ReviewMolecular diagnosis & therapy2020
The Future of In Utero Gene Therapy.
Review in Molecular diagnosis & therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 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
27 citing papers in PubMed, 40 citations in OpenAlex.
- Translating base editors: a decade of progress from the bench to the clinic.Nature reviews. Drug discovery · 2026Review
- Current Status of Cellular and Gene-Based Therapies for Congenital Metabolic Disorders: A Review.Medical science monitor : international medical journal of experimental and clinical research · 2026Review
- Review
- Defining a Midgestational Window forbioRxiv : the preprint server for biology · 2026Article
- Prenatal Management of Spinal Muscular Atrophy in the Era of Genetic Screening and Emerging Opportunities in In Utero Therapy.Biomedicines · 2025Review
- Prenatal gene editing for neurodevelopmental diseases: Ethical considerations.American journal of human genetics · 2025Review
- A Review over Mitochondrial Diseases Due to mtDNA Mutations: Recent Advances and Remedial Aspects.Infectious disorders drug targets · 2025Review
- Nanomedicine for Maternal and Fetal Health.Small (Weinheim an der Bergstrasse, Germany) · 2024Review
- Evaluating sheep hemoglobins with MD simulations as an animal model for sickle cell disease.Scientific reports · 2024Article
- Emerging Multi-omic Approaches to the Molecular Diagnosis of Mitochondrial Disease and Available Strategies for Treatment and Prevention.Current genomics · 2024Review
- In Utero Cell Treatment of Hemophilia A Mice via Human Amniotic Fluid Mesenchymal Stromal Cell Engraftment.International journal of molecular sciences · 2023Article
- Intraplacental injection of AAV9-CMV-iCre results in the widespread transduction of multiple organs in double-reporter mouse embryos.Experimental animals · 2023Article
- Ionizable Lipid Nanoparticles for Therapeutic Base Editing of Congenital Brain Disease.ACS nano · 2023Article
- Gene modification therapies for hereditary diseases in the fetus.Prenatal diagnosis · 2023Review
- Comment on: Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets.Gene therapy · 2023Article
- Trends in research related to fetal therapy from 2012 to 2022: a bibliometric analysis.Frontiers in pediatrics · 2023Article
- Enhancing Molecular Testing for Effective Delivery of Actionable Gene Diagnostics.Bioengineering (Basel, Switzerland) · 2022Review
- Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets.Gene therapy · 2022Article
- Regenerative medicine: prenatal approaches.The Lancet. Child & adolescent health · 2022Review
- Recent advances in mitochondrial diseases: From molecular insights to therapeutic perspectives.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
Abstract
Significant advances in the safety and efficacy of gene therapy have sparked a new frontier in therapeutics for genetic diseases as evidenced by the greater than 700 active gene therapy investigational new drug applications reported by the NIH and the US Food and Drug Association. Although postnatal gene therapy trials are encouraging, limitations to effective therapy including an immune barrier and initiation of treatment after disease onset can exist. Advances in prenatal diagnostics provide hope that many genetic abnormalities will be able to be diagnosed before birth. Prenatal gene therapy has the potential to take advantage of normal developmental properties of the fetus and overcome some of the current limitations to efficient postnatal gene therapy. The rationale for prenatal gene therapy includes the small fetal size, the tolerogenic fetal immune system, the presence of highly proliferative and accessible stem/progenitor cells of multiple organs, and, ultimately, the ability to treat diseases in which irreversible pathology begins prior to birth. This rationale is based on and supported by a number of published animal studies. Unique ethical considerations exist in the context of prenatal gene therapy, including the importance of rigorous evaluation of the effect of the therapy on fetal germ cells and developing organs as well as the mother. To date, animal studies have not demonstrated any significant germline or maternal effect of prenatal gene therapy. Finally, practical considerations of future clinical prenatal gene therapy will include, but not be limited to, determining the initial target disease characteristics and the importance of non-directive prenatal counseling of families carrying a fetus with a genetic diagnosis.
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.