All-in-one vectors for epigenetic CRISPR inhibition of DUX4-fl in facioscapulohumeral muscular dystrophy [0.03%]
用于面肩肱型肌营养不良症中DUX4-FL表观遗传学CRISPR抑制的一体化载体
Charis L Himeda,Takako I Jones,Peter L Jones
Charis L Himeda
Facioscapulohumeral muscular dystrophy (FSHD) is caused by incomplete epigenetic silencing of the disease locus, leading to pathogenic misexpression of DUX4 in skeletal muscle. Previously, we showed that CRISPR inhibition (CRISPRi) using se...
Method validation and assessment of the biodistribution and shedding for adenovirus vector-based vaccine using qPCR and dPCR [0.03%]
基于qPCR和dPCR的腺病毒载体疫苗的方法验证及组织分布和排泄研究
Yoichi Tanaka,Sayaka Hamano,Akiko Ishii-Watabe et al.
Yoichi Tanaka et al.
Assessment of the biodistribution and shedding is required for the development of viral vector-based vaccines. Vector copy number is commonly quantified using digital PCR (dPCR) or quantitative PCR (qPCR). However, the regulatory guidelines...
Erratum: Preclinical evaluation of the efficacy and safety of AAV1-hOTOF in mice and nonhuman primates [0.03%]
AAV1-hOTOF的药效学和安全性评价的补充：基于小鼠和非人灵长类模型的临床前评估中文标题：小干扰RNA沉默DDAH1减轻心肌梗死后的心脏重构及改善心脏功能
Longlong Zhang,Hui Wang,Mengzhao Xun et al.
Longlong Zhang et al.
[This corrects the article DOI: 10.1016/j.omtm.2023.101154.]. © 2025 The Author(s).
Safety through design: Expanding options for spinal muscular atrophy gene therapy [0.03%]
设计保障安全：扩大脊髓性肌萎缩症基因治疗的选择范围
Ewout J N Groen,Renske I Wadman
Ewout J N Groen
Effect of disease progression on CSF-directed AAV gene therapy in a large brain animal model of lysosomal storage disease [0.03%]
发病进展对大型动物脑性溶酶体贮积症基因治疗效果的影响
Jacqueline E Hunter,Caitlyn M Molony,Wojciech K Panek et al.
Jacqueline E Hunter et al.
The lysosomal storage disease alpha-mannosidosis (AMD) is caused by a genetic deficiency of lysosomal alpha-mannosidase, leading to the widespread presence of storage lesions in the brain and other tissues. Animal models of lysosomal diseas...
Wenhao Lin,Min Zhang,Sirui Zheng et al.
Wenhao Lin et al.
Leukodystrophies form a group of rare genetic disorders characterized by the progressive degeneration of white matter in the brain, often presenting in childhood with life-threatening consequences. Current therapeutic options are limited, p...
Erratum: Developing a minimally invasive gene therapy for multiple sclerosis [0.03%]
小颜：开发一种治疗多发性硬化症的微创基因疗法（英文）
Paul J H Nijhuis,Maurits Romijn,Roy Honing et al.
Paul J H Nijhuis et al.
[This corrects the article DOI: 10.1016/j.omtm.2025.101504.]. © 2025 The Author(s).
Enhanced B cell electroporation efficiency via inhibition of DNA-induced apoptosis and pyroptosis with pan-caspase inhibitor [0.03%]
广谱半胱氨酸凋亡抑制剂通过抑制DNA诱导的细胞凋亡和细胞焦亡现象提高B细胞电转效率
Gui-Lin Yang,Deng-Gao Wang,Xiang Zhao et al.
Gui-Lin Yang et al.
B cells have immense potential as gene therapy carriers because of their ability to secrete therapeutic proteins and mediate immunological memory and tolerogenic functions. However, efficient gene delivery into primary B cells remains a cha...
Špela Malenšek,Duško Lainšček,Hana Esih et al.
Špela Malenšek et al.
Cardiovascular diseases, especially atherosclerosis, are the main cause of death in the whole world. The risk can be reduced by lowering the serum low-density lipoprotein cholesterol by targeting proprotein convertase 9 (PCSK9) through geno...
Neonatal gene therapy effectively prevents disease manifestations in a murine model of Mucopolysaccharidosis type I [0.03%]
一种黏多糖贮积症I型小鼠模型的有效新生儿基因治疗可有效预防疾病表现形式
Giada De Ponti,Ludovica Santi,Giorgia Dina et al.
Giada De Ponti et al.
Mucopolysaccharidosis type I (MPS-I) is a rare pediatric disease caused by mutations in the α-L-iduronidase (IDUA) gene encoding for a lysosomal enzyme involved in glycosaminoglycan metabolism. While newborns with the severe Hurler variant...