Although great efforts have been made to address the bioavailability and tissue targeting of drugs, little is known about drug delivery to specific subcellular compartments that harbour the drug ...
FDA clearance of HG202 marks a significant milestone for CRISPR/Cas13 RNA-editing in clinical applications for nAMD. The BRIGHT trial will evaluate HG202's safety, efficacy, and potential to improve ...
该研究开发了一种基于CRISPR-Cas13的靶向RNA的转录组规模的CRISPR筛选技术,并使用该技术在来自不同组织的5种人类细胞中筛选鉴定了778个必需的lncRNA。
Abdelmageed et al. demonstrate POLK expression in neurons and report an important observation that POLK exhibits an age-dependent change in subcellular localization, from the nucleus in young tissue ...
However, a newer technology uses the enzyme Cas13 to more precisely target RNA without disrupting nearby protein-coding genes and other regulatory elements. Sanjana's lab previously demonstrated ...
However, a newer technology uses the enzyme Cas13 to more precisely target RNA without disrupting nearby protein-coding genes and other regulatory elements. Sanjana's lab previously demonstrated ...
HG202 是有史以来首个临床阶段的 CRISPR/Cas13 RNA 编辑疗法,也是唯一一个临床阶段的 RNA 靶向疗法,用于治疗 nAMD。 惠达基因联合创始人兼首席执行官Alvin Luk博士、工商管理硕士、CCRA表示:“美国 FDA 为 HG202 颁发的开放性 IND 是首个批准 CRISPR/Cas13 进行临床开发的 ...
This image shows the researchers’ subcellular-sized devices, which are designed to gently wrap around different parts of neurons, such as axons and dendrites ...
HG202 is the first-ever clinical-stage CRISPR/Cas13 RNA-editing and the only clinical-stage RNA-targeting therapy for nAMD. “This open IND for HG202 by the US FDA – the first regulator to have cleared ...