ReviewJournal of controlled release : official journal of the Controlled Release Society2011
Polymer delivery systems for site-specific genome editing.
Review in Journal of controlled release : official journal of the Controlled Release Society, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 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
11 citing papers in PubMed.
- In vivo correction of cystic fibrosis mediated by PNA nanoparticles.Science advances · 2022Article
- Advanced Nanotheranostics of CRISPR/Cas for Viral Hepatitis and Hepatocellular Carcinoma.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2021Review
- Peptide Nucleic Acids and Gene Editing: Perspectives on Structure and Repair.Molecules (Basel, Switzerland) · 2020Review
- Using Gene Editing Approaches to Fine-Tune the Immune System.Frontiers in immunology · 2020Review
- Therapeutic Peptide Nucleic Acids: Principles, Limitations, and Opportunities.The Yale journal of biology and medicine · 2017Review
- Gene-targeting pharmaceuticals for single-gene disorders.Human molecular genetics · 2016Review
- Modified poly(lactic-co-glycolic acid) nanoparticles for enhanced cellular uptake and gene editing in the lung.Advanced healthcare materials · 2015Article
- Therapeutic genome mutagenesis using synthetic donor DNA and triplex-forming molecules.Methods in molecular biology (Clifton, N.J.) · 2015Article
- Systemic delivery of triplex-forming PNA and donor DNA by nanoparticles mediates site-specific genome editing of human hematopoietic cells in vivo.Gene therapy · 2013Article
- The great migration of bone marrow-derived stem cells toward the ischemic brain: therapeutic implications for stroke and other neurological disorders.Progress in neurobiology · 2011Review
- Biodistribution of Polymeric Nanoparticles following in utero Delivery to a Nonhuman Primate.Biomedicine hubArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
Abstract
Triplex-forming peptide nucleic acids (PNAs) can be used to coordinate the recombination of short 50-60bp "donor DNA" fragments into genomic DNA, resulting in site-specific correction of genetic mutations or the introduction of advantageous genetic modifications. Site-specific gene editing in hematopoietic stem and progenitor cells (HSPCs) could result in the treatment or cure of inherited disorders of the blood such as β-thalassemia or sickle cell anemia. Gene editing in HSPCs and differentiated T cells could also help combat HIV infection by modifying the HIV co-receptor CCR5, which is necessary for R5-tropic HIV entry. However, translation of genome modification technologies to clinical practice is limited by challenges in intracellular delivery, especially in difficult-to-transfect hematolymphoid cells. Here, we review the use of engineered biodegradable polymer nanoparticles for site-specific genome editing in human hematopoietic cells, which represent a promising approach for ex vivo and in vivo 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.