首页/数据集/Interleukins 4 and 13 induce exon skipping of mutant dystrophin pre-mRNA to restore dystrophin production

Interleukins 4 and 13 induce exon skipping of mutant dystrophin pre-mRNA to restore dystrophin production

NIAID Data Ecosystem2026-03-10 收录

基因治疗

外显子跳跃

数据链接：

https://www.ncbi.nlm.nih.gov/bioproject/PRJNA414242 数据链接链接失效反馈

官方服务：

资源简介：

Interleukins 4 and 13 induce exon skipping of mutant dystrophin pre-mRNA to restore dystrophin production

应用场景：

创建时间：

2017-10-13

基因治疗

脂肪组织趋向性载体

AAV vectors are mainstream delivery platforms in gene therapy, yet AAV-mediated gene transfer to adipose tissue is underdeveloped due to low efficiency of natural AAVs. We previously demonstrated th

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Genetic screening of Russian Usher syndrome patients toward selection for gene therapy

基因筛查

基因治疗

Background : Usher syndrome (USH) is heterogeneous in nature and requires genetic test for diagnosis and management. Mutations in USH associated genes are reported in some populations except Rus

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Deep sequencing of prime editor target sequences

基因组编辑

基因治疗

Prime editing is a recent precision genome editing modality whose versatility offers the prospect for a wide range of applications, including the development of targeted genetic therapies. Yet, an out

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Dystrophin-deficient and dystrophin and utrophin double-deficient mice crossed with mice with full-length hDMD genes. Mus musculus

肌营养不良模型

基因治疗

Crossing of hDMD mice that contain the full-length 2.3 Mb hDMD gene were crossed with dystrophin-deficient mdx mice and dystrophin and utrophin double-deficient mdx x utrn-/- mice resulted in a full r

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Efficient creation and characterization of multi-kilobase knockins by CRISPR/Cas9 and multiple rAAVs

基因编辑

基因治疗

Long-read sequencing data for preprint: Efficient creation and characterization of multi-kilobase knockins by CRISPR/Cas9 and multiple rAAVs

DataCite Commons2025-05-01 更新100