Rewriting the script: gene therapy and genome editing for von Willebrand Disease [0.03%]
基因治疗和基因组编辑在韦尔弗兰德症中的应用
Alastair Barraclough,Isabel Bär,Tirsa van Duijl et al.
Alastair Barraclough et al.
In recent years gene therapy has emerged as a powerful technology for treatment of a large variety of inherited disorders. With the FDA approval of in vivo gene therapy of hemophilia A and B using AAV-mediated transgene delivery to hepatocy...
Editorial: Insights in genome editing in human health and disease 2023/2024 [0.03%]
特刊：基因组编辑在人类健康和疾病中的见解（2023-2024）
Baisong Lu,Haiwei Mou,Chen Liang
Baisong Lu
Single-stranded DNA (ssDNA) donor repair templates and CRISPR/Cas9 enable a high-frequency of targeted insertions in potato [0.03%]
单链DNA(ssDNA)供体修复模板和CRISPR/Cas9可在马铃薯中实现高效特异性插入突变
Matías N González,Neha Salaria,Martin Friberg et al.
Matías N González et al.
Homology-directed repair (HDR) holds great promise for plant genetic engineering but remains challenging due to its inherently low efficiency in gene editing applications. While studies in animal systems suggest that the structure of the do...
CRISPR activation: identifying and using novel genes for plant disease resistance breeding [0.03%]
CRISPR介导的植物抗病基因功能激活及其育种应用
John E McLaughlin,Idrice Carther Kue Foka,Michael A Lawton et al.
John E McLaughlin et al.
CRISPR-based technologies have revolutionized plant science by enabling precise modulation of gene function, including CRISPR activation (CRISPRa), a recently emerging strategy which shows particular promise for enhancing disease resistance...
The decision to purchase genome edited food products by Iranian consumers: theory of planned behavior as a social intervention tool [0.03%]
基于计划行为理论的社交干预工具在伊朗消费者基因编辑食品购买决策中的作用研究
Naser Valizadeh,Shobeir Karami
Naser Valizadeh
The main aim of present study was to analyze the consumers' preferences about genome/gene-edited food products in Iran. For this purpose, an extended version of the theory of planned behavior was used as a social intervention tool. The theo...
In pursuit of an HIV cure: from stem cell transplants to gene therapies [0.03%]
追寻HIV治愈之道：从干细胞移植到基因疗法
Jennifer Clees,Maya Basic,Pedro E Cruz et al.
Jennifer Clees et al.
Since 2009, seven people living with human immunodeficiency virus (PLHIV) have been declared cured of HIV after receiving allogeneic hematopoietic stem cell transplants (alloHSCTs) to treat hematologic malignancies. In this sense, cure sign...
Application of a multiplex CRISPR/Cas9 strategy for elimination of selection markers from transgenic plants [0.03%]
多重CRISPR/Cas9策略在转基因植物中去除筛选标记的应用
Mohammed Rafi,Mohamed ElSiddig,Maitha Aldarmaki et al.
Mohammed Rafi et al.
Selectable marker genes (SMGs) are essential for identifying transgenic plants but raise concerns regarding biosafety, regulatory compliance, and public acceptance. In this study, we used a CRISPR/Cas9-based strategy to eliminate the SMG fr...
CRISPR-FMC: a dual-branch hybrid network for predicting CRISPR-Cas9 on-target activity [0.03%]
CRISPR-FMC：一个用于预测CRISPR-Cas9靶向活性的双分支混合网络
Chuxuan Li,Jian Li,Quan Zou et al.
Chuxuan Li et al.
Introduction: Accurately predicting the on-target activity of sgRNAs remains a challenge in CRISPR-Cas9 applications, due to the limited generalization of existing models across datasets, small-sample settings, and comple...
Prime editing using paired pegRNAs targeting NG- or NGG-PAM in rice [0.03%]
利用成对的pegRNA在水稻中针对NG-或NGG-PAM进行基因编辑prime editing
Ayako Nishizawa-Yokoi,Keiko Iida,Akiko Mori et al.
Ayako Nishizawa-Yokoi et al.
Prime editing (PE) enables precise genome modification, i.e., all 12 types of base substitution, as well as designed insertion and deletion. Previously, we developed an efficient PE system using a pair of engineered pegRNAs (epegRNAs), appe...
Development of a homeolog-specific gene editing system in an evolutionary model for the study of polyploidy in nature [0.03%]
开发一种在家olog特异性基因编辑系统中的进化模型，用于研究自然界的多倍体现象
Shengchen Shan,Michael T Pisias,Evgeny V Mavrodiev et al.
Shengchen Shan et al.
Polyploidy, or whole-genome duplication (WGD), is a significant evolutionary force. Following allopolyploidy, duplicate gene copies (homeologs) have divergent evolutionary trajectories: some genes are preferentially retained in duplicate, w...