Treatment of spinal injury muscle spasticity by spinal subpial AAV9-GAD65/VGAT delivery: an efficacy and safety study in rat, pig and NHP [0.03%]
蛛网膜下腔给予AAV9-GAD65/VGAT治疗脊髓损伤后肌痉挛：大鼠、猪和非人灵长类的疗效与安全性研究
Duong Nguyen,Tetsuya Yoshizumi,Masanori Sawamura et al.
Duong Nguyen et al.
The loss in segmental inhibitory GABA-ergic tone plays the key role in the development of spinal injury-induced muscle spasticity. We use a subpial segment-targeted delivery of adeno-associated virus vector(s) expressing GAD65 (Glutamic aci...
Discovery of TCR-like antibodies to the KRAS G12D neoantigen via in silico-in vitro workflow [0.03%]
通过计算与体外实验结合的工作流程发现针对KRAS G12Dneo抗原的类TCR抗体
SangPhil Ahn,Tae-Sung Oh,Seonghyuk Suh et al.
SangPhil Ahn et al.
Antibodies that recognize peptide-loaded class I major histocompatibility complex (MHC) molecules could enable therapeutic targeting of intracellular oncogenic proteins, yet their discovery has been hampered by the small size of peptide ant...
Targeted antisense oligonucleotide therapy rescues PRPF31 expression in retinitis pigmentosa caused by a splicing mutation [0.03%]
靶向反义寡核苷酸治疗可修复由剪接突变引起的视网膜色素变性病中的PRPF31表达
Poulami Banik,Felix Zimmann,Prasoon K Thakur et al.
Poulami Banik et al.
Pathogenic variants in splicing factors are the second most common cause of autosomal dominant retinitis pigmentosa (RP), with mutations in PRPF31 being the most prevalent. Here, we characterize a novel intronic variant in PRPF31 (c.1074-11...
Therapeutic bacteria-trained NK cells confer long-term protection against cancer metastasis [0.03%]
经过治疗细菌训练的NK细胞可长期预防癌症转移
Li Rong,Jingchu Hu,Wei Jin et al.
Li Rong et al.
Metastasis causes 90% of cancer-related deaths, yet preventive strategies remain limited. Here, we report that a single dose of therapeutic Salmonella, a prominent anti-tumor bacterial therapy, provides long-lasting protection against metas...
Directing fratricide within T cell products using an anti-uPAR chimeric antigen receptor to drive the production of potent therapeutic cells [0.03%]
使用抗uPAR嵌合抗原受体引导T细胞制品内的同种相残效应以产生有效的治疗性细胞
Lauren Sarko,David Givand,Brendan Rattin et al.
Lauren Sarko et al.
Cell therapy manufacturing of primary T cells often results in heterogeneous cell populations within a final product, with many cells lacking desired receptor expression or exhausted and other dysfunctional phenotypes. Here, we design a nov...
Depletion of CD8+ CAR T-cells leads to superior anti-tumor efficacy of pure CD4+ CAR T-cells against Acute Leukemias [0.03%]
耗竭CD8+CAR-T细胞导致纯CD4+CAR-T细胞对急性白血病的抗肿瘤疗效更佳
Qian Chen,David Sedloev,Hao Yao et al.
Qian Chen et al.
Chimeric antigen receptor T-cell (CART) therapy has demonstrated clinical efficacy in hematologic malignancies; however, primary or secondary treatment failure remains major obstacles to durable responses. Defining the optimal cellular comp...
From Undruggable to Degradable: Strategic Expansion of Targeted Protein Degradation in Cancer Therapy [0.03%]
从不可成药到可降解：蛋白降解策略在癌症治疗中的扩展与发展
Jiyoon Sung,Yuna Seo,Jae-Young Lee
Jiyoon Sung
Targeted protein degradation (TPD) has reshaped modern pharmacology by shifting therapeutic strategies from occupancy-driven inhibition toward event-driven elimination of disease-causing proteins, including targets long regarded as undrugga...
Spatiotemporal Atlas of Pro-Inflammatory NF-κB and Anti-Inflammatory STAT6 Signalling Using Reporter Mice during mRNA Vaccination [0.03%]
用于mRNA疫苗接种期间的促炎NF-κB和抗炎STAT6信号转导的空间时间图谱：利用报告基因小鼠的研究
Electra Brunialti,Alessia Panzeri,Nicoletta Rizzi et al.
Electra Brunialti et al.
Immunization engages a highly coordinated series of innate and adaptive immune responses across multiple anatomical sites, a complexity that has contributed to the predominantly empirical nature of vaccine development. To address this limit...
AAV-mediated FGF21 gene therapy promotes healthspan extension by whole-body tissue-specific adaptations [0.03%]
基于全身组织特异性适应的AAV介导FGF21基因治疗促进健康寿命延长
Veronica Jimenez,Victor Sacristan,Miquel Garcia et al.
Veronica Jimenez et al.
The decline of organ function during aging limits healthspan. Despite the potential of lifestyle interventions to improve health, sustained maintenance of healthspan is challenging, and no gerotherapeutic drugs have been approved. Here, we ...