ArticleJournal of community genetics2021
Curative gene therapies for rare diseases.
Article in Journal of community genetics, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 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
12 citing papers in PubMed, 26 citations in OpenAlex.
- Advanced therapy medicinal products (ATMPs) - An analysis of the global regulatory status.British journal of clinical pharmacology · 2026Article
- Cell and Gene Therapy in Equine Ocular Disease.Veterinary ophthalmology · 2026Review
- Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program.Human gene therapy · 2025Article
- Public perceptions and willingness to accept somatic gene therapy: A Belgian survey study.Open research Europe · 2025Article
- Review
- Thymidylate synthase disruption to limit cell proliferation in cell therapies.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Generating Synthetic Light-Adapted Electroretinogram Waveforms Using Artificial Intelligence to Improve Classification of Retinal Conditions in Under-Represented Populations.Journal of ophthalmology · 2024Article
- CRISPR correction of the Finnish ornithine delta-aminotransferase mutation restores metabolic homeostasis in iPSC from patients with gyrate atrophy.Molecular genetics and metabolism reports · 2022Article
- A clinical, histopathological, and molecular study of two cases of VEXAS syndrome without a definitive myeloid neoplasm.Blood advances · 2022Article
- Gene Therapy and Hemophilia: Where Do We Go from Here?Journal of blood medicine · 2022Review
- Simultaneous high-efficiency base editing and reprogramming of patient fibroblasts.Stem cell reports · 2021Article
- Rare diseases: past achievements and future prospects.Journal of community genetics · 2021Article
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 at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Diseases caused by alterations in the DNA can be overcome by providing the cells or tissues with a functional copy of the mutated gene. The most common form of gene therapy implies adding an extra genetic unit into the cell. However, new genome engineering techniques also allow the modification or correction of the existing allele, providing new possibilities, especially for dominant diseases. Gene therapies have been tested for 30 years in thousands of clinical trials, but presently, we have only three authorised gene therapy products for the treatment of inherited diseases in European Union. Here, we describe the gene therapy alternatives already on the market in the European Union and expand the scope to some clinical trials. Additionally, we discuss the ethical and regulatory issues raised by the development of these new kinds of therapies.
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.