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, ...
Recent advancements in improving cross-species applicability of bioengineered AAV capsids [0.03%]
近年基因工程腺相关病毒载体衣壳跨物种应用研究进展
Haolai Pan,Nianci Li,Jieyu Qi et al.
Haolai Pan et al.
Adeno-associated virus (AAV) is widely accepted as a delivery vector for in vivo gene therapy due to its relatively low immunogenicity, minimal toxicity, sustained efficacy, and broad tropism. However, its unpredictable cross-species applic...
Assessment of F/HN-pseudotyped lentiviral vector following intravenous delivery to mice [0.03%]
静脉注射F/HN假病毒载体在小鼠体内的评估研究
Robyn V Bell,Nikhil B Faulkner,Anthony Sinadinos et al.
Robyn V Bell et al.
In pursuit of a gene transfer agent with efficient pulmonary transduction, the UK Respiratory Gene Therapy Consortium has developed a lentiviral vector pseudotyped with the envelope proteins, F and HN from Sendai virus (rSIV.F/HN). In contr...
Applications of genome editing technologies in the treatment of human diseases [0.03%]
基因编辑技术在人类疾病治疗中的应用
Jamal Alshorman,Mohammad Javad Mehran,Kingsley Miyanda Tembo et al.
Jamal Alshorman et al.
Genome editing has progressed from a laboratory capability for targeted DNA manipulation to a clinically relevant strategy for correcting, silencing, or regulating genes implicated in human disease. In this Review, we synthesize the mechani...