicity
KW - rare disease
KW - vectors
UR - http://www.scopus.com/inward/record.url?scp=85126830213&partnerID=8YFLogxK
U2 - 10.2174/1566523221666210922155413
DO - 10.2174/1566523221666210922155413
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C2 - 34551695
AN - SCOPUS:85126830213
VL - 22
SP - 185
EP - 190
JO - Current Gene Therapy
JF - Current Gene Therapy
SN - 1566-5232
IS - 3
ER -