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Product Name	AAV-8 Titration ELISA
Description	Enzyme Immunoassay for the Quantitative Determination of AAV Serotype 8 Particles in Cell Culture Supernatants and Purified Virus Preparations
Background	Adeno-associated virus (AAV) is a non pathogenic ssDNA virus that is a topic of intense study in gene therapy. The virus transduces a wide variety of dividing and non-dividing cells showing long-term gene expression with no cellular immune response. AAV has been used in several clinical trials (e.g. FIX, CFTR, Parkinson’s disease, Canavan disease) showing no serious vector-related adverse effects. Methods for the characterisation of AAV preparations currently include titration ELISA, realtime PCR, DNA dot blot, determination of transducing units, infectious center assay, SDSPAGE or electron microscopy. Immunotitration by ARP's AAV8 Titration ELISA offers a fast, sensitive and reproducible method for titration of intact AAV8 wt virions, AAV8 recombinant virions or assembled and intact empty AAV8 capsids.
Principle	The assay is based on the sandwich ELISA technique. A monoclonal antibody specific for a conformational epitope on assembled AAV8 capsids (ADK8) is coated onto microtiter strips and is used to capture AAV8 particles from the specimen. Captured AAV particles are detected in two steps. First a biotin-conjugated monoclonal antibody to AAV8 (ADK8) is bound to the immune complex. In the second step streptavidin peroxidase conjugate reacts with the biotin molecules. Addition of substrate solution results in a color reaction which is proportional to the amount of specifically bound viral particles. The absorbance is measured photometrically at 450 nm. The kit control provided contains an AAV 2/8 particle preparation, including empty capsids. It shows a typical titration curve when used in dilutions of steps of two (Fig. 1). It allows the quantitative determination of samples of an unknown particle titer (immunological titer) and the calibration of an in-house AAV8 preparation (e.g. infectious titer, transducing units, DNA dot blot titer).
Storage	2-8C
Intended Use	Limited Use Label License: Research Use OnlyProduct is exclusively licensed to PROGEN Biotechnik GmbH. The use of these products for the development, manufacturing and sale of secondary products/derivatives which are based on the purchased products and/or which include the purchased product require a royalty based sub-license agreement.

American Research Products抗AAV产品识别腺相关病毒（AAV）的单克隆抗体细小病毒家族的成员腺相关病毒A（AAV）是小型单链DNA病毒，可感染人类和某些非人类灵长类动物。目前，已经报道了13种人类和非人类灵长类动物AAV血清型和150种基因型。AAV目前被认为是非致病性的，大多数感染AAV的人只会产生轻度的免疫反应。腺伴随病毒衣壳将基因带入分裂细胞和静止细胞中，在那里它们可以将遗传信息插入宿主基因组。已显示出不同的AAV血清型可证明特定组织和细胞类型的向性。这是由于其氨基酸序列和衣壳结构的变化，使它们能够与不同的宿主细胞受体相互作用。AAV的这些特征已被用于基因治疗，重组AAV病毒已被工程化以将治疗性基因携带到各种宿主细胞中。目前正在进行各种利用不同AAV血清型的临床试验，包括：  据报道，AAV1显示出骨骼肌，心肌，视网膜色素上皮和中枢神经系统（CNS）的向性增强。AAV1目前用于α-1抗胰蛋白酶缺乏症，肢带肌肉萎缩症，杜兴肌营养不良症和心力衰竭治疗的临床试验研究中。AAV2在肾脏组织，视网膜色素上皮，感光细胞和中枢神经系统中表现出向性。AAV2目前用于各种疾病的临床试验研究中，包括B型血友病，Duchenne肌营养不良症，帕金森氏病，年龄相关性黄斑变性，脉络膜炎和类风湿性关节炎。AAV5证明视网膜色素上皮，感光细胞，肺组织和中枢神经系统的向性。AAV5目前用于B型血友病和类风湿关节炎基因治疗的临床试验研究中。AAV6证明骨骼肌和肺组织有向性。AAV6已在临床试验中用于治疗严重的心力衰竭。AAV8对包括肝脏，胰腺，骨骼肌，心脏和中枢神经系统在内的多种组织表现出向性性。AAV8目前在临床试验中用于一系列疾病，包括血友病A，血友病B，X连锁肌管肌病和丙型肝炎基因治疗。AAV9 表现出骨骼肌，心脏，肝脏，肺部和中枢神经系统的向性。AAV9目前用于庞贝病，巴滕病和脊髓性肌萎缩症基因治疗的临床试验研究中。