首页/数据集/Antisense oligonucleotides modulate aberrant inclusion of poison exons in SCN1A-related Dravet syndrome

Antisense oligonucleotides modulate aberrant inclusion of poison exons in SCN1A-related Dravet syndrome

NIAID Data Ecosystem2026-05-02 收录

基因治疗

神经遗传疾病

数据链接：

https://www.ncbi.nlm.nih.gov/bioproject/PRJNA1221960 数据链接链接失效反馈

官方服务：

资源简介：

Targeted long read sequencing of SCN1A

应用场景：

创建时间：

2025-02-10

PET成像技术

基因治疗

Positron emission tomography (PET) reporter systems are a valuable means of estimating the level of expression of a transgene in vivo . For example, the safety and efficacy of gene therapy appro

DataCite Commons2024-04-12 更新300

raw fig 3c

生物医学

基因治疗

Raw data - in vivo effect of TUSC2 + ER

DataONE2015-06-09 更新100

Source data for "Virus-like recombinant biosome: a targeted and effective gene delivery platform".zip

基因治疗

生物材料

Here, we report a virus-like recombinant biosome (reBiosome) as a safe and effective delivery vehicle for targeted gene therapy. A mutant biosome (mBiosome) was first prepared by genetically recombini

DataCite Commons2023-10-16 更新70

Enhancing site-specific DNA integration by a Cas9 nuclease fused with a DNA donor-binding domain

定点DNA整合

基因治疗

The CRISPR/Cas system is a widely used tool for genome editing. However, robust and targeted insertion of a DNA segment remains challenging. Here, we present a fusion nuclease (Cas9-N57) to enhance si

NIAID Data Ecosystem110

Additional file 8 of Retinal organoids with X-linked retinoschisis RS1 (E72K) mutation exhibit a photoreceptor developmental delay and are rescued by gene augmentation therapy

基因治疗

视网膜疾病

Supplementary Material 8

DataCite Commons2024-08-15 更新80