The co-delivery of Programmed Death 1 ligands enhances and prolongs rAAV-mediated gene expression in pre-immunized mice [0.03%]
程序性死亡配体的共同递送增强并延长rAAV在预免疫小鼠中的介导基因表达
Piia Käyhty,Tiina Nieminen,Reetta A E Eriksson et al.
Piia Käyhty et al.
Immune responses against recombinant adeno-associated virus (rAAV) are one of the major obstacles in gene therapy. We investigated the potential of Programmed Death 1 ligands 1 and 2 (PD-L1/2) to protect AAV-transduced cells from immunologi...
Metadata assessment of non-human primate studies of AAV9 uncovers potential tissue specific variation in expression efficiency [0.03%]
非人灵长类AAV9研究的元分析揭示了潜在的组织特异性表达效率差异
Muhammad Shahrukh,Julianne R Sweeney,Tony Del Rio et al.
Muhammad Shahrukh et al.
Adeno-associated virus (AAV)-based gene therapies have garnered significant attention and investment due to their clinical success and potential to address underlying causes of many diseases. AAV vectors provide effective delivery of therap...
Secondary malignancy of T-cell origin after CAR T-cell therapy: EMA's conclusions from the evaluation of 38 suspected cases [0.03%]
CAR-T细胞治疗后的T细胞起源继发性恶性肿瘤：对38例疑似病例评估的EMA结论
Philipp Berg,Charlotte Bakker,Moritz Sander et al.
Philipp Berg et al.
This article provides a regulatory perspective on secondary malignancy of T-cell origin as a rare adverse reaction to the currently marketed CD19- or BCMA-directed chimeric antigen receptor (CAR) T-cell therapies. To assess the risk, causal...
Lipid nanoparticle mediated base editing of the Q344X rhodopsin mutation associated with retinitis pigmentosa [0.03%]
视杆细胞内视紫红质Q344X突变的碱基编辑及对色素性视网膜炎的作用研究
Victoria A C Palmgren,Miffy Hok Yan Cheng,Yao Zhang et al.
Victoria A C Palmgren et al.
Retinitis pigmentosa (RP) associated with mutations in the rhodopsin gene (RHO) is a significant cause of blindness. Here we report on the application of adenine base editing of the c.1030C>T (p.Q344X) RHO mutation linked to RP. Using a flu...
Intranasal versus intravenous AAV delivery: A comparative analysis of brain-targeting efficiency and peripheral exposure in mice [0.03%]
鼻内给药与静脉给药的腺相关病毒载体递送效果比较：对小鼠脑靶向效率及全身暴露量的影响分析
Chinwendu Chukwu,Jinyun Yuan,Hong Chen
Chinwendu Chukwu
Adeno-associated viruses (AAVs) hold significant promise for gene therapy targeting the central nervous system (CNS). However, current delivery methods are either invasive or cause significant systemic exposure. Intranasal (IN) delivery pre...
Precision rewriting of muscle genetics: therapeutic horizons of base and prime editing in skeletal muscle disorders [0.03%]
精准重写肌肉遗传学：肌营养不良基因基础编辑和prime编辑的治疗前景
Selin Saydam,Pervin Dinçer
Selin Saydam
Base Editing (BE) and Prime Editing (PE), novel precision tools of the CRISPR/Cas toolbox, have emerged as transformative technologies that enable highly specific genetic modifications. Their compatibility with post-mitotic cell types makes...
Real-world experience with gene therapy in Duchenne muscular dystrophy center readiness and patients safety: report from Qatar [0.03%]
基因治疗在杜氏肌营养不良症的现实世界经验、中心准备及患者安全情况报告——来自卡塔尔的报道
Mahmoud Fawzi Osman,Khalid Ibrahim,Claire Gleeson et al.
Mahmoud Fawzi Osman et al.
Duchenne Muscular Dystrophy is a rare, X-linked neuromuscular disorder that leads to progressive muscle degeneration, loss of ambulation, and premature mortality due to respiratory and cardiac failure. Historically, Duchennke Muscular Dystr...
Antibody-guided AAV vectors for antigen-specific delivery of suicide genes [0.03%]
抗体导向的AAV载体实现自杀基因的抗原特异性递送
Shojiro Inano,Hiroyuki Morita,Daishi Nakagawa et al.
Shojiro Inano et al.
Antibody-drug conjugates (ADCs) are a promising class of targeted cancer therapeutics, but their efficacy is often limited by off-target toxicity caused by payload leakage after internalization into tumor cells. To overcome this, we develop...
Ad astra per aspera: treatment challenges and opportunities for children with spinal muscular atrophy and tracheostomy [0.03%]
艰难困苦，玉汝于成：儿童脊髓性肌萎缩症合并气管切开术的治疗挑战与机遇
Yasemin Erbas,Richard S Finkel
Yasemin Erbas
Codon-optimized human Smad7 gene therapy enhances skeletal muscle mass and function in a murine model of Duchenne muscular dystrophy [0.03%]
优化的Smad7基因治疗增强杜氏肌营养不良小鼠模型的肌肉质量和功能
Buel D Rodgers,Christopher W Ward
Buel D Rodgers
Commercial development of gene therapeutics often requires transitioning to human payload genes as initial proof-of-concept studies in animal models often use taxa-specific orthologs. Such transitions also provide opportunities to address p...