编者按：2025年以来，siRNA疗法在治疗凝血相关疾病的研究方面取得了重要里程碑进展。RNA干扰（RNAi）机制通过在肝脏细胞中沉默编码凝血因子或天然抗凝物的mRNA，为治疗凝血相关疾病提供了一种全新的治疗模式。基于GalNAc偶联的递送技术，是将 ...

TAMs are crucial immune cells in the tumor microenvironment and key targets for tumor immunotherapy, with gene therapy being an important approach. However, existing gene carriers are unsatisfactory ...

Aptamer Group plc (AIM: APTA), the developer of novel Optimer® binders to enable innovation in the life sciences industry, is pleased to announce a new agreement with AstraZeneca, a global ...

In a study published in Cell Reports Physical Science on Nov. 25, a research team led by Prof. CAI Lintao from the Shenzhen Institutes of Advanced Technology (SIAT) of the Chinese Academy of Sciences ...

A study published in the Journal of Controlled Release ("NMR-based analysis of impact of siRNA mixing conditions on internal structure of siRNA-loaded LNP"), led by Assistant Professor Keisuke Ueda ...

• New data confirm highly selective profile of cell-targeted lipid nanoparticle (ctLNP) and first-ever siRNA delivery to T cells in non-human primates • Lead siRNA candidates show potent knockdown of ...

Avidity expands its leading RNA delivery technology into precision cardiology with two new wholly-owned development candidates targeting rare genetic cardiomyopathies: AOC 1086 to treat PLN ...

Spinal Cord Injury (SCI) is a devastating neurological disorder that often leads to permanent neural dysfunction. Current treatments fail to address the core challenges of insufficient intrinsic ...

Nanomedicine. 2014;9(1):105-120. Overall, HDL-based siRNA delivery possesses many attractive features, including ultra-small size, favorable surface properties (no PEGylation is required for achieving ...

Table 2. Stable nucleic acid lipid particle formulations. Stable nucleic acid lipid particle formulations in systemic delivery Target siRNA and modification (yes/no) Animal model Ref.