Bilateral retinal rescue through unilateral AAV8-CYP4V2 gene delivery in Bietti crystalline dystrophy [0.03%]
贝蒂特结晶样视网膜色素上皮变性小鼠模型中单侧给药AAV8-CYP4V2实现双眼视网膜功能保护
Laura A Jenny,Yao Li,Stephen H Tsang
Laura A Jenny
SLC20A1 knockout to stably express a fusogenic gibbon ape leukemia virus envelope glycoprotein for lentiviral vector production [0.03%]
利用基因敲除的HEK293细胞生产含可融合的猿猴病毒40型衣壳蛋白包被液滴的慢病毒载体
Rodrigo José Nogueira,Ana Filipa Rodrigues,Daniel Alexandre Mestre et al.
Rodrigo José Nogueira et al.
Lentiviral vectors (LVs) derived from HIV-1 are widely used in gene therapy, with several approved products. However, establishing stable cell lines for constitutive LV production remains a challenge due to the cytotoxicity of vector protei...
Erratum: End-to-end characterization of AAV manufacturing process using charge detection mass spectrometry [0.03%]
Correction: 使用电荷检测质谱法对AAV制造过程进行端到端表征
Lohra M Young,Benjamin E Draper,Martin F Jarrold et al.
Lohra M Young et al.
[This corrects the article DOI: 10.1016/j.omtm.2025.101607.]. © 2025 The Author(s).
Preconditioning with StemRegenin 1 enhances human adipose stromal/stem cells proliferation, migration, and protection against antimycin A [0.03%]
前期用StemRegenin 1处理可增强人脂肪间充质/干细胞的增殖、迁移及抗安密妥能力
Jing Zhao,Bin Yu,Julie Fradette et al.
Jing Zhao et al.
Adipose-derived stromal/stem cells (ASCs) are promising for the treatment of many diseases, including tissue injury or degeneration associated with serious disease and high morbidity. The extent of cell therapy effectiveness, however, may b...
Transcriptional changes in non-human primate tissues after intrathecal delivery of serotype 9 adeno-associated viral vector: Insights into organ toxicities [0.03%]
注射血清型腺相关病毒载体后非人灵长类组织转录组变化：器官毒性的新见解
Fumiaki Aihara,Matthew Mardo,Vera Ruda et al.
Fumiaki Aihara et al.
Adeno-associated virus (AAV)-based gene transfer has brought transformative therapeutic benefits to patients with otherwise untreatable genetic diseases. However, treatment-related organ toxicities, particularly for high doses, remain a saf...
Harnessing DNA barcoding to enhance and sustain polyclonality in gene-edited hematopoietic stem cells [0.03%]
利用DNA条形码增强和维持基因编辑造血干细胞的多克隆性
Isabel Ojeda-Perez,Andrés Bustos,Sridhar Selvaraj et al.
Isabel Ojeda-Perez et al.
A challenge in gene editing for hematopoietic stem and progenitor cells (HSPCs) is achieving efficient editing while preserving long-term engraftment and clonal diversity. Tracking edited clones with high resolution is essential to understa...
From cold chain to ambient: Benefits, risks, and evidence across cell therapy logistics [0.03%]
从冷链到常温：细胞治疗物流中的益处、风险和证据
John Gostage,Daniel A Domingo-Lopez,Ruth Tarpey et al.
John Gostage et al.
With the exponential growth in the number of cell-based therapies, there is a need to look more closely at a critical factor that unites all these therapies, i.e., cryopreservation, which is currently deemed the gold standard for the storag...
Impact of pre-existing immunity on safety and biodistribution of a single AAV9 vectorintrathecal injection in cynomolgus monkeys [0.03%]
预先存在的免疫力对单次AAV9载体腰椎给药食蟹猴的安全性和生物分布的影响
Maria Vono,Tony Del Rio,Eduardo Magdaleno et al.
Maria Vono et al.
Adeno-associated virus (AAV) vectors are widely used for gene therapy as they deliver therapeutic genes with minimal toxicity. However, pre-existing anti-AAV immunity in a large percentage of humans poses a challenge to their efficacy and s...
Design and systematic evaluation of novel chemical modification patterns enable the identification of a potent siRNA targeting PCSK9 [0.03%]
设计和系统评估新型化学修饰模式可有效识别靶向PCSK9的高效siRNA分子
Ze-Ao Huang,Shuo Yang,Guang-Shen Ji et al.
Ze-Ao Huang et al.
Fully modified small interfering RNAs (siRNAs) have demonstrated remarkable advantages in medicine development since they enable the knockdown of the majority of disease-related proteins. However, unmodified siRNAs usually cannot be used fo...
Protocol of generation of tolerogenic dendritic cells affects their transcriptional and metabolic profiles leading to specific tolerogenic functions [0.03%]
耐受性树突细胞的生成协议影响其转录和代谢谱从而导致特定的耐受功能
Maaike Suuring,Giada Amodio,Mathieu Rouel et al.
Maaike Suuring et al.
Cell therapy is a promising approach for inducing tolerance and thus reducing the use of immunosuppressive drugs in several immune-mediated conditions. Tolerogenic dendritic cells (tolDCs) regulate antigen-specific tolerance and control the...