ReviewCritical reviews in biochemistry and molecular biology
Targeted gene therapies: tools, applications, optimization.
Review in Critical reviews in biochemistry and molecular biology. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 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
17 citing papers in PubMed.
- CRISPR-Cas9: Transforming Functional Genomics, Precision Medicine, and Drug Development - Opportunities, Challenges, and Future Directions.Current gene therapy · 2026Review
- Genome Editing Approaches Using Zinc Finger Nucleases (ZFNs) for the Treatment of Motor Neuron Diseases.Current pharmaceutical biotechnology · 2025Review
- Cyclin B1: A potential prognostic and immunological biomarker in pan-cancer.Biomolecules & biomedicine · 2024Article
- AlPaCas: allele-specific CRISPR gene editing through a protospacer-adjacent-motif (PAM) approach.Nucleic acids research · 2024Article
- Emerging Gene Therapeutics for Epidermolysis Bullosa under Development.International journal of molecular sciences · 2024Review
- Adeno-Associated Viral Vector Immobilization and Local Delivery from Bare Metal Surfaces.Methods in molecular biology (Clifton, N.J.) · 2022Article
- Sequence-specific recognition of a coding segment of humanRSC advances · 2021Article
- Targeted Therapy in Cardiovascular Disease: A Precision Therapy Era.Frontiers in pharmacology · 2021Review
- Cracking the Monoubiquitin Code of Genetic Diseases.International journal of molecular sciences · 2020Review
- CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future.Frontiers in oncology · 2020Review
- Methods for Enhancing Clustered Regularly Interspaced Short Palindromic Repeats/Cas9-Mediated Homology-Directed Repair Efficiency.Frontiers in genetics · 2019Review
- Quantifying on- and off-target genome editing.Trends in biotechnology · 2015Review
- Stimulation of oligonucleotide-directed gene correction by Redβ expression and MSH2 depletion in human HT1080 cells.Molecules and cells · 2015Article
- Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery.eLife · 2014Article
- Novel fluorescent genome editing reporters for monitoring DNA repair pathway utilization at endonuclease-induced breaks.Nucleic acids research · 2014Article
- TAL effectors: highly adaptable phytobacterial virulence factors and readily engineered DNA-targeting proteins.Trends in cell biology · 2013Review
- Epigenetic modification of the repair donor regulates targeted gene correction.Molecular therapy. Nucleic acids · 2012Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
Abstract
Many devastating human diseases are caused by mutations in a single gene that prevent a somatic cell from carrying out its essential functions, or by genetic changes acquired as a result of infectious disease or in the course of cell transformation. Targeted gene therapies have emerged as potential strategies for treatment of such diseases. These therapies depend upon rare-cutting endonucleases to cleave at specific sites in or near disease genes. Targeted gene correction provides a template for homology-directed repair, enabling the cell's own repair pathways to erase the mutation and replace it with the correct sequence. Targeted gene disruption ablates the disease gene, disabling its function. Gene targeting can also promote other kinds of genome engineering, including mutation, insertion, or gene deletion. Targeted gene therapies present significant advantages compared to approaches to gene therapy that depend upon delivery of stably expressing transgenes. Recent progress has been fueled by advances in nuclease discovery and design, and by new strategies that maximize efficiency of targeting and minimize off-target damage. Future progress will build on deeper mechanistic understanding of critical factors and pathways.
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.