HCS-Splice: A High-Content Screening Method to Advance the Discovery of RNA Splicing-Modulating Therapeutics

Nucleic acid therapeutics have demonstrated an impressive acceleration in recent years. They work through multiple mechanisms of action, including the downregulation of gene expression and the modulation of RNA splicing. While several drugs based on the former mechanism have been approved, few targe...

Full description

Bibliographic Details
Published in:	Cells
Main Authors:	Giuseppina Covello, Kavitha Siva, Valentina Adami, Michela Alessandra Denti
Format:	Article
Language:	English
Published:	MDPI AG 2023-07-01
Subjects:	alternative splicing two-colour (GFP/RFP) fluorescent reporter <i>MAPT</i> exon-skipping FTDP-17 high content screening
Online Access:	https://www.mdpi.com/2073-4409/12/15/1959

Similar Items

Pairwise Engineering of Tandemly Aligned Self-Splicing Group I Introns for Analysis and Control of Their Alternative Splicing
by: Tomoki Ueda, et al.
Published: (2023-04-01)

An autopsied FTDP-17 case with MAPT IVS 10 + 14C > T mutation presenting with frontotemporal dementia
by: Ryohei Watanabe, et al.
Published: (2021-09-01)

A survey of computational methods in transcriptome-wide alternative splicing analysis
by: Wang Jianbo, et al.
Published: (2015-03-01)

Intrinsic Alu affects for RNA splicing in a minigene model
by: Mina Nakama, et al.
Published: (2025-06-01)

Widespread Alternative Splicing Changes in Metastatic Breast Cancer Cells
by: Jagyeong Oh, et al.
Published: (2021-04-01)

Alternative splicing of IRF3 plays an important role in the development of hepatocarcinoma
by: Zhenyi Lv, et al.
Published: (2023-12-01)

Evidence for a Common Founder and Clinical Characteristics of Japanese Families with the MAPT R406W Mutation
by: Takeshi Ikeuchi, et al.
Published: (2011-09-01)

Ninein, a candidate gene for ethanol anxiolysis, shows complex exon-specific expression and alternative splicing differences between C57BL/6J and DBA/2J mice
by: E. R. Gnatowski, et al.
Published: (2024-09-01)

Genetic variations and alternative splicing. The Glioma associated oncogene 1, GLI1.
by: Peter eZaphiropoulos
Published: (2012-07-01)

Alternative Splicing of RAD6B and Not RAD6A is Selectively Increased in Melanoma: Identification and Functional Characterization
by: Ambikai Gajan, et al.
Published: (2019-11-01)

Identified five variants in CFTR gene that alter RNA splicing by minigene assay
by: Bingying Zhang, et al.
Published: (2025-03-01)

Double-target Antisense U1snRNAs Correct Mis-splicing Due to c.639+861C>T and c.639+919G>A GLA Deep Intronic Mutations
by: Lorenzo Ferri, et al.
Published: (2016-01-01)

In silico splicing analysis of the PMS2 gene: exploring alternative molecular mechanisms in PMS2-associated Lynch syndrome
by: Cătălin Vasile Munteanu, et al.
Published: (2024-11-01)

CRISPR-SKIP: programmable gene splicing with single base editors
by: Michael Gapinske, et al.
Published: (2018-08-01)

Unlocking the therapeutic potential of RNA splicing in lung fibrosis: Insights from the SRSF7–PKM axis
by: Bi-Sen Ding
Published: (2025-06-01)

Reverse complementary matches simultaneously promote both back-splicing and exon-skipping
by: Dong Cao
Published: (2021-08-01)

Splice site proximity influences alternative exon definition
by: Francisco Carranza, et al.
Published: (2022-12-01)

Incorporating exon–exon junction reads enhances differential splicing detection
by: Mai T. Pham, et al.
Published: (2025-07-01)

Identified eleven exon variants in PKD1 and PKD2 genes that altered RNA splicing by minigene assay
by: Xuyan Liu, et al.
Published: (2023-07-01)

Exon definitive regions for MPC1 microexon splicing and its usage for splicing modulation
by: Eunjin Koh, et al.
Published: (2023-03-01)

Rational Design of Chimeric Antisense Oligonucleotides on a Mixed PO–PS Backbone for Splice-Switching Applications
by: Bao T. Le, et al.
Published: (2024-07-01)

SimSpliceEvol2: alternative splicing-aware simulation of biological sequence evolution and transcript phylogenies
by: Wend Yam D. D. Ouedraogo, et al.
Published: (2024-07-01)

Twelve exonic variants in the SLC12A1 and CLCNKB genes alter RNA splicing in a minigene assay
by: Qing Xin, et al.
Published: (2022-08-01)

Development and Characterization of Fluorescent Protein-Tagged <i>Vibrio harveyi</i> Strains as a Versatile Tool for Studying Infection Dynamics and Strain Interactions
by: Charalampos Chalmoukis, et al.
Published: (2025-03-01)

Alternative splicing across the tree of life
by: Rebeca de la Fuente, et al.
Published: (2025-10-01)

hnRNP A1 Regulates Alternative Splicing of Tau Exon 10 by Targeting 3′ Splice Sites
by: Yongchao Liu, et al.
Published: (2020-04-01)

Childhood-Onset Refractory Hypertension Results from Neurofibromatosis Type 1 Caused by a Splicing NF1 Mutation
by: Yi-Ting Lu, et al.
Published: (2023-08-01)

A novel splice site variant c.1183 + 1 G > C in DFNA5 causing autosomal dominant nonsyndromic hearing loss in a Chinese family
by: Qiong Li, et al.
Published: (2022-07-01)

Evolutionary perspective of Big tau structure: 4a exon variants of MAPT
by: Itzhak Fischer
Published: (2022-12-01)

Sonic hedgehog signaling: Alternative splicing and pathogenic role in medulloblastoma
by: Meiyu Qu, et al.
Published: (2023-09-01)

The complexity of titin splicing pattern in human adult skeletal muscles
by: Marco Savarese, et al.
Published: (2018-03-01)

Epigenetic Alterations in Cryopreserved Human Spermatozoa: Suspected Potential Functional Defects
by: Wanxue Wang, et al.
Published: (2022-07-01)

A Sensitive Assay System To Test Antisense Oligonucleotides for Splice Suppression Therapy in the Mouse Liver
by: Lorena Gallego-Villar, et al.
Published: (2014-01-01)

Evolutionarily Developed Alternatively Spliced Exons Containing Translation Initiation Sites
by: Jun-ichi Takeda, et al.
Published: (2024-12-01)

Splicing mutations in AMELX and ENAM cause amelogenesis imperfecta
by: Zhenwei Zhang, et al.
Published: (2023-11-01)

A Novel splice-site mutation on the MLC1 gene leading to exon 9 skipping and megalencephalic leukoencephalopathy with subcortical cysts in a Turkish patient
by: Türkyılmaz A, et al.
Published: (2019-12-01)

Ctcf haploinsufficiency mediates intron retention in a tissue-specific manner
by: Adel B Alharbi, et al.
Published: (2021-01-01)

Comprehensive analysis of TCGA data reveals correlation between DNA methylation and alternative splicing
by: Shuting Lin, et al.
Published: (2022-11-01)

An anciently diverged family of RNA binding proteins maintain correct splicing of a class of ultra-long exons through cryptic splice site repression
by: Chileleko Siachisumo, et al.
Published: (2024-10-01)

Processing of Fluorescent Proteins May Prevent Detection of Prion Particles in [<i>PSI</i><sup>+</sup>] Cells
by: Andrew G. Matveenko, et al.
Published: (2022-11-01)