Extracellular Vesicles-Associated AAVs for the treatment of Machado-Joseph Disease [0.03%]
外泌体相关AAV用于治疗马查多约瑟夫病
Carina Henriques,Patrícia Albuquerque,David Rufino-Ramos et al.
Carina Henriques et al.
Machado-Joseph disease (MJD) is the most common dominant autosomal inherited ataxia worldwide, caused by the over-repetition of the trinucleotide CAG in the ATXN3 gene. This leads to the accumulation of ataxin-3 protein and neurodegeneratio...
Pancreatic Transdifferentiation of NOD Mouse Livers Prevented Development of Hyperglycemia [0.03%]
糖尿病小鼠模型中肝脏的胰腺转分化可预防高血糖出现
Alexandra L G Mahoney,Binhai Ren,Najah T Nassif et al.
Alexandra L G Mahoney et al.
Type 1 diabetes (T1D) is caused by the autoimmune destruction of the pancreatic insulin producing beta (β)-cells. This study investigated a novel gene therapy approach to prevent disease development by replacing pancreatic β-cell function...
A rapid immunization and antibody redesign platform for discovering broadly neutralizing antibodies against non-immunized SARS-CoV-2 variant [0.03%]
一种快速免疫和抗体重设计平台 用于发现针对未免疫的SARS-CoV-2变异体的广谱中和性抗体
Shusei Hamamichi,Narumi Uno,Kazuto Shimoya et al.
Shusei Hamamichi et al.
From the COVID-19, we learned valuable lessons related to development of broadly neutralizing antibodies (bnAbs). Here, we present a discovery platform termed Express Hu-mAb System that integrated fully human Ab-producing trans-chromosomic ...
Modulation of the unfolded protein response with a C-terminal fragment of MANF facilitates recovery in models of multiple sclerosis [0.03%]
C末端片段化MANF通过调节未折叠蛋白反应促进多发性硬化模型的恢复
Tapani K Koppinen,Carolina R Reyes,Jinhan Nam et al.
Tapani K Koppinen et al.
Inflammation in multiple sclerosis leads to chronic activation of a cellular stress mechanism, the unfolded protein response (UPR), which is thought to both exacerbate neuroinflammation and prevent regenerative tissue responses such as remy...
CRISPR for cystic fibrosis: Advances and insights from a systematic review [0.03%]
针对囊性纤维化的 crispr 研究进展及系统评价分析结果
Lucia Nicosia,Patrick T Harrison
Lucia Nicosia
Podocyte-directed VEGFC gene therapy prevents increased glomerular permeability and glycocalyx damage in a mouse model of type 1 diabetes [0.03%]
针对1型糖尿病小鼠模型足细胞的VEGFC基因治疗可预防肾小球通透性增加和糖萼损伤
Aldara Martin Alonso,Carl May,Holly Stowell-Connolly et al.
Aldara Martin Alonso et al.
Diabetic kidney disease (DKD) is the leading cause of end-stage renal failure, and current interventions fail to directly target the glomerulus, where the disease initiates. Vascular endothelial growth factor (VEGF)C is a key contributor to...
Gene supplementation with precise transgene expression rescues hearing loss in a mouse model with an Mpzl2 East Asian founder variant [0.03%]
具有MPZL2东亚创始人突变体的小鼠模型听力恢复研究及精准转基因表达基因补充疗法的研究
Seung Hyun Jang,Hyeong Gi Song,Sun Young Joo et al.
Seung Hyun Jang et al.
Hearing loss is the most common sensory organ disorder, with genetic factors substantially contributing to the disease. Among the 87 genes responsible for autosomal recessive nonsyndromic hearing loss, mutations in MPZL2 have been frequentl...
CD8 co-receptor modulates the specificity profile of the 1G4 TCR against NY-ESO-1 [0.03%]
共刺激受体调节1G4 T细胞受体对NY-ESO-1抗原的识别谱式
Heather F Jones,Zita Aretz,Ron S Gejman et al.
Heather F Jones et al.
Engineered T cells have shown efficacy in cancer treatment. However, the promiscuity of TCR engineered T cells may result in recognition of off-target epitopes, causing severe toxicities. A genetic screen of >3,000 proteomic epitopes in the...
Cancer Viroimmunotherapy Platforms Based on Varicella-Zoster Virus and Cytomegalovirus [0.03%]
基于水痘带状疱疹病毒和巨细胞病毒的癌症病毒免疫治疗平台技术路线研究进展
Haifei Jiang,Kah Whye Peng,Stephen J Russell
Haifei Jiang
Herpes simplex virus (HSV)-based oncolytic virotherapy has demonstrated promising antitumor effects across various cancer types. However, its application remains limited in scope, and expanding its use to additional cancers poses ongoing ch...
Comparative analysis of RNA versus protein splicing in dual AAV-mediated gene therapy in a mouse model of DFNB9 deafness [0.03%]
基于小鼠DFNB9型耳聋模型的RNA与蛋白质剪接的双AAV载体介导基因治疗效果比较分析
Mengzhao Xun,Xintai Fan,Hui Wang et al.
Mengzhao Xun et al.
Pathological mutations in the OTOF gene cause autosomal recessive deafness 9 (DFNB9). Although dual-AAV gene replacement therapy has been shown to partially rescue the hearing of patients with DFNB9, the therapeutic effects still need furth...