ArticleBone marrow transplantation2018
Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy.
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In one paragraph
Article in Bone marrow transplantation, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
1.5field-weighted citation impact, top 18% of its field
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
7 citing papers in PubMed, 12 citations in OpenAlex.
- Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.Lancet (London, England) · 2022Trial
- Astaxanthin limits atherosclerosis and dysmetabolism in mice by attenuating inflammatory cell recruitment and signaling.PloS one · 2025Article
- Is the Endothelium the Missing Link in the Pathophysiology and Treatment of COVID-19 Complications?Cardiovascular drugs and therapy · 2022Review
- Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.Biomolecules · 2021Review
- Insight into Mechanobiology: How Stem Cells Feel Mechanical Forces and Orchestrate Biological Functions.International journal of molecular sciences · 2019Review
- Adipose Stem Cell Translational Applications: From Bench-to-Bedside.International journal of molecular sciences · 2018Review
- Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
30 authors at 8 institutions in 2 countries.
Valeria CalbiSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Francesca FumagalliSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Giulia ConsiglieriPediatric Immunohematology Unit and Stem Cell Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Rachele PenatiPediatric Immunohematology Unit and Stem Cell Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Serena AcquatiSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Daniela RedaelliSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Vanessa AttanasioSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Facchini MarcellaSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Maria Pia CicaleseSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Maddalena MigliavaccaSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Federica BarzaghiSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Francesca FerruaSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Andrea AssanelliPediatric Immunohematology Unit and Stem Cell Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Paolo SilvaniDepartment of Anesthesia and Critical Care, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Matteo ZoccolilloSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Giovanna ChidiniPediatric Intensive Care Unit, Department of Anesthesia and Critical Care, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
Robert ChiesaBone Marrow Transplantation Unit, Great Ormond Street Hospital, London, UK.
Ruchi AroraDepartment of Paediatrics, Norfolk and Norwich University Hospital, Norwich, UK.
Francesca CiottiPediatric Immunohematology Unit and Stem Cell Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Marina SarzanaPediatric Immunohematology Unit and Stem Cell Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Gigliola AntonioliSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Cristina BaldoliNeuroradiology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Francesco MorenaDepartment of Chemistry, Biology and Biotechnologies, University of Perugia, Perugia, Italy.
Sabata MartinoDepartment of Chemistry, Biology and Biotechnologies, University of Perugia, Perugia, Italy.
Gian Luigi ArdissinoHUS Center, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Maria Grazia Natali SoraNeurology Department, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Luigi NaldiniSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Fabio CiceriVita-Salute San Raffaele University, Milan, Italy.
Alessandro AiutiSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy. aiuti.alessandro@hsr.it.
Maria Ester BernardoSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Istituti di Ricovero e Cura a Carattere Scientifico · ITIstituto di Ricovero e Cura a Carattere Scientifico San RaffaeleThe San Raffaele Telethon Institute for Gene Therapy · ITVita-Salute San Raffaele University · ITFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico · ITUniversity of Perugia · ITNorfolk and Norwich University Hospital · GBUniversity College London · GB
Funding
No grant is acknowledged in the PubMed record.
Abstract
PubMed holds no abstract for this paper.
Indexed as
Genetic TherapyHematopoietic Stem Cell TransplantationHumansLeukodystrophy, MetachromaticMalePlatelet Aggregation InhibitorsPolydeoxyribonucleotidesTransplantation ConditioningTwinsdefibrotidePlatelet Aggregation InhibitorsPolydeoxyribonucleotides
Identifiers
What OpenQuestion holds
Textmetadata
LicenceCC BY
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.