Elucidation of the binding interaction interface between AAV serotype 11 capsid protein and host nuclear import proteins [0.03%]
AAV血清型11衣壳蛋白与宿主核输入蛋白结合作用界面的阐明
Mikayla Hoad,Sepehr Nematollahzadeh,Justin A Roby et al.
Mikayla Hoad et al.
Adeno-associated viruses (AAVs) are widely acknowledged as versatile vectors for gene therapy due to their non-pathogenic nature, inherent capacity for tissue-specific targeting, and their potential for customizable engineering. The N termi...
Retinal organoids mirror CRISPR-Cas9 gene editing efficiency observed in vivo [0.03%]
视网膜类器官反映体内CRISPR-Cas9基因编辑效率
Juliette Pulman,Hugo Malki,Paul Oudin et al.
Juliette Pulman et al.
Human retinal organoids are in vitro 3D structures that recapitulate key molecular and structural characteristics of the in vivo retina. They include all essential retinal cell types including photoreceptors, making them relevant models for...
AAV-mediated gene therapy for the CNS: The case for early treatment [0.03%]
针对中枢神经系统的AAV基因治疗：早期治疗的案例分析
Luanna E Liebscher Vidal,Jose M Martinez-Navio
Luanna E Liebscher Vidal
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...