Recombinant Adeno-associated viruses (AAVs) are an attractive vector for gene therapy delivery which may be blocked by AAV neutralising antibodies (NAbs). As Type 1 Diabetes (T1DM) is an endocrine disease of immunological origin, it is likely that NAb profiles are altered in the disease. In this study NAb to AAV2, AAV5, AAV6, and AAV8 in 72 subjects with T1DM and 45 non-diabetic patients were measured over a 4-year follow-up period. AAV2 NAb titres were significantly lower in non-diabetic subjects (P = 0.036). The T1DM group had more AAV8 NAb activity at baseline (P = 0.019), whilst after 4 years follow-up the T1DM group displayed developed increased AAV 5 (P = 0.03), 6 (P = 0.03) and 8 (P = 0.002) activity relative to the control group, however, overall AAV5 and 8 NAb levels were very low in patients <40. AAV NAb titre activity and prevalence generally appears higher in T1DM, however, low levels of AAV 5 and 8, particular in younger adult age groups at which T1DM can be targeted, could make these attractive vectors to target the disease.

中文翻译：

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1型糖尿病中与腺相关的病毒中和抗体。

重组腺相关病毒（AAVs）是一种有吸引力的载体，可用于基因治疗，可被AAV中和抗体（NAbs）阻断。由于1型糖尿病（T1DM）是具有免疫学起源的内分泌疾病，因此该疾病中NAb谱可能会改变。在这项研究中，在4年的随访期内，对72名T1DM患者和45名非糖尿病患者的AAV2，AAV5，AAV6和AAV8的NAb进行了测量。在非糖尿病受试者中，AAV2 NAb滴度显着降低（P = 0.036）。T1DM组在基线时具有更多的AAV8 NAb活性（P = 0.019），而在随访4年后，T1DM组显示出AAV 5（P = 0.03），6（P = 0.03）和8（P = 0.002）升高。相对于对照组，AAV5和8 NAb的总体水平非常低。