In vivo gene targeting to treat hemophilia B mice

NIAID Data Ecosystem2026-03-11 收录

基因治疗

血友病B

数据链接：

https://www.ncbi.nlm.nih.gov/bioproject/PRJNA526239 数据链接链接失效反馈

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资源简介：

CRISPR/Cas9-mediated in vivo gene targeting to correct hemostasis in newborn and adult FIX-KO mice

创建时间：

2019-03-08

Additional file 4: of Viral-mediated gene delivery of TMBIM6 protects the neonatal brain via disruption of NPR-CYP complex coupled with upregulation of Nrf-2 post-HI

基因治疗

蛋白质表达分析

Statistical Analysis for Endogenous Protein Expression Levels post-HI. Representative table showing detailed statistical analysis for the endogenous expression levels of BI-1, NPR, P4502E1, pNrf-2 and

DataCite Commons2020-08-26 更新50

LEAPER

RNA靶向

基因治疗

Current tools for targeted RNA editing rely on the delivery of exogenous proteins or chemically modified guide RNAs, which may lead to aberrant effector activity, delivery or immunogenicity. Here we p

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Base editing mediated correction of severe β0 IVS2-1 thalassemia mutations.

基因治疗

碱基编辑技术

β-thalassemia is a highly prevalent monogenic recessive disease caused by mutations affecting the synthesis of the adult hemoglobin β-chains. Transplantation of autologous, genetically modified hemato

NIAID Data Ecosystem10

Co-transfection of hepatocyte growth factor and truncated TGF-β type II receptor inhibit scar formation

基因治疗

伤口愈合

Wound scarring remains a major challenge for plastic surgeons. Transforming growth factor (TGF)-β plays a key role in the process of scar formation. Previous studies have demonstrated that truncated T

DataCite Commons2024-02-08 更新40

Programmable RNA base editing with a single engineered protein

碱基编辑

基因治疗

Programmable base editing of RNA enables rewriting the genetic codes on specific sites. Current tools for specific RNA editing dependent on the assembly or recruitment of the guide RNA into an RNA/pro

NIAID Data Ecosystem10

In vivo gene targeting to treat hemophilia B mice

资源简介：

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