Omslagsbillede

Combining Engineered U1 snRNA and Antisense Oligonucleotides to Improve the Treatment of a BBS1 Splice Site Mutation

Manipulation of pre-mRNA processing is a promising approach toward overcoming disease-causing mutations and treating human diseases. We show that a combined treatment applying two splice-manipulating technologies improves therapeutic efficacies to correct mutation-induced splice defects. Previously,...

Fuld beskrivelse

Saved in:

Bibliografiske detaljer
Main Authors:	Saskia Breuel (Author), Mariann Vorm (Author), Anja U. Bräuer (Author), Marta Owczarek-Lipska (Author), John Neidhardt (Author)
Format:	Bog
Udgivet:	Elsevier, 2019-12-01T00:00:00Z.
Fag:	article
Online adgang:	Connect to this object online.
Tags:	Tilføj Tag Ingen Tags, Vær først til at tagge denne postø!

Lignende værker

Autosomal dominant optic atrophy: A novel treatment for OPA1 splice defects using U1 snRNA adaption
af: Christoph Jüschke, et al.
Udgivet: (2021)

The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery
af: Agathe Eckenfelder, et al.
Udgivet: (2012)

Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics
af: Elena Barbon, et al.
Udgivet: (2016)

An Exon-Specific U1snRNA Induces a Robust Factor IX Activity in Mice Expressing Multiple Human FIX Splicing Mutants
af: Dario Balestra, et al.
Udgivet: (2016)

Antisense Oligonucleotide Mediated Splice Correction of a Deep Intronic Mutation in OPA1
af: Tobias Bonifert, et al.
Udgivet: (2016)

Corrigendum to "Antisense Oligonucleotide Mediated Splice Correction of a Deep Intronic Mutation in OPA1"
Udgivet: (2016)

Exploring non-coding variants and evaluation of antisense oligonucleotides for splicing redirection in Usher syndrome
af: Belén García-Bohórquez, et al.
Udgivet: (2024)

Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching
af: Kole Ryszard, et al.
Udgivet: (2011)

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

Antisense Oligonucleotide- and CRISPR-Cas9-Mediated Rescue of mRNA Splicing for a Deep Intronic CLRN1 Mutation
af: Anna-Lena Panagiotopoulos, et al.
Udgivet: (2020)

Antisense oligonucleotide therapy corrects splicing in the common Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C
af: Melita Kaltak, et al.
Udgivet: (2023)

Splice-Switching Antisense Oligonucleotides Reduce LRRK2 Kinase Activity in Human LRRK2 Transgenic Mice
af: Joanna A. Korecka, et al.
Udgivet: (2020)

Tissue pharmacokinetics of antisense oligonucleotides
af: Erica Bäckström, et al.
Udgivet: (2024)

Pharmacokinetics of antisense oligonucleotide drugs
af: M. R. Khaitov, et al.
Udgivet: (2013)

From Cryptic Toward Canonical Pre-mRNA Splicing in Pompe Disease: a Pipeline for the Development of Antisense Oligonucleotides
af: Atze J Bergsma, et al.
Udgivet: (2016)

Corrigendum to "From Cryptic Toward Canonical Pre-mRNA Splicing in Pompe Disease: a Pipeline for the Development of Antisense Oligonucleotides"
Udgivet: (2016)

Antisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T>G GAA Splicing Variant in Pompe Disease
af: Erik van der Wal, et al.
Udgivet: (2017)

Rescue of mis-splicing of a common SLC26A4 mutant associated with sensorineural hearing loss by antisense oligonucleotides
af: Pengchao Feng, et al.
Udgivet: (2022)

Antisense Oligonucleotides: Concepts and Pharmaceutical Applications
af: Ariana Araya, et al.
Udgivet: (2023)

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

Antisense Oligonucleotide-based Splice Correction for USH2A-associated Retinal Degeneration Caused by a Frequent Deep-intronic Mutation
af: Radulfus WN Slijkerman, et al.
Udgivet: (2016)

Intravitreal Injection of Splice-switching Oligonucleotides to Manipulate Splicing in Retinal Cells
af: Xavier Gérard, et al.
Udgivet: (2015)

Chimeric Antisense Oligonucleotide Conjugated to α-Tocopherol
af: Tomoko Nishina, et al.
Udgivet: (2015)

Antisense Oligonucleotide-Based Therapy of Viral Infections
af: Woan-Yuh Tarn, et al.
Udgivet: (2021)

Antisense Oligonucleotide Therapeutic Approach for Suppression of Ataxin-1 Expression: A Safety Assessment
af: Brennon O'Callaghan, et al.
Udgivet: (2020)

ASOptimizer: Optimizing antisense oligonucleotides through deep learning for IDO1 gene regulation
af: Gyeongjo Hwang, et al.
Udgivet: (2024)

Intracerebral Infusion of Antisense Oligonucleotides Into Prion-infected Mice
af: Karah Nazor Friberg, et al.
Udgivet: (2012)

Antisense oligonucleotides: a novel Frontier in pharmacological strategy
af: D. Collotta, et al.
Udgivet: (2023)

Antisense oligonucleotide is a promising intervention for liver diseases
af: Kailing Lu, et al.
Udgivet: (2022)

New treatments for myasthenia: a focus on antisense oligonucleotides
af: Angelini C, et al.
Udgivet: (2013)

Targeting DMPK with Antisense Oligonucleotide Improves Muscle Strength in Myotonic Dystrophy Type 1 Mice
af: Dominic Jauvin, et al.
Udgivet: (2017)

l-Type amino acid transporter 1-targeting nanoparticles for antisense oligonucleotide delivery to the CNS
af: Yu Na Lim, et al.
Udgivet: (2024)

Lipophilic Peptide Dendrimers for Delivery of Splice-Switching Oligonucleotides
af: Haneen Daralnakhla, et al.
Udgivet: (2021)

Multiple antisense oligonucleotides targeted against monoacylglycerol acyltransferase 1 (Mogat1) improve glucose metabolism independently of Mogat1
af: Andrew J. Lutkewitte, et al.
Udgivet: (2021)

Scavenger Receptor Class A1 Mediates Uptake of Morpholino Antisense Oligonucleotide into Dystrophic Skeletal Muscle
af: Shouta Miyatake, et al.
Udgivet: (2019)

Cyclodextrin-Based Nanoparticles for Delivery of Antisense Oligonucleotides Targeting Huntingtin
af: Monique C. P. Mendonça, et al.
Udgivet: (2023)

Antisense Oligonucleotides Targeting Angiogenic Factors as Potential Cancer Therapeutics
af: Bao T. Le, et al.
Udgivet: (2019)

Allele-Selective Knockdown of MYH7 Using Antisense Oligonucleotides
af: Brian R. Anderson, et al.
Udgivet: (2020)

Deep learning facilitates efficient optimization of antisense oligonucleotide drugs
af: Shenggeng Lin, et al.
Udgivet: (2024)

Tritium labeling of antisense oligonucleotides via different conjugation agents
af: Martin R. Edelmann, et al.
Udgivet: (2021)