Functional Editing of the OTC locus by Targeted Integration with Phenotype Correction and Restoration of Physiological Patterns of Expression [0.03%]
OTC基因座的功能性编辑及表型矫正和表达模式的恢复
Samantha L Ginn,Fatemeh Doroudian,Sharntie Christina et al.
Samantha L Ginn et al.
Here we report highly efficient functional repair of the ornithine transcarbamylase (OTC) locus in mutant mouse and human hepatocytes in vivo using a dual AAV system delivering CRISPR-Cas9 editing reagents and a promoterless donor for targe...
Transgene Sequence Codon Optimization and Composition Determines Replication Competence of Self-Amplifying RNA [0.03%]
转基因序列密码子优化及组成决定自我复制RNA的复制能力
Nuthan Vikas Bathula,Shekinah K V Soriano,Cynthia Huang et al.
Nuthan Vikas Bathula et al.
Self-amplifying RNA (saRNA) is an emerging RNA therapeutic modality that can facilitate higher magnitude and more durable protein expression at substantially lower doses than non-replicating mRNA. Unlike conventional mRNA, alphavirus derive...
The oncolytic viral immunotherapy ASP1012 optimizes memory T-cell responses after systemic delivery [0.03%]
一种溶瘤病毒免疫疗法ASP1012可优化全身给药后的记忆T细胞反应
Ravikumar Muthuswamy,Carly Carter,Ming Zhang et al.
Ravikumar Muthuswamy et al.
Clinical use of Oncolytic viral immunotherapies (OVI) has been hindered by the requirement for intratumoral delivery and inconsistent activity. Here we describe a novel, engineered strain of vaccinia virus (ASP1012) designed to overcome bot...
Are the Current Preclinical Models Adequate to Evaluate CAR-T Cell Therapy in Paediatric High-Grade Gliomas? [0.03%]
现有的针对儿童高级别胶质瘤的CAR-T细胞疗法前期模型足够充分吗？
Ana Muñoz Grau,Fatima Hameedat,Bibi Sumayyah Hussein Sokeechand et al.
Ana Muñoz Grau et al.
Paediatric high-grade gliomas (pHGGs) are aggressive childhood brain tumours with five-year survival rates below 20%. They are distinct from adult high-grade gliomas (aHGGs), driven by histone mutations, disrupted developmental programs, an...
Distinct YY dinucleotide periodicity in Adeno-associated virus (AAV) DNA [0.03%]
腺相关病毒(AAV)DNA中独特的YY二核苷酸周期性
Conradin Baumgartl,Jonas Becker,Lena Naber et al.
Conradin Baumgartl et al.
Dinucleotide periodicity is a hallmark of genome organization, yet its role in single-stranded (ss)DNA viruses remains poorly understood. Here, we systematically analyzed dinucleotide spacing patterns in Adeno-associated virus (AAV) genomes...
Targeting of Aberrant αvβ6 Integrin Expression in Solid Tumors Using Chimeric Antigen Receptor-Engineered T Cells [0.03%]
利用嵌合抗原受体工程T细胞靶向实体肿瘤中的异常αvβ6整合素表达
Lynsey M Whilding,Ana C Parente-Pereira,Tomasz Zabinski et al.
Lynsey M Whilding et al.
Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease [0.03%]
新生婴儿全身基因治疗可改善Pompe病大鼠模型的心肺功能
David D Fuller,Sabhya Rana,Prajwal P Thakre et al.
David D Fuller et al.
Endothelial injury with capillary leak: A final common pathway in acute AAV toxicity? [0.03%]
内皮损伤与毛细血管渗漏：急性AAV毒性的一个最终共同途径？
Terence R Flotte
Terence R Flotte
Long-term functional synaptic integration of genome-edited retinal organoids in a primate model of macular degeneration [0.03%]
基因编辑视网膜类器官在灵长类黄斑变性模型中长期功能突触整合的研究
Atsuta Ozaki,Akihiro Kawai,Ryutaro Akiba et al.
Atsuta Ozaki et al.
Retinal organoids represent a promising regenerative strategy for restoring vision in retinal degenerative diseases, but the capacity of host cone bipolar cells in the primate macula to rewire with transplanted photoreceptors has not been e...
Nox1/4 inhibitor Setanaxib treatment ameliorates cardiac function in mouse models of Duchenne Muscular Dystrophy [0.03%]
诺氧酸抑制剂Setanaxib改善杜氏肌营养不良小鼠模型心脏功能
Rui Deng,Tabatha De Oliveira Silva,Xiang Liu et al.
Rui Deng et al.
Duchenne Muscular Dystrophy (DMD) is caused by mutations in dystrophin, leading to degeneration and weakness of skeletal and cardiac muscle. Despite great progress in the development of gene replacement therapies, DMD remains a devastating ...