An engineered helper plasmid generates differential E4orf6 and L4-22/33K gene expression increasing AAV vector production [0.03%]
一项工程辅佐质粒能够调节E4orf6和L4-22/33K基因的差异表达，从而提高AAV载体产量
Laura van Lieshout,Katrina Costa-Grant,Dimpal Lata et al.
Laura van Lieshout et al.
Helper plasmids that depend on native adenovirus gene expression have long been the standard for transient adeno-associated virus (AAV) production. Here, we demonstrate that engineering the required helper gene expression can greatly increa...
A gene therapy approach to prevent dilated intercellular space, a hallmark of gastroesophageal reflux disease [0.03%]
一种预防食管胃反流病特征的大胞间空间的方法的基因疗法研究
Gillian M Schiralli Lester,Jing Huang,Michael Barravecchia et al.
Gillian M Schiralli Lester et al.
The incidence of esophageal disease ranging from gastroesophageal reflux disease (GERD) to cancer has dramatically risen over the last 10 years. However, there have been limited descriptions of effective gene delivery methods for the esopha...
Precision correction of the GJB2 c.235delC mutation by prime editing in vitro [0.03%]
基于PE系统的GJB2基因c.235delC突变体的体外精准校正研究
Jiang Jin,Xiujuan Lv,Yangyang Li et al.
Jiang Jin et al.
Hereditary hearing loss, predominantly attributed to mutations in the GJB2 gene, constitutes a significant global health issue, with the c.235delC mutation being the most prevalent pathogenic allele in East Asian populations. Existing inter...
Bone- and muscle-targeted adeno-associated viral vectors enable tissue-selective vitamin D receptor knockdown in mice [0.03%]
针对骨骼和肌肉的腺相关病毒载体能够使小鼠特异性组织维生素D受体敲低
Alexandra K O&#x;Donohue,Julian Chu,Nicholas Norris et al.
Alexandra K O&#x;Donohue et al.
Vitamin D receptor (VDR) regulates musculoskeletal biology, but its adult, tissue-specific roles are difficult to resolve with germline or conventional conditional knockouts. We developed recombinant adeno-associated viral vectors (rAAVs) t...
SACF and GILA for in vitro transformation assessment of CRISPR/Cas9-edited cell therapy candidates: a multi-site study [0.03%]
基于CRISPR/Cas9编辑的细胞疗法候选产品的体外转化评估的SACF和GILA的多中心研究
Lena Dorsheimer,Joana Rita Ferreira,Bojing Wang et al.
Lena Dorsheimer et al.
Tumorigenicity is a key safety concern for CRISPR/Cas9-based gene therapies, yet its assessment remains challenging due to the lack of relevant and sensitive in vivo models. This HESI Global multi-site study evaluated the Soft Agar Colony F...
rAAV production cost analysis: Indication-specific cost per dose and reduction strategies [0.03%]
rAAV生产成本分析：特异性剂量的成本及减少策略
Min Tae Park,Dan Matuszek,Angela Andaluz et al.
Min Tae Park et al.
Recombinant adeno-associated virus (rAAV) vectors underpin many approved and late-stage gene therapies, yet manufacturing costs remain a major driver of therapy price. Here, we present the first comprehensive, platform-resolved cost analysi...
Gene augmentation therapy successfully treats mice with complete congenital stationary night blindness (cCSNB), improving retinal function and visual acuity [0.03%]
基因增强疗法成功治疗了完全性先天性静止性夜盲症小鼠，改善视网膜功能和视力
Nazarul Hasan,Cecilia A Attaway,Mattia Di Paolo et al.
Nazarul Hasan et al.
Recombinant adeno-associated virus (rAAV) mediated gene therapy is an effective approach for targeting therapeutic genes to retinal photoreceptors. Complete congenital stationary night blindness (cCSNB) is a genetically heterogeneous inheri...
Reversing cancer cell behavior using AI-guided CRISPR and quantum nanobiology: a systems-based approach to epigenetic reprogramming [0.03%]
利用人工智能引导的CRISPR和量子纳米生物学逆转癌细胞行为：表观遗传重编程的系统方法
Bakr Ahmed Taha,Ali J Addie,Adawiya J Haider et al.
Bakr Ahmed Taha et al.
Treatment effectiveness is hindered by the phenotypic plasticity of cancer and the genetic complexity of tumors. However, CRISPR-Cas-based medicines face challenges with specificity, off-target effects, and tumor heterogeneity adaptability....
FMR1 gene therapy restores translationally relevant phenotypes in a mouse model for fragile X syndrome [0.03%]
针对脆性X综合征小鼠模型的FMR1基因治疗恢复了翻译相关的表型
Richard K Lacher,Kari Henson,Lindsay N Wathen et al.
Richard K Lacher et al.
Fragile X Syndrome (FXS) is the most common inherited form of intellectual disability. It is caused by a trinucleotide expansion in the 5' UTR of the Fragile X messenger ribonucleoprotein 1 (FMR1) gene leading to loss of expression of Fragi...
Improving the precision of AAV lung gene therapy for SP-B deficiency using computationally derived lung-specific promoters [0.03%]
利用计算衍生的肺特异性启动子提高AAV载体肺部基因治疗SP-B缺乏症的精准性
Nicole Zielinska,Erin L Howard,Brenna A Y Stevens et al.
Nicole Zielinska et al.
Recombinant adeno-associated virus (rAAV) platforms have achieved significant success in clinical gene therapy; however, many still rely on ubiquitous promoters. This robust and widespread transgene expression can cause off-target effects, ...