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Repairing pathogenetic mutations using engineered Prime Editor in vivo

NIAID Data Ecosystem2026-05-02 收录

基因编辑治疗

遗传性心脏病干预

数据链接：

https://www.ncbi.nlm.nih.gov/sra/DRP012221 数据链接链接失效反馈

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资源简介：

We attempted to repaire genetic mutaions that cause hereditary heart disease using engineered Prime Editing in vivo.

应用场景：

创建时间：

2024-11-14

基因编辑治疗

地中海贫血临床研究

This is a non-randomized, open label, single-dose, phase 1/2 study in up to 12 participants with beta-thalassemia major.This study aims to evaluate the safety and efficacy of the treatment with gama-g

NIAID Data Ecosystem70

DataSheet1_Specific correction of pyruvate kinase deficiency-causing point mutations by CRISPR/Cas9 and single-stranded oligodeoxynucleotides.pdf

基因编辑治疗

遗传病点突变校正

Pyruvate kinase deficiency (PKD) is an autosomal recessive disorder caused by mutations in the PKLR gene. PKD-erythroid cells suffer from an energy imbalance caused by a reduction of erythroid pyruvat

NIAID Data Ecosystem60

In vivo Ryr2 editing corrects catecholaminergic polymorphic ventricular tachycardia

基因编辑治疗

遗传性心律失常

Catecholaminergic polymorphic ventricular tachycardia (CPVT) is a lethal inherited arrhythmia disorder most commonly caused by missense mutations in the RyR2 gene. The goal of this study was to determ

NIAID Data Ecosystem20

Prime editing in the rd12 mice

基因编辑治疗

遗传性视网膜疾病

Sequencing of genomic DNA in the RPE

NIAID Data Ecosystem70

Ex vivo therapeutic base and prime editing using chemically derived hepatic progenitors in a mouse model of tyrosinemia type 1. Kim et al.

基因编辑治疗

1型酪氨酸血症

Bipotent differentiation capacity of HT1-mCdHs. (A) Gene expression levels of mature hepatocyte-specific markers determined by RT-qPCR. Gapdh was used as an internal control. Data are mean ± SD (n=9).

NIAID Data Ecosystem40

Repairing pathogenetic mutations using engineered Prime Editor in vivo

资源简介：

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