Virus-mediated gene transfer of soluble amyloid precursor protein-alpha via systemic injection in a mouse model of Alzheimer's disease [0.03%]
溶型APP蛋白基因病毒载体介导的阿尔茨海默病小鼠模型全身给药治疗研究
Yuanyuan He,Bruce G Mockett,Lucia Schoderboeck et al.
Yuanyuan He et al.
Alzheimer's disease (AD) is the most common neurodegenerative disorder, yet effective preventive or therapeutic strategies remain limited. A hallmark of AD pathology is the accumulation of insoluble amyloid-β (Aβ) aggregates, which are ta...
Ultrasound-guided left heart injection: a safer and more efficient strategy for mouse thoracic aortic gene delivery [0.03%]
超声引导的左心注射：小鼠胸主动脉基因传递的安全性及效率更高的策略
Yanmiao Yang,Kunyao Xu,Ya Yuan et al.
Yanmiao Yang et al.
Thoracic aortic disease poses a significant threat due to its high mortality rates and genetic. underpinnings. While gene therapy holds promise for cures, the challenge lies in achieving. effective and safe gene delivery to the thoracic aor...
Cost-effectiveness of gene therapy for sickle cell disease in Uganda: tailoring high-income evidence to Uganda's context [0.03%]
乌干达镰状细胞病基因疗法的成本效益：根据乌干达国情调整高收入国家的证据
J Felipe Montano-Campos,Jennifer E Adair,Anirban Basu et al.
J Felipe Montano-Campos et al.
Blood stem cell gene therapy to treat hemoglobinopathies is beginning to transform health for small numbers of patients in the U.S. and Europe, where these conditions qualify as rare diseases. Yet hemoglobinopathies are common globally, dis...
Optimization and scale up strategies for reproducible AAV enrichment step on CIMmultus® QA HR line [0.03%]
CIMmultus® QA HR线的AAV富集步骤的可放大和优化策略以实现重复生产
Rok Žigon,Sara Drmota Prebil,Tomaž Švigelj et al.
Rok Žigon et al.
The advancement of adeno-associated virus (AAV) gene therapy applications faces significant challenges, particularly in the separation of empty (E), full (F), and intermediate capsids during manufacturing, as well as scaling up downstream p...
Drinkable gene therapy foam for the treatment of constrictive esophageal carcinoma [0.03%]
用于治疗食管狭窄型食管癌的可饮用基因疗法泡沫
Sirkka B Stephan,Carrie L Cummings,Katelyn Fitzgerald et al.
Sirkka B Stephan et al.
Patients diagnosed with esophageal cancer (EC) currently rely on treatments given at specialist care centers (surgery, chemotherapy, radiation), which despite their low cure rates are extremely life-disruptive, cause severe pain, and have s...
Tae Hee Kim,Chan You Kwon,Jae Yoon Song et al.
Tae Hee Kim et al.
Neovascular age-related macular degeneration (nAMD) is a major cause of irreversible vision loss in the elderly, driven by choroidal neovascularization and dysregulated vascular endothelial growth factor (VEGF) signaling. While anti-VEGF in...
Comprehensive forced degradation study revealing diverse chemical and physical degradation pathways of AAV8 [0.03%]
全面的强制降解研究揭示AAV8化学和物理降解的多种途径
Kuan-Yu Lai,Song Nie,Yu-Chieh Ariel Chen et al.
Kuan-Yu Lai et al.
While recombinant adeno-associated virus (AAV) holds significant promise for effective and durable gene delivery for gene therapy, a thorough understanding of the critical quality attributes (CQAs) along with the degradation pathways of AAV...
Baboon endogenous retrovirus (ERV) envelope pseudotyped lentiviral vectors outperform human ERV lentivectors for transduction of T, B, NK and HSPCs [0.03%]
猿类内源性逆转录病毒（ERV）假型慢病毒载体优于人类ERV慢病毒载体用于T、B、NK和HSPC的转导
Séverine Périan,Eva Castellano,Caroline Costa et al.
Séverine Périan et al.
Previously, we confirmed that BaEV-LVs outperformed VSV-G-LVs for gene delivery or correction of human T cells, B cells, NK cells and HSPCs correlating with high expression of its receptors, ASCT-1 and ASCT-2 on these cells. Since HERV-W gp...
CRISPR-AuNP: physicochemical optimization of a gold nanoparticle platform for cost-effective and modular non-viral gene editing in HSPCs [0.03%]
用于非病毒基因编辑的CRISPR-AuNP平台的成本效益和模块化物理化学优化研究：在HSPC中的应用
Karthikeya S V Gottimukkala,Daniel D Lane,Rachel Cunningham et al.
Karthikeya S V Gottimukkala et al.
Efficient delivery of CRISPR ribonucleoproteins into primary hematopoietic stem and progenitor cells (HSPCs) is essential for durable gene editing therapies but remains challenging. Here, we advance a modular, benchtop-assembled gold-polyme...
Evaluation of methods to increase the expression of cytokine-induced killer cell chemoattractant cytokines in pancreatic cancer [0.03%]
评估增加胰腺癌细胞趋化因子细胞细胞因子表达的方法的效果
Maria Bunuales,Susana Inoges,Ascension Lopez-Diaz de Cerio et al.
Maria Bunuales et al.
Adoptive Cell Therapies based on cytokine-induced killer cells (CIKs) can address the heterogeneity of solid tumors due to their multiple mechanisms of cancer cell recognition. However, tumor trafficking is one of the main limitations. In t...