ReviewHuman genetics2013
Silencing human genetic diseases with oligonucleotide-based therapies.
Review in Human genetics, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
23 citing papers in PubMed, 49 citations in OpenAlex.
- Gene therapy of adeno-associated virus (AAV) vectors in preclinical models of ischemic stroke.CNS neuroscience & therapeutics · 2023Review
- Review
- Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7.Science translational medicine · 2018Article
- Converging pathways in neurodegeneration, from genetics to mechanisms.Nature neuroscience · 2018Review
- [Small interfering RNA-mediated programmed cell death-ligand 1 silencing in human glioma cells enhances human CD8Nan fang yi ke da xue xue bao = Journal of Southern Medical University · 2018Article
- Therapy development in Huntington disease: From current strategies to emerging opportunities.American journal of medical genetics. Part A · 2018Review
- Transfer of genetic therapy across human populations: molecular targets for increasing patient coverage in repeat expansion diseases.European journal of human genetics : EJHG · 2016Article
- Effective Small Interfering RNA Therapy to Treat CLCN7-dependent Autosomal Dominant Osteopetrosis Type 2.Molecular therapy. Nucleic acids · 2015Article
- Oligonucleotide Therapies: The Past and the Present.Human gene therapy · 2015Review
- CD33-Targeted Lipid Nanoparticles (aCD33LNs) for Therapeutic Delivery of GTI-2040 to Acute Myelogenous Leukemia.Molecular pharmaceutics · 2015Article
- Modulation of the RNA Interference Activity Using Central Mismatched siRNAs and Acyclic Threoninol Nucleic Acids (aTNA) Units.Molecules (Basel, Switzerland) · 2015Article
- Guanidinium-rich, glycerol-derived oligocarbonates: a new class of cell-penetrating molecular transporters that complex, deliver, and release siRNA.Molecular pharmaceutics · 2015Article
- Screening nylon-3 polymers, a new class of cationic amphiphiles, for siRNA delivery.Molecular pharmaceutics · 2015Article
- Short-interference RNAs: becoming medicines.EXCLI journal · 2015Article
- In vivo gene silencing following non-invasive siRNA delivery into the skin using a novel topical formulation.Journal of controlled release : official journal of the Controlled Release Society · 2014Article
- Targets for future clinical trials in Huntington's disease: what's in the pipeline?Movement disorders : official journal of the Movement Disorder Society · 2014Review
- Oligonucleotide-based strategies to combat polyglutamine diseases.Nucleic acids research · 2014Review
- Integrating the roles of long and small non-coding RNA in brain function and disease.Molecular psychiatry · 2014Review
- E-cadherin and gastric cancer: cause, consequence, and applications.BioMed research international · 2014Review
- Striatal synaptic dysfunction and hippocampal plasticity deficits in the Hu97/18 mouse model of Huntington disease.PloS one · 2014Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
RNA interference is an endogenous mechanism present in most eukaryotic cells that enables degradation of specific mRNAs. Pharmacological exploitation of this mechanism for therapeutic purposes attracted a whole amount of attention in its initial years, but was later hampered due to difficulties in delivery of the pharmacological agents to the appropriate organ or tissue. Advances in recent years have to a certain level started to address this specific issue. Genetic diseases are caused by aberrations in gene sequences or structure; these particular abnormalities are in theory easily addressable by RNAi therapeutics. Sequencing of the human genome has largely contributed to the identification of alterations responsible for genetic conditions, thus facilitating the design of compounds that can address these diseases. This review addresses the currently on-going programs with the aim of developing RNAi and other antisense compounds for the treatment of genetic conditions and the pros and cons that these products may encounter along the way. The authors have focused on those programs that have reached clinical trials or are very close to do so.
Indexed as
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What OpenQuestion holds
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.