所述重组腺相关病毒为表达OGA蛋白的腺相关病毒,所述OGA蛋白是氨基酸序列是序列表中序列1的蛋白质。实验证明,通过尾静脉注射所构建的重组AAV9‑cTNT‑OGA病毒载体至所构建扩张型心肌病小鼠模型中,可以有效缓解小鼠扩张型心肌病表型,达到了治疗扩张型心肌病...
29, 30 The gene insert is bounded by the AAV2 inverted terminal repeats (ITRs), where one terminal repeat has the wild-type (WT) 144-nt sequence and the other ITR (ΔITR) is mutated to delete the AAV DNA resolution site and D sequence to direct preferential replication and packing of ...
At day 5 post-TAC we administered by retro-orbital injection AAV9 encoding MLK3 or TdTomato negative control, each under control of the CM-specific cTNT promoter (AAV-MLK3: n=12 TAC, 7 sham; AAV-TdTomato: n=10 Tac, 8 sham. At day 28 post-AAV9 injection, we measured cardiac ...
纯化后的蛋白用ni-nt亲和层析分离纯化上清中的重组蛋白。纯化后的重组蛋白经尿素浓度梯度递减的透析液,分步透析去除尿素而复性。透析缓冲溶液为50mmol/l tris-hcl(ph为7.5) 250mmol/l nacl 0.01mmol/l edta 1mol/l dtt其中尿素浓度梯度下降,其含量分别为5、2.5、1和0mol/l(各透析12h)透析后的蛋白于-80℃...
苷酸(nt)的线性、单链DNA分子。反向末端重复(ITR)在对病毒生命周期至关重要的蛋白 (Rep)的独特编码核苷酸序列以及重叠衣壳蛋白(Cap)的序列的侧翼。Cap基因编码形成衣 壳的VP蛋白(VP1、VP2和VP3)以及AAP(激活腺相关病毒(AAV)组装的蛋白)(Sonntag F, K,Schmidt K,等人The assembly‑activating protein promotes...
Food and Drug Administration (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) Meeting #70; Toxicity Risks of Adeno-Associated Virus (AAV) Vectors for Gene Therapy (2021) Google Scholar 51 M.G. Davey, J.S. Riley, A. Andrews, A. Tyminski, M. Limberis, J.E. Pogo...
We designed six different 20 nt-long gRNAs fully homologous to nt 1964–2002 of the human TMC1 mRNA (GenBank: NM_138691.3), using a strategy similar to the one previously described for the mouse Tmc1 sequence (Figure 4B). HAP cells transfected with the U6-gRNA constructs were sorted ...