Evidence map›Paper›PMID 41358590›Full record

Trial reportThe New England journal of medicine2026

Prime Editing for p47

Jennifer L Gori, Elie Haddad, Haydar Frangoul, Donald B Kohn, Emma C Morris, Bradley N Martin, Briana A Deary, McKinley Nickerson, Rebecca L Scholz, Isabel Fernandez and 24 more

Abstract readClinical Trial, Phase IClinical Trial, Phase IIMulticenter Study
In one paragraph

Trial report in The New England journal of medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.

0numbers the graph read from it
0cells of the map it votes in
23citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

23 citing papers in PubMed.

  1. Prime Editing: An Overview.Methods in molecular biology (Clifton, N.J.) · 2027
    Review
  2. Review
  3. Article
  4. Review
  5. Article
  6. Review
  7. Fungal Infections in Disorders of Inborn Errors of Immunity.Clinical reviews in allergy & immunology · 2026
    Review
  8. Review
  9. Article
  10. Review
  11. Article
  12. A primer on prime: A prime editing update from advances to first-in-human trial.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Review
  13. Review
  14. Review
  15. How I Treat: Chronic granulomatous disease.Journal of human immunity · 2026
    Review
  16. Review
  17. Review
  18. Article
  19. Review
  20. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

34 authors.

Jennifer L GoriPrime Medicine, Cambridge, MA.
Elie HaddadDepartment of Pediatrics, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal.ORCID 0000-0003-2446-6879
Haydar FrangoulSarah Cannon Research Institute, Children's Hospital at TriStar Centennial, Nashville.ORCID 0000-0002-0590-2826
Donald B KohnDepartment of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles.
Emma C MorrisNational Institute for Health and Care Research, University College London Hospitals Biomedical Research Centre, London.
Bradley N MartinPrime Medicine, Cambridge, MA.
Briana A DearyPrime Medicine, Cambridge, MA.
McKinley NickersonPrime Medicine, Cambridge, MA.
Rebecca L ScholzPrime Medicine, Cambridge, MA.
Isabel FernandezDepartment of Pediatrics, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal.
Karine LeveilleDepartment of Pediatrics, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal.
Suk See De RavinLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.
Elizabeth M KangLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.
Marie PierzynskiLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.
Tyra EstwickLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.
Patricia LittelLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.
Douglas B KuhnsLeidos Biomedical Research, Frederick National Laboratory for Cancer Research, Frederick, MD.
Debra A Long PrielLeidos Biomedical Research, Frederick National Laboratory for Cancer Research, Frederick, MD.
Pierre TeiraDepartment of Pediatrics, CHU Sainte-Justine Azrieli Research Center, Université de Montréal, Montreal.
Stuart TurveyDepartment of Pediatrics, British Columbia Children's Hospital, University of British Columbia, Vancouver, Canada.
Jordyn ArnoldDepartment of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles.
Misty D EvansSarah Cannon Research Institute, Children's Hospital at TriStar Centennial, Nashville.
Meghann McManusSarah Cannon Research Institute, Children's Hospital at TriStar Centennial, Nashville.
Ben CarpenterDepartment of Hematology, University College London Hospitals NHS Foundation Trust, London.
David P WatermanPrime Medicine, Cambridge, MA.
Andrew V AnzalonePrime Medicine, Cambridge, MA.
Alexandria PetrusichPrime Medicine, Cambridge, MA.
Christa E OsunaPrime Medicine, Cambridge, MA.
Tiernan T O'MalleyPrime Medicine, Cambridge, MA.
Jacob Stewart-OrnsteinPrime Medicine, Cambridge, MA.
Jack M HeathPrime Medicine, Cambridge, MA.
Barrett J NehillaPrime Medicine, Cambridge, MA.
Mohammed AsmalPrime Medicine, Cambridge, MA.
Harry L MalechLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.

Funding

Gene Therapy Basic Research to Treat Inherited Primary Immune DeficienciesZIAAI000644 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2025
$12.1M
Development and Clinical Application of Gene and Cell therapies for Patients with Immune DeficienciesZIAAI000645 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI DE RAVIN, SUK SEE · 2009 to 2025
$10.1M
PERIPHERAL BLOOD PROGENITORS-TARGET FOR GENE TRANSFERZ01AI000644 · NIAID · NIAID EXTRAMURAL ACTIVITIES · PI MALECH, HARRY L · 1992 to 2008
$4.3M
GENE THERAPY FOR IMMUNE DEFICIENCIESZ01AI000645 · NIAID · NIAID EXTRAMURAL ACTIVITIES · PI DE RAVIN, SUK · 1992 to 2008
$873k
CCR NIH HHS HHSN261200800001CIntramural NIH HHS Z01 AI000644Intramural NIH HHS Z01 AI000645Intramural NIH HHS ZIA AI000644Intramural NIH HHS ZIA AI000645NCI NIH HHS HHSN261200800001ENIH HHS HHSN261200800001E
6 · The paper itself

Abstract

Chronic granulomatous disease (CGD) is a severe monogenic immunodeficiency caused by damaging variants in genes required for microbicidal NADPH oxidase activity. Autosomal recessive p47

Indexed as

Gene EditingGenetic TherapyGranulomatous Disease, ChronicHematopoietic Stem Cell TransplantationNADPH OxidasesAdolescentBusulfanExonsFollow-Up StudiesHumansMaleMiddle AgedNeutrophilsPseudogenesSequence DeletionTransplantation, AutologousBusulfanNADPH Oxidasesneutrophil cytosolic factor 1

Identifiers

PMID41358590
PMCPMC13533071

What Socratic holds

Textmetadata
Read underepoch 390

Registered trials

None linked

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.