ArticleThe New England journal of medicine2025
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease.
Article in The New England journal of medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 221 papers.
What it found
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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
221 citing papers in PubMed.
- Trial
- First-in-human nuclease-free homologous recombination-dependent gene editing in pediatric patients with methylmalonic acidemia: results of a phase 1/2 study.Gene therapy · 2026Trial
- Base Editing: Mechanisms and Therapeutic Applications.Methods in molecular biology (Clifton, N.J.) · 2027Review
- Lipid Nanoparticle-Mediated Delivery of CRISPR-Cas9 Components for Genome Editing.Methods in molecular biology (Clifton, N.J.) · 2027Article
- Non-Viral CRISPR carriers: transient delivery with lasting effects.Drug delivery · 2026Review
- Opportunities to enhance protections for individualized interventions involving gene-editing.Molecular therapy. Nucleic acids · 2026Article
- Gene therapy for hereditary hematological disorders: From clinical breakthroughs to future horizons.Molecular therapy. Nucleic acids · 2026Review
- Clinical and genetic characterization of intellectual disability.Developmental medicine and child neurology · 2026Article
- Editing the rulebook: building the policy environment for children to access gene therapy.Pediatric research · 2026Article
- Reappraising the topology of cell-free fetal DNA in maternal blood toward improved prenatal genetic diagnostics.Science advances · 2026Review
- Towards mRNA therapeutics 2.0.Nature reviews. Drug discovery · 2026Review
- Reclaiming Anatomy as Method: From Morphological Reasoning to Clinical Relevance.Clinical anatomy (New York, N.Y.) · 2026Review
- CRISPR in clinical oncology: translational advances from molecular diagnostics to therapeutics.Nature reviews. Clinical oncology · 2026Review
- From N-of-1 therapy to health-system implementation.Nature reviews. Drug discovery · 2026Article
- Systematic Profiling of ssODN Determinants Establishes Optimized LbCas12a-Mediated HDR for Precise Genome Editing inJournal of fungi (Basel, Switzerland) · 2026Article
- Individualized antisense oligonucleotide treatment eligibility of patients living with neurodevelopmental diseases.Cell reports. Medicine · 2026Review
- Amelioration of the neurological phenotype in PKU mouse model emphasizes the potential of base editing for metabolic diseases.Molecular therapy. Advances · 2026Article
- Base editing for precision therapeutics.Cell genomics · 2026Review
- CRISPR and Gene Augmentation Rescue Trabecular Meshwork Dysfunction in iPSC Models of Lowe Syndrome.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Applying the FDA's plausible mechanism guidance across the rare disease landscape.Nature genetics · 2026Article
161 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
45 authors.
Funding
Abstract
Base editors can correct disease-causing genetic variants. After a neonate had received a diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency, a disease with an estimated 50% mortality in early infancy, we immediately began to develop a customized lipid nanoparticle-delivered base-editing therapy. After regulatory approval had been obtained for the therapy, the patient received two infusions at approximately 7 and 8 months of age. In the 7 weeks after the initial infusion, the patient was able to receive an increased amount of dietary protein and a reduced dose of a nitrogen-scavenger medication to half the starting dose, without unacceptable adverse events and despite viral illnesses. No serious adverse events occurred. Longer follow-up is warranted to assess safety and efficacy. (Funded by the National Institutes of Health and others.).
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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.