Research progress of base editing and prime editing tools based on the CRISPR/Cas system [0.03%]
基于CRISPR/Cas系统的碱基编辑和先导编辑工具研究进展
Herui Gao,Song Gao,Guoshi Kan et al.
Herui Gao et al.
The base editor (BE) and prime editing guide RNA (pegRNA)-based prime editor (PE) technologies relying on the CRISPR/Cas system are very efficient gene editors that have been developed in recent years. The BEs include cytosine base editors ...
Adenine base editor correction of pathogenic variations associated with inherited retinal dystrophy in patient iPSC and retinal organoids [0.03%]
腺嘌呤碱基编辑器校正视网膜色素变性相关致病突变
Amy Leung,Pedro R L Perdigão,Almudena Sacristan-Reviriego et al.
Amy Leung et al.
Inherited retinal dystrophies (IRDs) are a group of incurable, genetically heterogeneous diseases that cause progressive degeneration of the retina, leading to the loss of vision. Genome editing technologies offer a powerful prospect for mu...
Novel allogeneic CAR T-cell platform involving microhomology-mediated end joining repair and low off-targeting potential [0.03%]
一种新型同种异体CAR-T平台技术，兼具有微同源重组介导的末端连接修复和低脱靶效应潜力
Tanya Hundal,Yan Luo,Yaqing Qie et al.
Tanya Hundal et al.
Several allogeneic chimeric antigen receptor (CAR) T-cell therapies in clinical trials rely on CRISPR-Cas genome editing, but the enzyme's random repair mechanism increases the risk of undesired off-target effects, challenging safe CAR T-ce...
Base editing strategies for in vivo correction of two highly recurrent phenylketonuria variants [0.03%]
体内矫正高复发性苯酮尿症两种变异的碱基编辑策略
Aidan Quigley,Ishaan Jindal,Thomas Campion et al.
Aidan Quigley et al.
Phenylketonuria (PKU) is an autosomal recessive disorder caused by mutations in the phenylalanine hydroxylase (PAH) gene. Previously, we have shown correction of the most recurrent PKU variant using both base editing and prime editing. In t...
Johanna Kohn,Anna-Maria Lauerer,Philipp Hegner et al.
Johanna Kohn et al.
Myeloid cell-targeted lipid nanoparticles for B cell lymphoma immunotherapy [0.03%]
靶向髓系细胞的脂质纳米颗粒介导B细胞淋巴瘤免疫治疗
Elaine Y Kang,Dongfang Wang,Damian Kaniowski et al.
Elaine Y Kang et al.
We previously demonstrated that blocking tolerogenic signal transducer and activator of transcription 3 (STAT3) signaling in the tumor microenvironment can unleash Toll-like receptor 9 (TLR9)-mediated antitumor immunity. To enable systemic ...
Rational design and applications of piperazine and cyclohexane ionizable lipids for PKU and SSADH deficiency [0.03%]
用于PKU和SSADH缺乏症的脯氨酯和环己基两性离子脂质的理性设计及应用
Jiyeon Son,Hyunjung Yoo,Joonyoung Park et al.
Jiyeon Son et al.
A significant challenge of mRNA-based protein replacement therapies is the diminishing efficacy and escalating toxicity associated with repeated dosing of lipid nanoparticles (LNPs). Many existing lipid formulations were originally designed...
Liver-directed base editing of ABCC6 prevents ectopic calcification in a variant-humanized mouse model of pseudoxanthoma elasticum [0.03%]
肝细胞定向ABCC6碱基编辑可预防假黄色瘤小鼠模型中弹性组织的异位钙化
Lauren C Testa,Dora Obiri-Yeboah,Hooda Said et al.
Lauren C Testa et al.
Pseudoxanthoma elasticum (PXE) is an autosomal recessive connective tissue disorder characterized by ectopic calcification of elastic fibers throughout the skin, retina, and arteries. It is caused by pathogenic variants in ABCC6, which enco...
Bailun Li,Zhike Lu,Ting Zhou et al.
Bailun Li et al.
Prime editor (PE) offers significant translational potential due to its diverse editing outcomes. However, its editing efficiency is generally lower than that of wild-type Cas9 and base editor (BE). Previous investigations have suggested th...
A novel bispecific siRNA concept: Efficient dual knockdown of YAP1 and WWTR1 with a single guide strand [0.03%]
一种新颖的双特异性siRNA概念：单引导链高效同时敲低YAP1和WWTR1基因
Kaito Ueda,Yuki Yamada,Hiroyuki Tanaka et al.
Kaito Ueda et al.
Yes-associated protein (YAP, encoded by YAP1) and transcriptional coactivator with PDZ-binding motif (TAZ, encoded by WWTR1) are master transcriptional regulators in the Hippo pathway. Because of their complementary roles, simultaneously in...