根据Clinical http://Trials.gov上统计,目前有超过250多种AAV基因治疗临床项目在全球范围开展,迄今为止,美国FDA已批准了2个AAV基因治疗法:第一种为Luxturna,于2017年获得批准用于治疗莱伯氏先天性黑蒙症,这是一种遗传性视网膜疾病;第二种为Zolgensma,于2019...
而AAV基因治疗正是针对这些问题而发展的一种新兴技术,它的出现有望重新定义基因疗法的未来。 基因治疗(Gene Therapy)是指将外源正常基因导入靶细胞,以纠正或补偿缺陷和异常基因引起的疾病,从而达到治疗疾病的目的。一直以来,传统大/小分子药物主要针对蛋白靶点发挥作用,这也使得其适应症有限,特别对于基因因素占据主要发病...
21年12月 ,在Science Advances期刊上也发表了一篇“Dual-vector gene therapy restores cochlear amplification and auditory sensitivity in a mouse model of DFNB16 hearing loss”研究论文,波士顿儿童医院首创一种双AAV载体递送的基因治疗技术,成功...
in trans for high yields of recombinant AAV, recombinant AAV vectors for use in gene therapy, novel packaging cell lines which obviate the need for cotransfection of vector and helper plasmids, helper plasmids and vector plasmid backbone constructs, a reporter assay for determining AAV vector yield...
[1] Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019 May;18(5):358-378. [2] AAV manufacturing for clinical use: insights on current challenges from the upstream process perspective[J]. Current Opinion in Biomedical Engineering, 2021, 20: ...
[1] Wang D. et al. , Adeno-associated virus vectoras a platform for gene therapy delivery [J]. NATURE REVIEWS DRUG DISCOVERY,2019, 18(5): 358-378.[2] "https://luxturnahcp.com/about-luxturna/mechanism-of-action/"[3] Mendell J.R. et al. , Current ClinicalApplications of In Vivo...
6. Christine Kay. AAV Gene Therapy Trials To Watch. July/August 2021. https://retinatoday.com/articles/2021-july-aug/aav-gene-therapy-trials-to-watch 7. Bulcha, J.T., Wang, Y., Ma, H. et al. Viral vector platforms within the gene therapy landscape. Sig Transduct Target Ther (2021...
【预订】Adeno-Associated Virus (AAV) Vectors in Gene Therapy 9783642802096 美国库房发货,通常付款后3-5周到货! 作者:Berns出版社:暂无出版社信息出版时间:2012年01月 手机专享价 ¥ 当当价 降价通知 ¥1217 配送至 北京 至 北京市东城区 服务 由“中国进口图书旗舰店”发货,并提供售后服务。
近日,Nature子刊Nature Reviews Drug Discovery发表了题为:Adeno-associated virus vector as a platform for gene therapy delivery的综述长文。针对“腺相关病毒(AAV)载体作为基因治疗递送的平台”展开讨论。 这篇综述论文的通讯作者为高光坪教授,高光坪教授是全球基因治疗领域领导者之一,美国基因与细胞治疗学会主席、麻省...
Santiago-Ortiz, J.L. and D.V. Schaffer, Adeno-associated virus (AAV) vectors in cancer gene therapy. Journal of Controlled Release, 2016. 240: p. 287-301. Naso, M.F., et al., Adeno-Associated Virus (AAV) as a Vector for Gene Therapy. BioDrugs, 2017. 31(4): p. 317-334. ...