Genetic mutations in HSV-1 replication-defective vectors: Implications for their safety in gene therapy applications [0.03%]
HSV-1复制缺陷型载体中的基因突变：其在基因治疗应用中的安全性启示
Stefano Cattaneo,Barbara Bettegazzi,Selene Ingusci et al.
Stefano Cattaneo et al.
Beyond its well-known role in orofacial recurrent infections, HSV-1 has garnered significant attention in neuroscience for contrasting reasons. On one hand, it has been found to be involved in neurodegenerative processes; on the other, it m...
Correction: PPARγ is essential for protection against nonalcoholic steatohepatitis [0.03%]
correction：pparγ对于防治非酒精性steatohepatitis是必不可少的
C W Wu,E S H Chu,C N Y Lam et al.
C W Wu et al.
Published Erratum
Gene therapy. 2025 Sep 26. DOI:10.1038/s41434-025-00568-z 2025
Overcoming matrix effects in AAV neutralization assays with a constant serum concentration approach [0.03%]
用恒定血清浓度法克服AAV中和试验中的矩阵效应
Beatrix Kovács,Viktória Szabó,Domonkos Horváth et al.
Beatrix Kovács et al.
Sensitive quantification of adeno-associated virus (AAV) neutralizing antibodies (NAbs) is essential for gene therapy success. Conventional cell-based transduction inhibition assays often encounter matrix-induced artifacts resulting from va...
Correction: Identification of AAV variants with improved transduction of human vascular endothelial cells by screening AAV capsid libraries in non-human primates [0.03%]
纠正：通过在非人灵长类动物中筛选AAV衣壳文库来识别可改善人类血管内皮细胞转导的AAV变体
Maria Stamataki,Julia Lüschow,Christina Schlumbohm et al.
Maria Stamataki et al.
Published Erratum
Gene therapy. 2025 Sep 5. DOI:10.1038/s41434-025-00565-2 2025
AAV-mediated MUC5AC siRNA delivery to prevent mucociliary dysfunction in asthma [0.03%]
运用腺相关病毒介导的小干扰RNA递送系统预防哮喘患者黏液高分泌及纤毛功能障碍
Sahana Kumar,Maria Corkran,Yahya Cheema et al.
Sahana Kumar et al.
The main structural components of mucus produced in the lung are mucin 5B (MUC5B) and mucin 5AC (MUC5AC) where a relatively higher expression of MUC5B is typical in health. In the lungs of individuals with asthma, there is a shift from MUC5...
Identification of AAV variants with improved transduction of human vascular endothelial cells by screening AAV capsid libraries in non-human primates [0.03%]
通过在非人灵长类动物中筛选AAV衣壳文库来识别可改善人血管内皮细胞转导的AAV变异体
Maria Stamataki,Julia Lüschow,Christina Schlumbohm et al.
Maria Stamataki et al.
The development of targeted vector systems for gene therapy has made impressive progress during the last decade. Promising vector candidates were identified by screening large pools of adeno-associated virus (AAV) mutants in small animal mo...
Rapid detection of AAV8 binding antibodies in gene therapy candidates: development of a point-of-care approach [0.03%]
基因治疗候选人的AAV8结合抗体快速检测法：一种即时检测方法的发展
Alexander Kozikowski,Qing Wang,Cheng Yang et al.
Alexander Kozikowski et al.
Preexisting anti-AAV antibodies pose a significant challenge to the success of Adeno-associated Virus (AAV) mediated gene therapies, as they can diminish therapeutic effectiveness, restrict patient eligibility for treatment, and cause serio...
Gene therapy restores auditory function and rescues damaged inner hair cells in an aged Vglut3 knockout mouse model [0.03%]
基因治疗恢复听觉功能并挽救老化Vglut3敲除小鼠模型损伤的内毛细胞
Xingle Zhao,Hongen Xu,Chengyu Lian et al.
Xingle Zhao et al.
Vesicular glutamate transporter 3 (VGLUT3) is prominently expressed in the inner hair cells of the cochlea, playing a vital role in auditory signal transmission to the brain. Previous studies have shown that Vglut3 gene knockout in mice cau...
Correction: Expression of anti-amyloid CARs in microglia promotes efficient and selective phagocytosis of Aβ1‒42 [0.03%]
纠正：在小胶质细胞中表达抗淀粉样CARs可促进有效且选择性的Aβ1﹣42吞噬作用
Christina N Heiss,Rebecca Riise,Eric Hanse et al.
Christina N Heiss et al.
Published Erratum
Gene therapy. 2025 Aug 15. DOI:10.1038/s41434-025-00562-5 2025
AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects [0.03%]
杜氏肌营养不良症中微型dystrophin基因置换疗法的研究进展与前景展望
Katarzyna Chwalenia,Vivi-Yun Feng,Nicole Hemmer et al.
Katarzyna Chwalenia et al.
Duchenne muscular dystrophy (DMD) is caused by pathogenic sequence variants occurring in the DMD gene which lead to the loss of the dystrophin protein, a molecular 'shock absorber' that protects muscle from contraction-induced injury. The l...