Genetic stability in FA cells before and after gene editing and cell reprogramming.

ChEBI2014-05-01 更新2026-07-21 收录

基因治疗安全性

细胞遗传稳定性分析

数据链接：

https://www.ebi.ac.uk/biostudies/ArrayExpress/studies/E-GEOD-56790 数据链接链接失效反馈

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Genetic stability in FA cells before and after gene editing and cell reprogramming.

创建时间：

2014-05-01

Senescence and inflammation are unintended consequences of CRISPR-Cas9 gene-editing in hematopoietic stem cells [WES]. undefined

基因编辑造血干细胞

基因治疗安全性

Gene editing (GE) in hematopoietic stem and progenitor cells (HSPCs) represents a promising strategy for site-specific gene correction of several inherited diseases. However, its clinical translation

NIAID Data Ecosystem30

Analyzing the Number of Common Integration Sites of Viral Vectors – New Methods and Computer Programs

载体整合位点

基因治疗安全性

Vectors based on γ-retroviruses or lentiviruses have been shown to stably express therapeutical transgenes and effectively cure different hematological diseases. Molecular follow up of the insertional

Figshare2016-01-18 更新20

Genome-wide off target detection of Un1Cas12f1 in mouse models

基因编辑脱靶检测

基因治疗安全性

Hypercompact Cas12f1 were demonstrated with eukaryotic nuclease activity showing great potential as AAV-based gene therapy system. Our present study provided definitive results for developing Cas12f1-

NIAID Data Ecosystem10

Transient in vivo base editing with no detectable DNA off-target mutations. in vivo base editing without DNA off-target mutations

碱基编辑技术

基因治疗安全性

RNA-programmable deaminases, known as base editors (BEs), enable precise single base conversions on genomic DNA and hold great promise for therapeutic application in patients. Recent studies, however,

NIAID Data Ecosystem20

rAAV9 integration in C57BL/6:C129 mus musculus

基因治疗安全性

AAV载体

Neonatal mice were treated with an rAAV9 gene therapy vector, some of whom later developed tumors. We sampled the tumors of these mice, as well as the livers of two additional mice that did not have v

NIAID Data Ecosystem20

Genetic stability in FA cells before and after gene editing and cell reprogramming.

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