4. Beginner’s guide to the production of AAV vectors for gene therapy–Mentored by a CDMO.https://biovian.com/beginners-guide-to-the-production-of-aav-vectors-for-gene-therapy-mentored-by-a-cdmo/ 5. Mark White. Addressing the Top Five Challenges to AAV-Based Gene Therapy with ddPCR.ht...
1.https://www.biospace.com/article/releases/fda-grants-fast-track-designation-to-sbt101-the-first-investigational-aav-based-gene-therapy-for-patients-with-adrenomyeloneuropathy-amn-/ 2.https://swanbiotx.com/investors-and...
基因治疗 (gene therapy)的概念起源于20世纪70年代初[1]。基因治疗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,从根本上解决传统疗法存在的一系列问题。基因治疗递送载体分为病毒载体和非病毒载体。相比非病毒载体,病毒载体具有更高转导效率、更强的工程化能力和高度特异性基因递送特点[2]。
5. Chen YC, Ma NX, Pei ZF, et al. A NeuroD1 AAV-Based Gene Therapy for Functional Brain Repair after Ischemic Injury through In Vivo Astrocyte-to-Neuron Conversion. Mol Ther 2020; 28(1): 217-34. 6. Wu Z, Parry M, Hou XY, et al. Gene therapy conversion of striatal astrocytes i...
https://www.fractyl.com/fractyl-health-demonstrated-significant-improvement-in-weight-loss-in-head-to-head-preclinical-obesity-studies-of-its-rejuva-glp-1-based-pancreatic-gene-therapy-candidate-glp-1-pgtx-compared-to-chr/ 设置星标,...
The invention relates to the field of andeno associated virus (AAV) based gene therapy in particular to the use of a combination of recombinant AAV transgene vectors with an immunosuppressant and/or empty AAV capsids. The invention further provides a composition and a kit of parts based on ...
[11]Askou AL, Alsing S, Benckendorff JNE, et al., Suppression of Choroidal Neovascularization by AAV-Based Dual-Acting Antiangiogenic Gene Therapy. Mol Ther Nucleic Acids. 2019 Jun 7;16:38-50. doi: 10.1016/j.omtn.2019.01.012.
Guidance for industry: Design and Analysis of Shedding Studies for Virus or Bacteria-Based Gene Therapy and Oncolytic Products. 2015 [9] Zen Z, Espinoza Y, Bleu T, Sommer JM, Wright JF. Infectious titer assay for adeno-associated virus vectors with sensitivity sufficient to detect single ...
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 基因突变导致的罕...
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