ArticleGene therapy1999
Peptide nucleic acid delivery to human mitochondria.
Article in Gene therapy, 1999. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 43 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
43 citing papers in PubMed, 177 citations in OpenAlex.
- Emerging technology has a brilliant future: the CRISPR-Cas system for senescence, inflammation, and cartilage repair in osteoarthritis.Cellular & molecular biology letters · 2024Review
- Mitochondrial Homeostasis in VSMCs as a Central Hub in Vascular Remodeling.International journal of molecular sciences · 2023Review
- Role of mitophagy in ocular neurodegeneration.Frontiers in neuroscience · 2023Review
- Advances in Human Mitochondria-Based Therapies.International journal of molecular sciences · 2022Review
- Artificial genetic polymers against human pathologies.Biology direct · 2022Review
- Mitochondrial Genome Editing to Treat Human Osteoarthritis-A Narrative Review.International journal of molecular sciences · 2022Review
- Mitochondrial DNA disorders: from pathogenic variants to preventing transmission.Human molecular genetics · 2021Review
- A photoswitchable fluorescent protein for hours-time-lapse and sub-second-resolved super-resolution imaging.Microscopy (Oxford, England) · 2021Article
- Engineering Genetic Systems for Treating Mitochondrial Diseases.Pharmaceutics · 2021Review
- Natural and Artificial Mechanisms of Mitochondrial Genome Elimination.Life (Basel, Switzerland) · 2021Review
- Myogenic Cell Transplantation in Genetic and Acquired Diseases of Skeletal Muscle.Frontiers in genetics · 2021Review
- Non-Peptidic Cell-Penetrating Motifs for Mitochondrion-Specific Cargo Delivery.Angewandte Chemie (International ed. in English) · 2018Article
- Improving the Phototherapeutic Efficiencies of Molecular and Nanoscale Materials by Targeting Mitochondria.Molecules (Basel, Switzerland) · 2018Review
- Recent Advances in Mitochondria-Targeted Gene Delivery.Molecules (Basel, Switzerland) · 2018Review
- Advances in methods for reducing mitochondrial DNA disease by replacing or manipulating the mitochondrial genome.Essays in biochemistry · 2018Review
- Assessing the Delivery of Molecules to the Mitochondrial Matrix Using Click Chemistry.Chembiochem : a European journal of chemical biology · 2016Article
- Emerging therapies for mitochondrial disorders.Brain : a journal of neurology · 2016Review
- Mitochondrial DNA disease-molecular insights and potential routes to a cure.Experimental cell research · 2014Review
- Alkyne-azide "click" chemistry in designing nanocarriers for applications in biology.Molecules (Basel, Switzerland) · 2013Review
- Novel therapeutic approaches for Leber's hereditary optic neuropathy.Discovery medicine · 2013Review
Corrections and comments
- Commented on by
- Erratum issued
Authors and funding
6 authors at 2 institutions in 2 countries.
Funding
Abstract
Peptide nucleic acids (PNAs) are synthetic polynucleobase molecules, which bind to DNA and RNA with high affinity and specificity. Although PNAs have enormous potential as anti-sense agents, the success of PNA-mediated gene therapy will require efficient cellular uptake and sub-cellular trafficking. At present these mechanisms are poorly understood. To address this, we have studied the uptake of biotinylated PNAs into cultured cell lines using fluorescence confocal microscopy. In human myoblasts, initial punctate staining was followed by the release of PNAs into the cytosol and subsequent localisation and concentration in the nucleus. To determine whether PNAs could also be used as therapeutic agents for mtDNA disease, we attempted to localise PNAs to the mitochondrial matrix. When attached to the presequence peptide of the nuclear-encoded human cytochrome c oxidase (COX) subunit VIII, the biotinylated PNA was successfully imported into isolated organelles in vitro. Furthermore, delivery of the biotinylated peptide-PNA to mitochondria in intact cells was confirmed by confocal microscopy. These studies demonstrate that biotinylated PNAs can be directed across cell membranes and to a specific sub-cellular compartment within human cells - highlighting the importance of these novel molecules for human gene therapy.
Indexed as
Identifiers
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.