Nonclinical toxicity study duration in AAV gene therapy development: Evidence from industry survey supports adequacy of short-term assessments [0.03%]
AAV基因疗法开发中的非临床毒性研究持续时间：来自行业调查的证据支持短期评估的充分性
Basel T Assaf,David Edwards,Shawn Jay Berens et al.
Basel T Assaf et al.
Adeno-associated virus (AAV)-based gene therapies are emerging as transformative treatments for serious diseases; however, determining the optimal duration of nonclinical toxicity studies remains a key regulatory and scientific question. To...
Jane Hsi-Bell,Mario Mietzsch,Robert McKenna
Jane Hsi-Bell
Over the past ∼25 years, structural studies of adeno-associated virus (AAV) capsids have greatly aided our understanding of the biology of these single-stranded DNA viruses and provided insights into their utilization as gene therapy vecto...
Tailoring monocyte-derived tolerogenic cell therapies to the needs; what's in a name? [0.03%]
按需定制单核细胞来源的耐受性细胞疗法；名字很重要吗？
Tatjana Nikolic,Bart O Roep
Tatjana Nikolic
Molecular Therapy Advances: Building the bridge between discovery and cure [0.03%]
分子疗法进展：连接发现与治愈的桥梁
Mohamed Abou-El-Enein
Mohamed Abou-El-Enein
Characterization of difficult-to-remove host cell proteins in adeno-associated virus downstream processing [0.03%]
腺相关病毒下游处理过程中宿主蛋白的表征
Thomas M Leibiger,Lie Min,Kelvin H Lee
Thomas M Leibiger
A scalable, serotype-agnostic, and fully chromatographic downstream platform was developed for the purification of adeno-associated virus (AAV) based on affinity chromatography (AC), anion-exchange chromatography (AEX), and multi-modal poli...
Gene therapy supports long-term reconstitution of patient hematopoietic stem cells in deficiency of adenosine deaminase 2 [0.03%]
腺苷脱氨酶2缺乏症患者的基因治疗可长期重建患者造血干细胞
Chiara Rigamonti,Dimitri Bulté,Federica Barzaghi et al.
Chiara Rigamonti et al.
Deficiency of adenosine deaminase 2 (DADA2) is a monogenic autoinflammatory disorder characterized by systemic inflammation, vasculopathy, immunodeficiency, and bone marrow failure. Current therapies-including anti-TNF agents and allogeneic...
Intrathalamic delivery of adeno-associated viral vector expressing progranulin as gene therapy for GRN-related frontotemporal dementia [0.03%]
针对GRN相关额颞叶痴呆的腺相关病毒载体介导的脑内プログランリン基因治疗
Youn Bok Lee,Do Young Lee,Zoe Walker et al.
Youn Bok Lee et al.
GRN mutations leading to progranulin haploinsufficiency can cause frontotemporal dementia. AVB-101, an investigational gene therapy comprising an adeno-associated virus construct expressing codon-optimized human GRN under a neuronal promote...
End-to-end characterization of AAV manufacturing process using charge detection mass spectrometry [0.03%]
采用电荷检测质谱法对AAV生产工艺进行端到端表征
Lohra M Young,Benjamin E Draper,Martin F Jarrold et al.
Lohra M Young et al.
Charge detection mass spectrometry (CD-MS) has previously been shown to be effective for analyzing predominantly clean bulk drug substances and drug products of recombinant adeno-associated virus (rAAV). CD-MS holds potential for assessing ...
Adeno-associated virus serotype 9 structural heterogeneity and stability characterized by charge detection mass spectrometry [0.03%]
基于电荷检测质谱法测定的腺相关病毒9型衣壳结构异质性和稳定性
Zachary M Miller,Li F Lin,David V Schaffer et al.
Zachary M Miller et al.
The variability in structure and stability of adeno-associated virus serotype 9 (AAV9) in response to changes in buffer composition, pH, and temperature was investigated using charge detection mass spectrometry (CDMS). AAV9 virus-like parti...