Growing clinical evidence for successful in vivo generation of CAR T cells directed against hematologic malignancies [0.03%]
针对血液系统恶性肿瘤的CAR-T细胞体内成功生成的临床证据越来越多
Attila Sebe,Christian J Buchholz
Attila Sebe
Addressing the package: Cell-specific gene delivery using lentiviral vectors [0.03%]
针对目标的载体包装：利用慢病毒载体进行细胞特异性基因传递
Anjali Shrivastava,Felix L Warnecke,Juliane W Schott et al.
Anjali Shrivastava et al.
Lentiviral vectors have revolutionized gene therapy by efficient and stable transduction of dividing and non-dividing cells, their large packaging capacity, and their compatibility with pseudotyping to alter viral tropism. The vesicular sto...
DDX3X overexpression in mice can cause rapid tissue-specific toxicity and mortality [0.03%]
DDX3X过表达可导致小鼠组织特异性毒性和致死性
Andrea Boitnott,Yuhui Hu,Mary Wight-Carter et al.
Andrea Boitnott et al.
DEAD-Box Helicase 3 X-Linked (DDX3X) is a ubiquitously expressed RNA helicase with diverse cellular roles implicated in a neurodevelopmental disorder called DDX3X syndrome. Although DDX3X is a leading genetic cause of intellectual disabilit...
Multivalent DNA vaccines induce potent cellular responses and prolong BCG-mediated control of Mycobacterium tuberculosis [0.03%]
多价DNA疫苗可诱导强大的细胞反应并延长BCG介导的对结核分枝杆菌的控制作用
Elizabeth M Parzych,Mamadou A Bah,Nicholas J Tursi et al.
Elizabeth M Parzych et al.
Mycobacterium tuberculosis remains a leading public health concern worldwide. While BCG vaccination is effective at protecting infants/ children, efficacy wanes over time. Advancements in synthetic DNA technology have resulted in the delive...
Zijia Guo,Jiayan Fu,Luna Ran et al.
Zijia Guo et al.
Messenger RNA (mRNA)-based vaccines, including circular RNA (circRNA) and self-amplifying RNA (saRNA), have emerged as the most transformative platforms, offering tremendous potential in prophylactic and therapeutic medicine, particularly i...
In vivo-directed evolution identifies AAV-WM04 as a next-generation vector for potent and sustained hearing restoration in DFNB9 [0.03%]
体内定向进化鉴定出AAV-WM04是一种针对DFNB9的高效且持久的治疗性载体病毒.vector病毒这里可以不译或者翻译为基因载体。
Yong Tao,Cenfeng Chu,Zhenzhe Cheng et al.
Yong Tao et al.
Efficient and cell-specific gene delivery to cochlear inner hair cells (IHCs) remains a major challenge for inner ear gene therapy. Here, we identify and characterize a novel AAV2-derived capsid, AAV-WM04, that enables highly efficient and ...
miR-146a Mimic Therapy Protects Against Platelet-Mediated Thrombosis [0.03%]
microRNA-146a 模拟物治疗可防治血小板介导的血栓形成
Sonia Águila,Juan C García-Cañaveras,Laura Zapata-Martínez et al.
Sonia Águila et al.
miR-146a exhibits variable expression levels in humans due to rs2431697, which is associated with cardiovascular events. However, the functional mechanisms linking this genotype with the occurrence of thrombotic events remain unknown. Here,...
Programmable engineered bacteria manipulate metabolism and remodel the TME in situ for enhancing adoptive cell therapy [0.03%]
可控工程细菌重塑肿瘤微环境增强过继细胞治疗功效
Jin Chen,Tianliang Liu,Xiumin Liu et al.
Jin Chen et al.
In solid tumors, the efficacy of adoptive T cell therapy (ACT) is limited by a metabolically constrained tumor environment that undermines T-cell persistence, fitness and infiltration. Here we engineered a hypoxia-activated bacterial hybrid...
C9orf72-associated poly-GR in skeletal muscle leads to neuromuscular junction deficits and muscle atrophy [0.03%]
C9orf72相关的聚体-GR导致神经肌肉接头缺陷和肌肉萎缩
Xinyi Tan,Shuangshuang Sun,Yuqing Yan et al.
Xinyi Tan et al.
Hexanucleotide repeat expansions in C9orf72 produce dipeptide repeat (DPR) proteins that are widely expressed, including the nervous system and skeletal muscle. Among these DPRs, arginine-containing proteins, poly-GR and poly-PR are toxic i...
Rescue of stereocilia architecture and hearing function by AAV-CIB2 and AAV-CIB3 in a mouse model of recessive deafness DFNB48 [0.03%]
在DFNB48小鼠模型中利用AAV-CIB2和AAV-CIB3恢复立体毛细胞结构和听力功能
Sakina Rehman,Arnaud P J Giese,Abigail K Dragich et al.
Sakina Rehman et al.
Pathogenic variants in the calcium and integrin binding protein 2 (CIB2) are among the leading causes of nonsyndromic prelingual hearing loss (DFNB48). Loss of Cib2 in mice causes severe stereocilia bundle defects, impaired mechano-electric...