ReviewCurrent gene therapy2011
Meganucleases and other tools for targeted genome engineering: perspectives and challenges for gene therapy.
Review in Current gene therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 167 papers, 1 of them a synthesis that pooled it.
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
167 citing papers in PubMed, 1 synthesis or guideline pooled it, 404 citations in OpenAlex.
- Pooled it
- Clinical translation of epigenome editing technologies.Current opinion in biomedical engineering · 2026Article
- From mechanism to medicine: CRISPR‒Cas9 delivery strategies, therapeutic applications and translation challenges.Discover nano · 2026Review
- Non-clinical safety considerations on genome editing using the CRISPR/Cas system.Genes & diseases · 2026Review
- Heart Failure in the Molecular Era: Redefining Our Understanding of Disease Mechanisms and Perspectives.Biomedicines · 2026Review
- CRISPR and Fanzor in sickle cell disease: current progress and future prospects.Frontiers in genome editing · 2026Review
- Next-generation T cell immunotherapies engineered with CRISPR base and prime editing: challenges and opportunities.Nature reviews. Clinical oncology · 2025Review
- CRISPR-Based genome editing in pulses: current approaches, challenges, and future prospects.Plant molecular biology · 2025Review
- Genome-edited allogeneic CAR-T cells: the next generation of cancer immunotherapies.Journal of hematology & oncology · 2025Review
- Halting Recombinant Adeno-Associated Virus Transgene Expression Using mRNA-Lipid Nanoparticle-Delivered Meganucleases.Human gene therapy · 2025Article
- Genetically Modified Animal-Derived Products: From Regulations to Applications.Animals : an open access journal from MDPI · 2025Review
- Progress and challenges in CRISPR/Cas applications in microalgae.Journal of microbiology (Seoul, Korea) · 2025Review
- The Future of Gene Therapy: A Review of In Vivo and Ex Vivo Delivery Methods for Genome Editing-Based Therapies.Molecular biotechnology · 2025Review
- Review
- Antiapoptosis Engineering for Improved Protein Production from CHO Cells.Methods in molecular biology (Clifton, N.J.) · 2025Article
- Evolution of Prime Editing Systems: Move Forward to the Treatment of Hereditary Diseases.Current gene therapy · 2025Review
- Emerging tools in plant genome editing.Frontiers in genome editing · 2025Review
- Nanotechnology in aquaculture: Transforming the future of food security.Food chemistry: X · 2024Review
- CRISPR/Cas technology: fueling the future of Biofuel production with sugarcane.Functional & integrative genomics · 2024Review
- Cell-Penetrating Peptides and CRISPR-Cas9: A Combined Strategy for Human Genetic Disease Therapy.Human gene therapy · 2024Review
107 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The importance of safer approaches for gene therapy has been underscored by a series of severe adverse events (SAEs) observed in patients involved in clinical trials for Severe Combined Immune Deficiency Disease (SCID) and Chromic Granulomatous Disease (CGD). While a new generation of viral vectors is in the process of replacing the classical gamma-retrovirus-based approach, a number of strategies have emerged based on non-viral vectorization and/or targeted insertion aimed at achieving safer gene transfer. Currently, these methods display lower efficacies than viral transduction although many of them can yield more than 1% of engineered cells in vitro. Nuclease-based approaches, wherein an endonuclease is used to trigger site-specific genome editing, can significantly increase the percentage of targeted cells. These methods therefore provide a real alternative to classical gene transfer as well as gene editing. However, the first endonuclease to be in clinic today is not used for gene transfer, but to inactivate a gene (CCR5) required for HIV infection. Here, we review these alternative approaches, with a special emphasis on meganucleases, a family of naturally occurring rare-cutting endonucleases, and speculate on their current and future potential.
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.