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Precision base and prime editing in the eye using ribonucleoprotein nanoparticles

NIAID Data Ecosystem2026-05-02 收录

基因编辑治疗

纳米颗粒递送

数据链接：

https://www.ncbi.nlm.nih.gov/bioproject/PRJNA1124167 数据链接链接失效反馈

官方服务：

资源简介：

Delivery of base and prime editor ribonucleoproteins in vivo by cell-penetrating peptide and lipid nanoparticle

应用场景：

创建时间：

2024-06-14

基因编辑治疗

扩张型心肌病

Mutations in RNA binding motif protein 20 (RBM20) are a common cause of dilated cardiomyopathy (DCM). Many RBM20 mutations cluster within an arginine/serine rich (RS-rich) domain, resulting in mis-loc

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Universal correction of blood coagulation factor VIII in patient-derived induced pluripotent stem cells using CRISPR/Cas9

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细胞疾病模型

Confirmation of the nuclease acivities at the On- and Off-target sites; Hemophilia A (HA) is caused by genetic mutations in the blood coagulation factor VIII (FVIII). Genome editing approaches can be

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Therapeutic adenine base editing of human hematopoietic stem cells

基因编辑治疗

β-地中海贫血

In this paper, we confirmed for the first time that ABE8e-sgRNA RNP complex can achieve efficient multiplex editing in both healthy and beta-thalassemia human HSPCs. After strict detection by deep seq

NIAID Data Ecosystem20

Datasheet for the BloodVoyagerS Dataset

纳米颗粒递送

血流模拟

This Dataset is created to enable researchers to explore the traveling paths of nanoparticles in the human circulatory system (HCS).

IEEE50

Single-Dose CRISPR/Cas9 Therapy Extends Lifespan of Mice with Hutchinson-Gilford Progeria Syndrome

基因编辑治疗

早衰症研究

Hutchinson-Gilford Progeria Syndrome (HGPS) is a rare lethal genetic disorder characterized with symptoms reminiscent of accelerated aging. The major underlying genetic cause is a substitution mutati

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Precision base and prime editing in the eye using ribonucleoprotein nanoparticles

资源简介：

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