ReviewNature medicine2019
Principles of and strategies for germline gene therapy.
Review in Nature medicine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 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
27 citing papers in PubMed, 1 synthesis or guideline pooled it.
- A Decade of Progress in Gene Targeted Therapeutic Strategies in Duchenne Muscular Dystrophy: A Systematic Review.Frontiers in bioengineering and biotechnology · 2022Pooled it
- Gene Therapy Using Recombinant Adeno-Associated Virus for Leber Congenital Amaurosis Induced by RPE65 Mutation.Clinical ophthalmology (Auckland, N.Z.) · 2026Review
- The Fomivirsen, Patisiran, and Givosiran Odyssey: How the Success Stories May Pave the Way for Future Clinical Translation of Nucleic Acid Drugs.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2025Review
- Telomerase reverse transcriptase gene knock-in unleashes enhanced longevity and accelerated damage repair in mice.Aging cell · 2025Article
- Current views and trends of nanomaterials as vectors for gene delivery since the 21st century: a bibliometric analysis.Nanomedicine (London, England) · 2025Article
- Smart Nanobiomaterials for Gene Delivery in Localized Cancer Therapy: An Overview from Emerging Materials and Devices to Clinical Applications.Current cancer drug targets · 2025Review
- Developing hydrogels for gene therapy and tissue engineering.Journal of nanobiotechnology · 2024Review
- Developmental progression of DNA double-strand break repair deciphered by a single-allele resolution mutation classifier.Nature communications · 2024Article
- Current Landscape of Gene Therapy for the Treatment of Cardiovascular Disorders.Current gene therapy · 2024Review
- Molecular basis for maternal inheritance of human mitochondrial DNA.Nature genetics · 2023Article
- Initial heritable genome editing: mapping a responsible pathway from basic research to the clinic.Medicine, health care, and philosophy · 2023Article
- Transdermal delivery for gene therapy.Drug delivery and translational research · 2022Review
- Site-specific genome editing in treatment of inherited diseases: possibility, progress, and perspectives.Medical review (2021) · 2022Review
- Single-cell multiomics analyses of spindle-transferred human embryos suggest a mostly normal embryonic development.PLoS biology · 2022Article
- Gene Therapy for Mitochondrial Diseases: Current Status and Future Perspective.Pharmaceutics · 2022Review
- Correction of the pathogenic mutation in TGM1 gene by adenine base editing in mutant embryos.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Article
- New progress in the study of germline susceptibility genes of myeloid neoplasms.Oncology letters · 2021Review
- Pressure-Driven Mitochondrial Transfer Pipeline Generates Mammalian Cells of Desired Genetic Combinations and Fates.Cell reports · 2020Article
- Gene therapy: a double-edged sword with great powers.Molecular and cellular biochemistry · 2020Review
- Ante-natal counseling in phacomatoses.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2020Article
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
Monogenic disorders occur at a high frequency in human populations and are commonly inherited through the germline. Unfortunately, once the mutation has been transmitted to a child, only limited treatment options are available in most cases. However, means of correcting disease-causing nuclear and mitochondrial DNA mutations in gametes or preimplantation embryos have now been developed and are commonly referred to as germline gene therapy (GGT). We will discuss these novel strategies and provide a path forward for safe, high-efficiency GGT that may provide a promising new paradigm for preventing the passage of deleterious genes from parent to child.
Indexed as
Identifiers
31160821What 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.