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...
High resolution ES-DMA quantifies AAV capsid DNA content by electrical mobility to mass correlation [0.03%]
高分辨率ES-DMA通过电泳迁移率与质量的关联来量化AAV衣壳DNA含量
Preston Dennett,Lohra M Young,Benjamin E Draper et al.
Preston Dennett et al.
Recombinant Adeno-Associated Virus (rAAV) is the leading viral vector platform for gene therapy. A persistent challenge in rAAV manufacturing and quality control is accurate assessment of the DNA content of purified viral capsids. Empty par...
AAV8-mediated mouse/human PROC expression rescues thrombophilia in hereditary protein C-deficient mice [0.03%]
人和小鼠PROC基因腺相关病毒8载体转导逆转遗传性蛋白质C缺乏小鼠血栓病模型的凝血功能紊乱
Tingting Wu,Yanyi Tao,Hui Lu et al.
Tingting Wu et al.
Hereditary protein C (PC) deficiency, which is caused by PROC gene mutations, increases the risk of venous thromboembolism and offers limited treatment options. In this study, we developed adeno-associated virus serotype 8 (AAV8) vectors ca...
Godwin I Iroanya,Ilangovan Raju,Chandra Boosani et al.
Godwin I Iroanya et al.
Use of adeno-associated virus (AAV) vectors has revolutionized in vivo gene therapy, but the presence of pre-existing neutralizing antibodies remains a major barrier that can hinder clinical application. While large-animal models such as no...
Characterization of CAR-T cell factors that contribute to myeloid cell activation [0.03%]
有助于髓系细胞活化的CAR-T细胞因素的特征分析
Supreet Khanal,Md Kamal Hossain,Joseph Fischer et al.
Supreet Khanal et al.
Chimeric antigen receptor (CAR) T cell therapies have shown remarkable success in the treatment of hematologic cancers; however, their use is often accompanied by inflammatory toxicities, including cytokine release syndrome (CRS) and immune...
DOCTER: a genetically encoded switchable protein module for ERα-mediated transcriptional regulation [0.03%]
一种遗传编码的可切换蛋白质模块DOCTER用于雌激素受体介导的转录调控
Jinmin Wang,Jingfang Liu,Dandan Peng et al.
Jinmin Wang et al.
The inhibition of estrogen receptor (ER)-mediated genomic signaling in ER-positive cancer cells has long been a primary focus of therapeutic strategies. Here, we introduce a switchable competitive inhibition system for ERα-mediated transcr...
Research progress and development strategies of antibody-oligonucleotide conjugates [0.03%]
抗体寡核苷酸偶联物的研究进展与开发策略
Wenshuang Fan,Weijing Luan,Wenjun Yu et al.
Wenshuang Fan et al.
Antibody-oligonucleotide conjugates (AOCs) effectively integrate the delivery capability of antibodies with the specific gene regulatory function of oligonucleotides, offering a novel strategy for extrahepatic delivery. Unlike antibody-drug...
rAAV8 encapsidated HMR-001/z enables high efficiency hepatic transduction and restores hemostasis in hemophilic mice [0.03%]
rAAV8衣壳化HMR-001/z可高效转导肝脏并修复血友病小鼠的凝血功能
Xiaomo Wu,Jinying Zhong,Xiaorong He et al.
Xiaomo Wu et al.
Hemophilia A (HA), an X-linked bleeding disorder caused by factor VIII (FVIII) deficiency, is primarily managed with exogenous therapeutic agents; however, this treatment approach remains burdensome and fails to provide durable hemostatic c...
A dose-escalation and safety gene therapy study in a model of CMT4C neuropathy [0.03%]
评估基因治疗剂量和安全性的CMT4C模型研究
Elena Georgiou,Alexia Kagiava,Andreas Hentschel et al.
Elena Georgiou et al.
Charcot-Marie-Tooth disease type 4C is a demyelinating neuropathy caused by loss of function mutations in the SH3TC2 gene, that is highly expressed in myelinating Schwann cells. We generated and tested a clinical stage vector with a minimal...