ArticleCell2025
In vivo prime editing rescues alternating hemiplegia of childhood in mice.
Abstract read
In one paragraph
Article in Cell, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers, 1 of them a synthesis that pooled it.
0numbers the graph read from it
0cells of the map it votes in
29citing papers in PubMed, 1 pooled it
–field-weighted citation impact
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
29 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Pooled it
- Personalized drug discovery from rare diseases to cancer.npj drug discovery · 2026Review
- Mechanism of Rescue of NaInternational journal of molecular sciences · 2026Article
- CRISPR-Cas9 and precision editing technologies linking functional genomics to clinical translation in genetic diseases.Clinical and translational medicine · 2026Review
- Donor-complementary prime editing enables precise kilobase and library-compatible DNA insertions.Nature biotechnology · 2026Article
- On-target and off-target activities of CRISPR therapeutics across scales.Trends in biotechnology · 2026Review
- Mechanistic machine learning for prediction of prime editing outcomes.Nature biotechnology · 2026Article
- Therapeutic Gene Editing of APOE4 in Sporadic Alzheimer's Disease via Prime Editor 7.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Efficient prime editing in vivo and in vitro using lipid nanoparticles.Nature nanotechnology · 2026Article
- Viral Infections and Neurodegenerative Diseases: Reinterpreting the Crosstalk Through a Dual-Role Lens.Current microbiology · 2026Review
- AI-guided redesign of laboratory-evolved reverse transcriptases enhances prime editing.Nature biotechnology · 2026Article
- Directed evolution of small RNA-stabilizing motifs that improve prime-editing efficiency.Nature biotechnology · 2026Article
- Recent Developments in Lipid Nanoparticle-Mediated Delivery of Biotherapeutics and Gene Therapy Across the Blood-Brain Barrier.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2026Review
- DNA and RNA editing for the therapy of human diseases: current status, challenges, and future prospects.Molecular biomedicine · 2026Review
- Review
- eIF3 musketeers: loyal in health, rogue in disease, and redeemed by therapeutic targeting.The EMBO journal · 2026Review
- Advances in Engineered Virus-Like Particles for Genome Editing and Therapy.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2026Review
- Prime Editing Exhibits Limited Genome-Wide Off-Target Effects in Cellular and Embryonic Gene Editing.Cells · 2026Article
- The New Wave of Gene and Cell Therapies Across Diseases.Journal of clinical medicine · 2026Review
- Mechanistic machine learning enables interpretable and generalizable prediction of prime editing outcomes.bioRxiv : the preprint server for biology · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
19 authors.
Alexander A SousaMerkin Institute of Transformative Technologies in Healthcare, The Broad Institute of Massachusetts Institute of Technology and Harvard, Cambridge, MA 02142, USA; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA 02138, USA; Howard Hughes Medical Institute, Harvard University, Cambridge, MA 02142, USA.
Markus TerreyRare Disease Translational Center, The Jackson Laboratory, Bar Harbor, ME 04609, USA.
Holt A SakaiMerkin Institute of Transformative Technologies in Healthcare, The Broad Institute of Massachusetts Institute of Technology and Harvard, Cambridge, MA 02142, USA; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA 02138, USA; Howard Hughes Medical Institute, Harvard University, Cambridge, MA 02142, USA.
Christine Q SimmonsDepartment of Pharmacology, Northwestern University Feinberg School of Medicine, Chicago, IL 60611, USA.
Elena ArystarkhovaDepartment of Neurosurgery, Massachusetts General Hospital, Boston, MA 02129, USA; Harvard Medical School, Boston, MA 02115, USA.
Natalia S MorsciRARE Hope, Washington, DC, USA.
Laura C AndersonRare Disease Translational Center, The Jackson Laboratory, Bar Harbor, ME 04609, USA.
Jun XieDepartment of Genetics & Cellular Medicine, Horae Gene Therapy Center, Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, University of Massachusetts, Worcester, MA 01605, USA; Department of Microbiology, UMass Chan Medical School, University of Massachusetts, Worcester, MA 01605, USA.
Fabian Suri-PayerDepartment of Molecular and Cellular Biology, Harvard University, Cambridge, MA 02138, USA; Broad Institute of Massachusetts Institute of Technology and Harvard, Cambridge, MA 02142, USA.
Linda C LauxDepartment of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL 60611, USA; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL 60611, USA.
Emmanuel RozeSorbonne University, Inserm, CNRS, Paris Brain Institute, APHP, Institute of Neurology, 75005 Paris, France.
Sylvie ForlaniSorbonne University, Inserm, CNRS, Paris Brain Institute, APHP, Hôpital de la Pitié Salpêtrière, 75013 Paris, France.
Guangping GaoDepartment of Genetics & Cellular Medicine, Horae Gene Therapy Center, Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, University of Massachusetts, Worcester, MA 01605, USA; Department of Microbiology, UMass Chan Medical School, University of Massachusetts, Worcester, MA 01605, USA.
Simon FrostRARE Hope, Washington, DC, USA.
Nina FrostRARE Hope, Washington, DC, USA.
Kathleen J SweadnerDepartment of Neurosurgery, Massachusetts General Hospital, Boston, MA 02129, USA; Harvard Medical School, Boston, MA 02115, USA.
Alfred L GeorgeDepartment of Pharmacology, Northwestern University Feinberg School of Medicine, Chicago, IL 60611, USA.
Cathleen M LutzRare Disease Translational Center, The Jackson Laboratory, Bar Harbor, ME 04609, USA; JAX Center for Precision Genetics, The Jackson Laboratory, Bar Harbor, ME 04609, USA. Electronic address: cat.lutz@jax.org.
David R LiuMerkin Institute of Transformative Technologies in Healthcare, The Broad Institute of Massachusetts Institute of Technology and Harvard, Cambridge, MA 02142, USA; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA 02138, USA; Howard Hughes Medical Institute, Harvard University, Cambridge, MA 02142, USA. Electronic address: drliu@fas.harvard.edu.
Funding
Center for Genomic Editing and Recording: Development and Application of Next-Generation Genome and Epigenome Editing Methods to Advance the Study and Treatment of Human DiseaseRM1HG009490 · NHGRI · WHITEHEAD INSTITUTE FOR BIOMEDICAL RES · PI Brittany S. Adamson, Martin Joseph Ankrah Aryee · 2017 to 2026
$22.7MThe Mutant Mouse Resource and Research Center at The Jackson LaboratoryU42OD010921 · OD · JACKSON LABORATORY · PI LAURA G REINHOLDT · 2012 to 2026
$21.4MThe Jackson Laboratory Center for Precision GeneticsU54OD030187 · OD · JACKSON LABORATORY · PI Cathleen M Lutz · 2020 to 2026
$17.2MSupplement to Clinical, Genetic, and Cellular Consequences of Mutations in Na,K-ATPase ATP1A3R01NS058949 · NINDS · WAKE FOREST UNIVERSITY HEALTH SCIENCES · PI BRASHEAR, ALLISON · 2008 to 2025
$8.9MIntegrating Chemistry and Evolution to Illuminate Biology and Enable Novel TherapeuticsR35GM118062 · NIGMS · HARVARD UNIVERSITY · PI LIU, DAVID R · 2016 to 2025
$6.4MCellular Pathophysiology of Neuronal Na/K-ATPase DysfunctionR01NS125785 · NINDS · NORTHWESTERN UNIVERSITY AT CHICAGO · PI Alfred L. George · 2022 to 2026
$2.0MNHGRI NIH HHS RM1 HG009490NIGMS NIH HHS R35 GM118062NIH HHS U42 OD010921NIH HHS U54 OD030187NINDS NIH HHS R01 NS058949NINDS NIH HHS R01 NS125785
Abstract
Alternating hemiplegia of childhood (AHC) is a neurodevelopmental disorder with no disease-modifying treatment. Mutations in ATP1A3, encoding an Na
Indexed as
Gene EditingHemiplegiaSodium-Potassium-Exchanging ATPaseAnimalsBrainCRISPR-Cas SystemsDisease Models, AnimalFemaleGenetic TherapyHEK293 CellsHumansMaleMiceMice, Inbred C57BLMutationATP1A3 protein, humanAtp1a3 protein, mouseSodium-Potassium-Exchanging ATPasealternating hemiplegia of childhoodCRISPR-Cas9genetic therapygenome editingneurological disorderprime editing
Identifiers
What Socratic holds
Textmetadata
LicenceCC BY
Read underepoch 390
Registered trials
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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 Socratic graph.