Long-Term Follow-Up of Patients Receiving Cell and Gene Therapy Products [0.03%]
细胞和基因治疗产品的接受者长期随访研究
Laszlo Irinyi,Barbara Mintzes,Julia Warning et al.
Laszlo Irinyi et al.
Cell and gene therapies present unique challenges for long-term follow-up as they may lead to adverse events that could emerge years after treatment. Long-term follow-up helps identify potential delayed adverse events, such as oncogenesis o...
Patient Dies after Treatment with Intellia CRISPR Therapy in Phase III Trial [0.03%]
CRISPR疗法3期临床受试者死亡事件
Alex Philippidis
Alex Philippidis
Multivariate Process Optimization for Fixed-Bed Bioreactor-Based AAV Production Improves Total Batch Yield [0.03%]
基于固定床生物反应器的AAV生产多变量过程优化可提高总批次产量
Ruchita Selot,Ashish Khaparde,Sharath Babu G R et al.
Ruchita Selot et al.
Adeno-associated viral vectors (AAVs) are promising tools for gene therapy. However, scaling up the production of AAVs to produce high-quality vectors at high yields for clinical purposes has proven to be challenging. In the present study, ...
Stable and Predictable Lentiviral Vector Production at Clinical Scale [0.03%]
临床规模的慢病毒载体生产工艺的稳定性和可预测性
Niels Heinz,Laura Mosbacher,Lisa C Cordie et al.
Niels Heinz et al.
Gene therapy has become a widely accepted treatment for inherited or acquired genetic diseases. Lentiviral vectors are of particular interest because of their favorable biosafety profile and ability to introduce their therapeutic cargo into...
Regulatory Perspective for Biologics License Application of Recombinant Adeno-Associated Virus Products in China [0.03%]
我国重组腺相关病毒产品生物制品许可申请监管要素思考
Jing Cui,Wenbo Wang,Dan Liu et al.
Jing Cui et al.
Recombinant adeno-associated virus (rAAV) vectors have emerged as a leading viral vector for in vivo gene therapy because they offer significant advantages over other viral vectors, such as stable physicochemical properties, low pathogenici...
Ethical, Legal, and Social Issues (ELSI) in Human Somatic Gene Therapy Clinical Research: A Scoping Review [0.03%]
人类体细胞基因治疗临床研究中的伦理、法律和社会问题(ELSＩ)的系统综述
Carolyn Riley Chapman,Mena Shaikh,Ava Glazier et al.
Carolyn Riley Chapman et al.
Dozens of gene therapies (GTs) have received regulatory approval, and hundreds more are in various research phases. To characterize the ethical, legal, and social implications (ELSI) associated with the clinical development of this relative...
Stathmin 1 Attenuates the Myocardial Fibrosis in Rat Model of Heart Failure [0.03%]
肌抑素1通过抑制心力衰竭模型大鼠心肌纤维化起到保护作用
Ting Liu,Zhuo Li,Yun Sun et al.
Ting Liu et al.
Myocardial fibrosis represents a maladaptive response to some pathological stimulus affecting the heart's functions, predisposing to arrhythmia and ultimately heart failure (HF). Stathmin 1 (Stmn1) is a core protein in regulating the format...
uniQure Gene Therapy Significantly Slows Huntington Disease Progression [0.03%]
uniQure基因疗法可显著减缓亨廷顿病的进展
Alex Philippidis
Alex Philippidis
Pharmacokinetic and Pharmacodynamic Evaluation of Bidridistrogene Xeboparvovec in an Aged Murine Model of Limb-Girdle Muscular Dystrophy Type 2E/R4 [0.03%]
评估Bidridistrogene Xeboparvovec在老年小鼠肢带肌营养不良症2E/R4模型中的药代动力学和药效学性质研究
Stephen Baine,Jared Cui,Young-Eun Seo et al.
Stephen Baine et al.
Limb-girdle muscular dystrophy type 2E/R4 (LGMD2E/R4) is an ultra-rare autosomal recessive disorder caused by mutations in SGCB, the gene that encodes for β-sarcoglycan (SGCB), a component of the dystrophin-associated protein complex that ...
Characteristics of Transfection Reagents that Achieve High Production of Recombinant Adeno-Associated Virus Vectors [0.03%]
高效生产重组腺相关病毒载体的转染试剂特性分析
Kyoko Masumi-Koizumi,Emi Ito-Kudo,Yuzhe Yuan et al.
Kyoko Masumi-Koizumi et al.
Recombinant adeno-associated virus (rAAV) vectors are among the most effective for gene therapy. A significant advancement in rAAV vector production is developing the triple-plasmid transfection method, which remains the most widely used te...