Synchronized Latency Reversal and Immune Clearance by a Multifunctional Fusion Protein Enables HIV-1 Reservoir Reduction [0.03%]
多功能融合蛋白同步逆转 latency 并清除免疫系统使 HIV-1 储存库减少
Fei Luo,Yinghui Cao,Na Liu et al.
Fei Luo et al.
Strategies that concurrently reactivate latent reservoirs and enhance immune-mediated clearance hold significant promise for achieving an HIV cure. Here, we developed hyperIL-15×sCD4-Fc (15×sCD4-Fc), a fusion protein that integrates laten...
Tissue-Specific Silencing of Synthetic mRNAs By De-Targeting Elements Maps Vaccination-Competent Tissues, and Allows Cas9 De-Immunization [0.03%]
组织特异性沉默合成mRNA以筛选疫苗候选组织，并实现Cas9脱免疫化
Guendalina Froechlich,Annagiulia Scognamiglio,Simone Totaro et al.
Guendalina Froechlich et al.
The clinical success of mRNA/LNP-based vaccines has established a leading platform for in vivo nucleic acid delivery, yet the relationship between biodistribution, tissue-specific translation, and immune activation remains poorly defined. H...
Duowei Zhao,Xiangjie Zhao,Yuhang Gao et al.
Duowei Zhao et al.
Homologous recombination (HR) mediated large-size fragment knock-in (ls-KI) remains inefficient in mammalian cells, even with the assistance of CRISPR/Cas9. We hypothesized that adding DNA helicase activity to CRISPR/Cas9 could enhance ls-K...
Development of Recombinant Anti-TLR2 Antibodies and PLGA Nanoparticle-Based Gene Therapy for the Treatment of Neuropathic Pain [0.03%]
重组抗TLR2抗体及基于PLGA纳米粒子的基因治疗神经病理性疼痛的研究开发
Subeen Lee,Jaekyung Jeon,Hyunji Lee et al.
Subeen Lee et al.
Neuroinflammation is a key contributor to neuropathic pain, with microglial Toll-like receptor 2 (TLR2) playing a central role in initiating and sustaining proinflammatory responses. However, existing TLR2-targeting antibodies are limited b...
Plugging the diastolic calcium leak: MYBPC3 fragment for CPVT gene therapy [0.03%]
针对CPVT的基因治疗：插补舒张期钙泄漏-MYBPC3片段
Jianan Wang,Vincent M Christoffels,Christian van der Werf et al.
Jianan Wang et al.
Prothrombin recognition and conformational modulation by anti-thrombin anticoagulant aptamers [0.03%]
抗凝血酶寡核苷酸适配体对凝血酶的识别及构象调控作用
Romualdo Troisi,Alessandro Cangiano,Nathan Cowieson et al.
Romualdo Troisi et al.
Disorders of the blood coagulation cascade continue to pose a major clinical challenge, necessitating the development of new therapeutic agents capable of modulating this process. Several oligonucleotide aptamers targeting coagulation facto...
Evaluating cases of possible insertional mutagenesis after AAV gene therapy [0.03%]
评估AAV基因治疗后可能的插入突变病例
Lindsey A George,Rebecca C Ahrens-Nicklas,Frederic D Bushman
Lindsey A George
Identification of AAV Vector Integration Sites in Primary Human and Macaque Hepatocytes in vivo, and Analysis of Oncogenic Risk [0.03%]
AAV载体原位嵌合肝细胞基因疗法潜在致癌风险评估
Suzanne Scott,Claus V Hallwirth,Natsuki Sasaki et al.
Suzanne Scott et al.
Recombinant adeno-associated virus (rAAV) vectors are widely used for in vivo gene therapy, yet their potential to integrate into the host genome raises concerns about insertional mutagenesis and oncogenic risk, particularly in the liver wh...
The dermis as a gene therapy target: AAVrh32.33 finds an unexpected home in skin [0.03%]
以皮肤为基因治疗目标：AAVrh32.33在皮肤中意外发现其作用场所
Eline S Herman,Irina Filz von Reiterdank
Eline S Herman