Sijia Xu,Zhenzhen Hu,Fenglin Song et al.
Sijia Xu et al.
Lipid nanoparticles (LNPs) are lead non-viral vectors for delivering nucleic acids. LNPs can efficiently encapsulate nucleic acids, protect them from degradation, enhance cellular uptake and induce endosome escape, which show high transfect...
Preclinical studies of an AAV8-CYP4V2 gene therapy VGR-R01 for the treatment of Bietti crystalline dystrophy [0.03%]
用于治疗贝特氏结晶体病的AAV8-CYP4V2基因疗法VGR-R01的药物临床前研究
Wenjing Luo,Lu Guo,Liang Lu et al.
Wenjing Luo et al.
Bietti crystalline dystrophy (BCD) is an autosomal recessive disorder caused by loss-of-function mutations in the CYP4V2 gene, characterized by crystal-like lipid deposits in the retina, progressive photoreceptor loss, and retinal pigment e...
AAV2 delivery of the saCas9 gene results in presentation of an HLA-A∗02:01-restricted T cell epitope potent to induce T cell cytotoxicity [0.03%]
腺相关病毒(AAV)介导的saCas9基因递送可导致产生呈现在HLA-A*02：01限制性T细胞表位并能够诱导细胞毒性的T细胞
Susana S Najera,Annalisa Nicastri,Sojin Bing et al.
Susana S Najera et al.
In vivo genome editing with CRISPR-Cas9 systems is generating worldwide attention and enthusiasm for the possible treatment of genetic disorders. However, the consequences of potential immunogenicity of the bacterial Cas9 protein and the AA...
Development and implementation of an LC-MS-based multi-attribute method for adeno-associated virus [0.03%]
一种用于腺相关病毒的基于LC-MS的多属性方法的研发与实施
Thomas W Powers,Shawn Mariani,Halyna Narepekha et al.
Thomas W Powers et al.
The multi-attribute method (MAM), a mass spectrometry technique for quantifying amino acid modifications at the peptide level, is becoming a prominent analytical tool in the development of biotherapeutics. The method has promise for adeno-a...
Hematopoietic stem cell gene therapy for the treatment of X-linked agammaglobulinemia [0.03%]
X连锁无丙种球蛋白血症的造血干细胞基因治疗
Christopher R Luthers,Annika Mittelhauser,Aurelien Colamartino et al.
Christopher R Luthers et al.
X-linked agammaglobulinemia (XLA) is a rare inborn error of immunity caused by loss-of-function mutations in the gene encoding Bruton's tyrosine kinase (BTK). XLA patients lack mature B cells and have negligible antibody levels, leaving the...
Beneficial effect of resveratrol on T cell oxidative metabolism and anti-tumor function is conditioned by prior in vivo T cell history [0.03%]
白藜芦醇对T细胞的抗氧化代谢和抗肿瘤功能的有益作用取决于之前的体内T细胞发育过程中的遭遇事件
Patricia Mercier-Letondal,Chrystel Marton,Bernard Royer et al.
Patricia Mercier-Letondal et al.
Despite the clinical success of redirected T cells in the setting of cancer adoptive cell immunotherapy, patients may exhibit resistance to treatment, resulting in uncontrolled disease and relapses. This phenomenon partly relies on impaired...
Reversing PAI-1 deficiency in blood using mRNA lipid nanoparticles [0.03%]
使用mRNA脂质纳米粒逆转血液中PAI-1缺乏的影响
Francesca Ferraresso,Chad W Skaer,Katherine Badior et al.
Francesca Ferraresso et al.
Plasminogen activator inhibitor-1 (PAI-1) deficiency is a rare disorder that causes moderate to severe bleeding and cardiac fibrosis, caused by mutation in the SERPINE-1 gene and no detectable circulating PAI-1 protein. There are currently ...
Wild-type and engineered adeno-associated viral vectors produce comparable opsin expression and light-evoked responses in rat skeletal muscle [0.03%]
野生型和工程改造的腺相关病毒载体在大鼠骨骼肌中产生相当的视蛋白表达和光诱发反应
Fiona L Knapman,E Myfanwy Cohen,Tom Kulaga et al.
Fiona L Knapman et al.
Optogenetics offers a minimally invasive, low-fatigue, and temporally precise alternative to electrical stimulation for skeletal muscle control. After opsin expression in muscle cells, contraction can be stimulated with light. Obstructive s...
Development of LC-MS methods for AAV capsid protein quantification and host cell protein profiling [0.03%]
用于AAV衣壳蛋白定量和宿主细胞蛋白谱分析的LC-MS方法开发
Theodoros Kontogiannis,Christopher McElroy,Milena Quaglia et al.
Theodoros Kontogiannis et al.
Accurate quantification and characterization of recombinant adeno-associated virus (rAAV) capsid proteins are critical for evaluating product quality and safety, ensuring batch consistency, and informing process development of their manufac...
Innate immune response to AAV-based gene therapy vectors: Mechanisms of complement activation and cytokine release [0.03%]
基于腺相关病毒的基因治疗载体的先天免疫反应：补体激活和细胞因子释放的机制
Rebecca Xicluna,Petra C Schwalie,Emma Bell et al.
Rebecca Xicluna et al.
Recombinant adeno-associated viruses (rAAV) have emerged as a preferred strategy for in vivo gene delivery. However, the immune response to rAAV presents a major limitation, leading to serious adverse events in clinical trials. This study i...