ReviewHuman genetics2011
RNAi: a potential new class of therapeutic for human genetic disease.
Review in Human genetics, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 44 papers.
What it found
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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
44 citing papers in PubMed, 99 citations in OpenAlex.
- Active pH Modulation by Proton Channel-Peptide Nucleic Acid Complex for Effective siRNA Endosomal Escape.Small (Weinheim an der Bergstrasse, Germany) · 2026Article
- RNAi-based therapeutics targeting the F gene in human metapneumovirus: An in-silico approach.Journal, genetic engineering & biotechnology · 2026Article
- Designing siRNAs against non-structural genes of all serotypes of Dengue virus using RNAi technology - A computational investigation.Journal, genetic engineering & biotechnology · 2025Article
- Virus-specific Dicer-substrate siRNA swarms inhibit SARS-CoV-2 infection in TMPRSS2-expressing Vero E6 cells.Frontiers in microbiology · 2024Article
- Chitosan and Its Structural Modifications for siRNA Delivery.Advanced pharmaceutical bulletin · 2023Review
- Recruitment of a splicing factor to the nuclear lamina for its inactivation.Communications biology · 2022Article
- Chemical modifications to mRNA nucleobases impact translation elongation and termination.Biophysical chemistry · 2022Review
- siRNA Therapeutics: Future Promise for Neurodegenerative Diseases.Current neuropharmacology · 2021Review
- Applying switchable Cas9 variants to in vivo gene editing for therapeutic applications.Cell biology and toxicology · 2020Review
- Quantitative PCR primer design affects quantification of dsRNA-mediated gene knockdown.Ecology and evolution · 2019Article
- The Codelivery of siRNA and QDs by pH-Responsive Micelle for Hepatoma Cancer Cells.Frontiers in pharmacology · 2019Article
- Advances in the Application and Impact of MicroRNAs as Therapies for Skin Disease.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2017Review
- ASPsiRNA: A Resource of ASP-siRNAs Having Therapeutic Potential for Human Genetic Disorders and Algorithm for Prediction of Their Inhibitory Efficacy.G3 (Bethesda, Md.) · 2017Article
- Lentivirus-mediated RNA interference of tripartite motif 68 inhibits the proliferation of colorectal cancer cell lines SW1116 and HCT116Oncology letters · 2017Article
- Quantum Dots-siRNA Nanoplexes for Gene Silencing in Central Nervous System Tumor Cells.Frontiers in pharmacology · 2017Article
- Reductive nanocomplex encapsulation of cRGD-siRNA conjugates for enhanced targeting to cancer cells.International journal of nanomedicine · 2017Article
- Reduced stability and intracellular transport of dsRNA contribute to poor RNAi response in lepidopteran insects.RNA biology · 2016Article
- Targeting HPV-infected cervical cancer cells with PEGylated liposomes encapsulating siRNA and the role of siRNA complexation with polyethylenimine.Bioengineering & translational medicine · 2016Article
- Transfer of genetic therapy across human populations: molecular targets for increasing patient coverage in repeat expansion diseases.European journal of human genetics : EJHG · 2016Article
- Rapid Exchange Between Free and Bound States in RNA-Dendrimer Polyplexes: Implications on the Mechanism of Delivery and Release.Biomacromolecules · 2016Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Dominant negative genetic disorders, in which a mutant allele of a gene causes disease in the presence of a second, normal copy, have been challenging since there is no cure and treatments are only to alleviate the symptoms. Current therapies involving pharmacological and biological drugs are not suitable to target mutant genes selectively due to structural indifference of the normal variant of their targets from the disease-causing mutant ones. In instances when the target contains single nucleotide polymorphism (SNP), whether it is an enzyme or structural or receptor protein are not ideal for treatment using conventional drugs due to their lack of selectivity. Therefore, there is a need to develop new approaches to accelerate targeting these previously inaccessible targets by classical therapeutics. Although there is a cooling trend by the pharmaceutical industry for the potential of RNA interference (RNAi), RNAi and other RNA targeting drugs (antisense, ribozyme, etc.) still hold their promise as the only drugs that provide an opportunity to target genes with SNP mutations found in dominant negative disorders, genes specific to pathogenic tumor cells, and genes that are critical for mediating the pathology of various other diseases. Because of its exquisite specificity and potency, RNAi has attracted a considerable interest as a new class of therapeutic for genetic diseases including amyotrophic lateral sclerosis, Huntington's disease (HD), Alzheimer's disease (AD), Parkinson's disease (PD), spinocerebellar ataxia, dominant muscular dystrophies, and cancer. In this review, progress and challenges in developing RNAi therapeutics for genetic diseases will be discussed.
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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.