近日,加拿大病童医院联合波士顿儿童医院及德克萨斯大学西南医学中心的研究人员在Nature Medicine期刊发表了题为:AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single patient的论文。 Michael Pirovolakis是一名确诊...
[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...
研究人员将携带正常AP4M1基因的AAV载体注射到了迈克尔的脑脊液中,这些AAV载体会迅速地前往神经细胞发挥作用。 现在,这项1期临床试验的结果已经以“AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single p...
参考文献：[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, 20...
2025-02-24报道，国际顶尖医学期刊《柳叶刀》发表一篇题为：Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study 的研究论文。 这项首次人体（first-in-human）初步研究表明，AAV 基因疗法 rAAV8.hRKp.AIPL1 对患有 AIPL1 基因突变导致...
14.Boutin, Sylvie, et al. "Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors." Human gene therapy 21.6 (2010): 704-712.“基因治疗领域”相关文章...
NEJM最新综述 | 简述基因治疗的最新临床研究进展;邦耀生物官网 腺相关病毒(七):AAV载体对肝脏的亲和性可能是安全性问题根源;星耀研究院 AAV-Induced Cancer: Cause for Concern in Gene Therapy? At FDA meeting, gene therapy experts wrestle with field's blindspots...
4. NEJM最新综述 | 简述基因治疗的最新临床研究进展;邦耀生物官网 5.腺相关病毒(七):AAV载体对肝脏的亲和性可能是安全性问题根源;星耀研究院 6.AAV-Induced Cancer: Cause for Concern in Gene Therapy? 7.At FDA meeting, gene therapy experts wrestle with field's blindspots...
5.腺相关病毒(七):AAV载体对肝脏的亲和性可能是安全性问题根源;星耀研究院 6.AAV-Induced Cancer: Cause for Concern in Gene Therapy? 7.At FDA meeting, gene therapy experts wrestle with field's blindspots $诺华制药(NVS)$$bluebird bio(BLUE)$$辉瑞(PFE)$ #基因疗法##新药研发##安全性#...
2021年5月19日,英国伦敦大学学院的研究人员在 Science 子刊 Science Translational Medicine 期刊上发表了题为:Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism 的研究论文。 研究团队通过敲除SLC6A3基因,构建了多巴胺转运蛋白缺乏综合征...