ArticleMolecular therapy : the journal of the American Society of Gene Therapy2014
Cis-splicing and translation of the pre-trans-splicing molecule combine with efficiency in spliceosome-mediated RNA trans-splicing.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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Who cites it
20 citing papers in PubMed, 36 citations in OpenAlex.
- RNAMolecular therapy. Nucleic acids · 2025Article
- RNA exon editing: Splicing the way to treat human diseases.Molecular therapy. Nucleic acids · 2024Review
- Unheeded SARS-CoV-2 proteins? A deep look into negative-sense RNA.Briefings in bioinformatics · 2022Article
- Therapeutic applications of trans-splicing.British medical bulletin · 2020Article
- Efficient system for upstream mRNA trans-splicing to generate covalent, head-to-tail, protein multimers.Scientific reports · 2019Article
- Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA.Molecular therapy. Nucleic acids · 2018Article
- RNA Structure Design Improves Activity and Specificity of trans-Splicing-Triggered Cell Death in a Suicide Gene Therapy Approach.Molecular therapy. Nucleic acids · 2018Article
- RNA Trans-Splicing Modulation via Antisense Molecule Interference.International journal of molecular sciences · 2018Article
- Gene Therapy via Trans-Splicing for LMNA-Related Congenital Muscular Dystrophy.Molecular therapy. Nucleic acids · 2018Article
- Centronuclear myopathies under attack: A plethora of therapeutic targets.Journal of neuromuscular diseases · 2018Review
- Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid.Vision (Basel, Switzerland) · 2017Review
- Expression of Herpes Simplex Virus Thymidine Kinase/Ganciclovir by RNA Trans-Splicing Induces Selective Killing of HIV-Producing Cells.Molecular therapy. Nucleic acids · 2017Article
- RNA-based therapies for genodermatoses.Experimental dermatology · 2017Review
- Optimization ofFrontiers in neuroscience · 2017Article
- Construction and validation of an RNA trans-splicing molecule suitable to repair a large number of COL7A1 mutations.Gene therapy · 2016Article
- Designing Efficient Double RNA trans-Splicing Molecules for Targeted RNA Repair.International journal of molecular sciences · 2016Article
- Reprogramming the Dynamin 2 mRNA by Spliceosome-mediated RNA Trans-splicing.Molecular therapy. Nucleic acids · 2016Article
- mRNA trans-splicing in gene therapy for genetic diseases.Wiley interdisciplinary reviews. RNA · 2016Review
- Can we be SMaRT-er in our approach to cancer therapy?EBioMedicine · 2015Article
- Repair of rhodopsin mRNA by spliceosome-mediated RNA trans-splicing: a new approach for autosomal dominant retinitis pigmentosa.Molecular therapy : the journal of the American Society of Gene Therapy · 2015Article
Corrections and comments
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Authors and funding
7 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Muscular dystrophies are a group of genetically distinct diseases for which no treatment exists. While gene transfer approach is being tested for several of these diseases, such strategies can be hampered when the size of the corresponding complementary DNA (cDNA) exceeds the packaging capacity of adeno-associated virus vectors. This issue concerns, in particular, dysferlinopathies and titinopathies that are due to mutations in the dysferlin (DYSF) and titin (TTN) genes. We investigated the efficacy of RNA trans-splicing as a mode of RNA therapy for these two types of diseases. Results obtained with RNA trans-splicing molecules designed to target the 3' end of mouse titin and human dysferlin pre-mRNA transcripts indicated that trans-splicing of pre-mRNA generated from minigene constructs or from the endogenous genes was achieved. Collectively, these results provide the first demonstration of DYSF and TTN trans-splicing reprogramming in vitro and in vivo. However, in addition to these positive results, we uncovered a possible issue of the technique in the form of undesirable translation of RNA pre-trans-splicing molecules, directly from open reading frames present on the molecule or associated with internal alternative cis-splicing. These events may hamper the efficiency of the trans-splicing process and/or lead to toxicity.
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Registered trials
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