ArticleNucleic acids research2016
Automated design of hammerhead ribozymes and validation by targeting the PABPN1 gene transcript.
Article in Nucleic acids research, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.
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Who cites it
9 citing papers in PubMed, 28 citations in OpenAlex.
- Enhanced hammerhead ribozyme turnover rates: Reevaluating therapeutic space for small catalytic RNAs.Molecular therapy. Nucleic acids · 2025Article
- Hammerhead ribozymes directed against mRNA of an essential gene inhibitFrontiers in microbiology · 2025Article
- Specificity of oligonucleotide gene therapy (OGT) agents.Theranostics · 2022Review
- Detection ofLife (Basel, Switzerland) · 2021Article
- MoiRNAiFold: a novel tool for complex in silico RNA design.Nucleic acids research · 2021Article
- Oligonucleotide-Based Approaches to Inhibit Dengue Virus Replication.Molecules (Basel, Switzerland) · 2021Review
- RNA-Based Therapy Utilizing Oculopharyngeal Muscular Dystrophy Transcript Knockdown and Replacement.Molecular therapy. Nucleic acids · 2019Article
- Argonaute-based programmable RNase as a tool for cleavage of highly-structured RNA.Nucleic acids research · 2018Article
- PABPN1 gene therapy for oculopharyngeal muscular dystrophy.Nature communications · 2017Article
Corrections and comments
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Authors and funding
10 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
We present a new publicly accessible web-service, RiboSoft, which implements a comprehensive hammerhead ribozyme design procedure. It accepts as input a target sequence (and some design parameters) then generates a set of ranked hammerhead ribozymes, which target the input sequence. This paper describes the implemented procedure, which takes into consideration multiple objectives leading to a multi-objective ranking of the computer-generated ribozymes. Many ribozymes were assayed and validated, including four ribozymes targeting the transcript of a disease-causing gene (a mutant version of PABPN1). These four ribozymes were successfully tested in vitro and in vivo, for their ability to cleave the targeted transcript. The wet-lab positive results of the test are presented here demonstrating the real-world potential of both hammerhead ribozymes and RiboSoft. RiboSoft is freely available at the website http://ribosoft.fungalgenomics.ca/ribosoft/.
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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.