Evading the AAV Immune Response in Mucopolysaccharidoses.
作者： Matthew Piechnik , Kazuki Sawamoto , Hidenori Ohnishi , Norio Kawamoto , Yasuhiko Ago
DOI: 10.3390/IJMS21103433
关键词: Serotype 、 Antibody 、 Adeno-associated virus 、 Immune system 、 Immunosuppression 、 Medicine 、 Transgene 、 Genetic enhancement 、 Immunology 、 Virus
摘要: The humoral immune response elicited by adeno-associated virus (AAV)-mediated gene therapy for the treatment of mucopolysaccharidoses (MPS) poses a significant challenge to achieving therapeutic levels transgene expression. Antibodies targeting AAV capsid as well product diminish production glycosaminoglycan (GAG)-degrading enzymes essential MPS. Patients who have antibodies against increase in number with age, serotype, and racial background are excluded from clinical trials at present. In addition, patients undergone often additional same since their acquired (antibody) will limit further efficacy treatment. Several methods being developed overcome this response, such novel serotype design, antibody reduction plasmapheresis immunosuppression, evasion using empty capsids enveloped vectors. review, we examine mechanisms anti-AAV evaluate strengths weaknesses current strategies order provide an evidence-based recommendation on evading future AAV-mediated therapies
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