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翻訳と辞書　辞書検索 [ 開発暫定版 ] スポンサード リンク Self-complementary adeno-associated virus (scAAV) ： ウィキペディア英語版 Self-complementary adeno-associated virus (scAAV) Self-complementary adeno-associated virus (scAAV) is a viral vector engineered from the naturally occurring adeno-associated virus (AAV) to be used as a tool for gene therapy. Use of recombinant AAV (rAAV) has been successful in clinical trials addressing a variety of diseases.〔http://www.wiley.com//legacy/wileychi/genmed/clinical/〕 This lab-made progeny of rAAV is termed "self-complementary" because the coding region has been designed to form an intra-molecular double-stranded DNA template. A rate-limiting step for the standard AAV genome involves the second-strand synthesis since the typical AAV genome is a single-stranded DNA template. However, this is not the case for scAAV genomes. Upon infection, rather than waiting for cell mediated synthesis of the second strand, the two complementary halves of scAAV will associate to form one double stranded DNA (dsDNA) unit that is ready for immediate replication and transcription. The caveat of this construct is that instead of the full coding capacity found in rAAV (4.7-6kb) scAAV can only hold about half of that amount (≈2.4kb). In gene therapy application utilizing rAAV, the virus transduces the cell with a single stranded DNA (ssDNA) flanked by two Inverted Terminal Repeats (ITRs). These ITRs form hairpins at the end of the sequence to serve as primers to initiate synthesis of the second strand before subsequent steps of infection can begin. The second strand synthesis is considered to be one of several blocks to efficient infection. Additional advantages of scAAV include increased and prolonged transgene expression ''in vitro'' and ''in vivo'', as well as "higher in vivo DNA stability and more effective circularization." ==scAAV in Gene Therapy== scAAV is an attractive vector for use in gene therapy for many reasons. Its parent vector, AAV, is already being used in clinical trials. Due to a variety of scAAV serotypes available, scientists can choose a serotype which has properties desirable for their therapy. Selecting only a subset of cells improves specificity and lowers the risk of being inhibited by the immune system. Different scAAV and AAV serotypes can efficiently transfect a variety of cellular targets.〔Zincarelli C, Soltys S, Rengo G, Rabinowitz JE. "Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. ''Mol Ther.'' 2008 Jun;16(6):1073-80. PubMed PMID 18414476.〕 Like all vector-based approaches to gene therapy, one obstacle in translating therapies from pre-clinical trials into a human clinical application will be the production of large quantities of highly concentrated virus One disadvantage that scAAV faces is that due to robust gene expression, transgene products delivered via scAAV elicit a stronger immune response than those same transgenes delivered via a single-stranded AAV vector. 抄文引用元・出典: フリー百科事典『 ウィキペディア（Wikipedia）』 ■ウィキペディアで「Self-complementary adeno-associated virus (scAAV)」の詳細全文を読む スポンサード リンク 翻訳と辞書 : 翻訳のためのインターネットリソース Copyright(C) kotoba.ne.jp 1997-2016. All Rights Reserved.