Evidence map›Paper›PMID 40437086›Full record

ArticleNature2025

In vivo haemopoietic stem cell gene therapy enabled by postnatal trafficking.

Michela Milani, Anna Fabiano, Marta Perez-Rodriguez, Raisa Jofra Hernandez, Alessandra Zecchillo, Erika Zonari, Sofia Ottonello, Luca Basso-Ricci, Cesare Canepari, Monica Volpin and 19 more

Abstract read
In one paragraph

Article in Nature, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing 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

11 citing papers in PubMed.

  1. Article
  2. Article
  3. Review
  4. Review
  5. Clinical Significance of IKBKB Mutations:Saudi medical journal · 2026
    Review
  6. Article
  7. Review
  8. International journal of biological sciences · 2026
    Review
  9. Review
  10. Article
  11. 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

29 authors.

Michela MilaniSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy. milani.michela@hsr.it.ORCID 0000-0002-0363-678X
Anna FabianoSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.ORCID 0000-0002-7604-6967
Marta Perez-RodriguezBiomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain.ORCID 0009-0007-8115-4093
Raisa Jofra HernandezSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Alessandra ZecchilloSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Erika ZonariSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Sofia OttonelloSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Luca Basso-RicciSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Cesare CanepariSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Monica VolpinSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Valeria IannelloSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Valentina CapoSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.ORCID 0000-0002-1851-1429
Pamela QuarantaSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Luca SeffinSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Fabio RussoSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.ORCID 0000-0001-5280-5677
Mauro BiffiSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.ORCID 0000-0002-6914-2854
Leonardo OrmoliSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Chiara BrombinUniversity Center for Statistics in the Biomedical Sciences, Vita-Salute San Raffaele University, Milan, Italy.
Filippo CarlucciDepartment of Medical Biotechnologies, University of Siena, Siena, Italy.
Antonella ForlinoDepartment of Molecular Medicine, Biochemistry Unit, University of Pavia, Pavia, Italy.
Marta FilibianBiomedical Imaging Laboratory, Centro Grandi Strumenti, University of Pavia, Pavia, Italy.
Eugenio MontiniSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.ORCID 0000-0003-1771-6067
Serena ScalaSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Anna VillaSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Juan Antonio BuerenBiomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain.ORCID 0000-0002-3228-7013
Paula RioBiomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain.ORCID 0000-0002-5424-5543
Alessandro AiutiSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.ORCID 0000-0002-5398-1717
Alessio CantoreSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.ORCID 0000-0002-9741-997X
Luigi NaldiniSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy. naldini.luigi@hsr.it.ORCID 0000-0002-7835-527X

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Lentiviral vector (LV)-mediated ex vivo gene therapy for haematopoietic stem and progenitor cells (HSPCs) has delivered on the promise of a 'one-and-done' treatment for several genetic diseases

Indexed as

Cell MovementGenetic TherapyGene Transfer TechniquesHematopoietic Stem CellsHematopoietic Stem Cell TransplantationAdenosine DeaminaseAnimalsAnimals, NewbornBone MarrowCell LineageDisease Models, AnimalFemaleGenetic VectorsHumansLentivirusLiverAdenosine Deaminase

Identifiers

PMID40437086
PMCPMC12286858

What OpenQuestion holds

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LicenceCC BY-NC-ND
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 OpenQuestion graph.