ArticleNucleic acids research2011
A novel screening system improves genetic correction by internal exon replacement.
Article in Nucleic acids research, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
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Who cites it
21 citing papers in PubMed.
- Is It Scientifically Possible To 'Cure" Reward Deficiency Syndrome (RDS) Via Transplice Molecular Genetic Technology?Acta scientific neurology · 2025Article
- RNA exon editing: Splicing the way to treat human diseases.Molecular therapy. Nucleic acids · 2024Review
- Evaluating a Targeted Cancer Therapy Approach Mediated by RNAInternational journal of molecular sciences · 2022Article
- Investigational Treatments for Epidermolysis Bullosa.American journal of clinical dermatology · 2021Review
- Therapeutic applications of trans-splicing.British medical bulletin · 2020Article
- Epidermolysis bullosa: Advances in research and treatment.Experimental dermatology · 2019Review
- An ex vivo RNA trans-splicing strategy to correct human generalized severe epidermolysis bullosa simplex.The British journal of dermatology · 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
- Molecular barcoding of viral vectors enables mapping and optimization of mRNARNA (New York, N.Y.) · 2018Article
- RNA Trans-Splicing Modulation via Antisense Molecule Interference.International journal of molecular sciences · 2018Article
- Junctional Epidermolysis Bullosa: Allelic Heterogeneity and Mutation Stratification for Precision Medicine.Frontiers in medicine · 2018Article
- RNA-based therapies for genodermatoses.Experimental dermatology · 2017Review
- 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
- A Gene Gun-mediated Nonviral RNA trans-splicing Strategy for Col7a1 Repair.Molecular therapy. Nucleic acids · 2016Article
- 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
- Trans-splicing improvement by the combined application of antisense strategies.International journal of molecular sciences · 2015Article
- Considerations for a Successful RNA Trans-splicing Repair of Genetic Disorders.Molecular therapy. Nucleic acids · 2014Article
- The design and optimization of RNA trans-splicing molecules for skin cancer therapy.Molecular oncology · 2013Article
Corrections and comments
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Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Trans-splicing is a powerful approach to reprogram the genome. It can be used to replace 5', 3' or internal exons. The latter approach has been characterized by low efficiency, as the requirements to promote internal trans-splicing are largely uncharacterized. The trans-splicing process is induced by engineered 'RNA trans-splicing molecules' (RTMs), which target a selected pre-mRNA to be reprogrammed via two complementary binding domains. To facilitate the development of more efficient RTMs for therapeutic applications we constructed a novel fluorescence based screening system. We incorporated exon 52 of the COL17A1 gene into a GFP-based cassette system as the target exon. This exon is mutated in many patients with the devastating skin blistering disease epidermolysis bullosa. In a double transfection assay we were able to rapidly identify optimal binding domains targeted to sequences in the surrounding introns 51 and 52. The ability to replace exon 52 was then evaluated in a more endogenous context using a target containing COL17A1 exon 51-intron 51-exon 52-intron 52-exon 53. Two selected RTMs produced significantly higher levels of GFP expression in up to 61% assayed cells. This novel approach allows for rapid identification of efficient RTMs for internal exon replacement.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.