Multi-targeting zinc finger nuclease vector unsilences paternal UBE3A in a mouse model of Angelman syndrome [0.03%]
一种安格曼综合征小鼠模型的多目标锌指核酸酶载体无法激活父系UBE3A基因
Hannah O Bazick,Lucas M James,Bonnie Taylor-Blake et al.
Hannah O Bazick et al.
Angelman syndrome (AS) is a severe neurodevelopmental disorder most often caused by deletion of the maternally inherited UBE3A allele (matUBE3A). In neurons, a long non-coding antisense RNA (Ube3a-ATS) silences the paternally-inherited UBE3...
Correction: Anti-inflammatory effect of MAPK phosphatase-1 local gene transfer in inflammatory bone loss [0.03%]
关于MAPK磷酸酶-1基因局部转移的抗炎效应在炎性骨丢失中的纠正评论
H Yu,Q Li,B Herbert et al.
H Yu et al.
Published Erratum
Gene therapy. 2025 Nov 24. DOI:10.1038/s41434-025-00579-w 2025
Murine toxicology assessment of avgn7.2, a novel gene therapeutic for inclusion body myositis and other muscle wasting diseases [0.03%]
评估AVGN7.2的鼠类毒性，AVGN7.2是IBM和其他肌肉萎缩症的一种新型基因治疗药物
Sarah K Herring,Buel D Rodgers
Sarah K Herring
Sporadic inclusion body myositis (IBM) is a highly debilitating muscle degenerative and rare disease of the middle aged and elderly. Because immunosuppressants fail to prevent muscle wasting in IBM patients and can even exacerbate it, drugs...
AAV-mediated BDNF and GAS6 muscle delivery delays disease onset in SOD1G93A ALS mice [0.03%]
AAV介导的BDNF和GAS6肌肉递送延迟SOD1G93A渐冻症小鼠发病时间
Yicong Le,Gongjie Liu,Shenzhe Wu et al.
Yicong Le et al.
Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease, with limited treatments. Gene therapy offers an alternative strategy for treating a large portion of ALS patients, however, the disparate genetic alterations in ALS c...
Therapeutic in vivo genome editing: innovations and challenges in rAAV vector-based CRISPR delivery [0.03%]
体内治疗性基因编辑：基于rAAV载体的CRISPR传递的创新和挑战
Jin-Seok Gil,Soyeon Lee,Taeyoung Koo
Jin-Seok Gil
The integration of CRISPR systems with recombinant adeno-associated virus (rAAV) vectors has opened new possibilities for therapeutic genome editing, offering potential treatments for both genetic and non-genetic disorders. rAAV vectors hav...
How far have we come? From understanding collagen disorders to advancing treatment strategies [0.03%]
我们取得了多少进展？从理解胶原蛋白紊乱到推进治疗策略
James Williamson,Yan Yu Chong,Wai Kit Hung et al.
James Williamson et al.
Cardiac troponin T promoters lead the way: optimizing cardiac specificity in AAV-mediated gene therapy [0.03%]
心脏肌钙蛋白T启动子引领方向：优化AAV介导的心脏特异性基因治疗
Anagh Astavans,Andreas S Barth
Anagh Astavans
Therapeutic precision gene editing of cholesterol pathways as a gene therapy strategy for cardiovascular disease [0.03%]
胆固醇通路的精准治疗性基因编辑作为心血管疾病的基因治疗方法
Ezgi Erbasan,Melike Aliciaslan,Fulya Erendor et al.
Ezgi Erbasan et al.
Hypercholesterolemia, defined by high low-density lipoprotein cholesterol levels, critically increases the risk of atherosclerotic cardiovascular disease, which represents the foremost cause of worldwide morbidity and mortality. While estab...
Characteristics of long-term follow-up studies for gene therapies registered on ClinicalTrials.gov [0.03%]
ClinicalTrials.gov注册的长期随访基因治疗研究的特点分析
Carolyn Riley Chapman,Ava Glazier,Emina Berbić et al.
Carolyn Riley Chapman et al.
Guidance from the U.S. Food and Drug Administration and other regulatory agencies recommends long-term follow-up (LTFU) studies of gene therapy recipients. The primary objective of LTFU studies is to understand the long-term safety of gene ...
Die Wu,Shengqian Ding,Nian Liu et al.
Die Wu et al.
Genetic/genomic manipulation techniques (gene transfer/delivery, gene editing, etc.) have become more and more mature, and the illegal use as gene doping in sports has drawn attentions. World Anti-Doping Agency (WADA) strictly prohibits gen...