Evidence map›Paper›PMID 33633114›Full record

ArticleNature communications2021

Lentivirus-mediated gene therapy for Fabry disease.

Aneal Khan, Dwayne L Barber, Ju Huang, C Anthony Rupar, Jack W Rip, Christiane Auray-Blais, Michel Boutin, Pamela O'Hoski, Kristy Gargulak, William M McKillop and 14 more

Registry-linked trialOpen access · goldAbstract readClinical Trial, Phase IMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Article in Nature communications, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02800070 (Clinical Pilot Study of Autologous Stem Cell Transplantation of Cluster of Differentiation 34 Positive), which is not on this map. Cited by 69 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
69citing papers in PubMed, 3 pooled it
10.9field-weighted citation impact, top 1% of its field
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.

NCT02800070 phase1completednot on this map

Clinical Pilot Study of Autologous Stem Cell Transplantation of Cluster of Differentiation 34 Positive (CD34+) Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease

TypeinterventionalSponsorUniversity Health Network, TorontoRan2016 to 2024Enrolled5ConditionsFabry DiseaseArmsLentivirus Alpha-gal A transduced stem cells
3 · Its place in the literature

Who cites it

69 citing papers in PubMed, 3 syntheses or guidelines pooled it, 116 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Pooled it
  4. Review
  5. Review
  6. Review
  7. Article
  8. The kidney in genetic metabolic disorders.Medizinische Genetik : Mitteilungsblatt des Berufsverbandes Medizinische Genetik e.V · 2026
    Article
  9. Molecular therapy. Methods & clinical development · 2025
    Article
  10. Reduced-intensity conditioning in LV-mediated gene therapy for Fabry disease targeting HSPCs.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  11. Review
  12. Review
  13. Article
  14. Progress and Challenges in the Treatment of Fabry Disease.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2025
    Review
  15. Review
  16. Review
  17. Status and frontiers of Fabre disease.Orphanet journal of rare diseases · 2025
    Review
  18. Review
  19. Article
  20. Natural History and Clinicopathological Associations of TRPC6-Associated Podocytopathy.Journal of the American Society of Nephrology : JASN · 2025
    Article

9 more citing papers are in PubMed but not listed here.

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

24 authors at 10 institutions in 2 countries.

Aneal KhanDepartment of Medical Genetics, Metabolics and Pediatrics, Alberta Children's Hospital, Cumming School of Medicine, Research Institute, University of Calgary, Calgary, AB, Canada.
Dwayne L BarberUniversity Health Network, Toronto, ON, Canada.ORCID 0000-0001-5528-8150
Ju HuangUniversity Health Network, Toronto, ON, Canada.
C Anthony RuparDepartment of Pathology and Laboratory Medicine, Western University, London, ON, Canada.
Jack W RipDepartment of Pathology and Laboratory Medicine, Western University, London, ON, Canada.
Christiane Auray-BlaisDivision of Medical Genetics, Department of Pediatrics, CIUSSS de l'Estrie-CHUS Hospital Fleurimont, Université de Sherbrooke, Sherbrooke, QC, Canada.
Michel BoutinDivision of Medical Genetics, Department of Pediatrics, CIUSSS de l'Estrie-CHUS Hospital Fleurimont, Université de Sherbrooke, Sherbrooke, QC, Canada.
Pamela O'HoskiDepartment of Pathology and Molecular Medicine, McMaster University and Juravinski Hospital and Cancer Centre, Hamilton, ON, Canada.
Kristy GargulakDepartment of Pediatrics, Medical College of Wisconsin, Milwaukee, WI, USA.
William M McKillopDepartment of Pediatrics, Medical College of Wisconsin, Milwaukee, WI, USA.
Graeme FraserDepartment of Oncology, McMaster University and Juravinski Hospital and Cancer Centre, Hamilton, ON, Canada.
Syed WasimCancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, ON, Canada.
Kaye LeMoineNova Scotia Health Authority, QEII Health Sciences Centre, Canadian Fabry Disease Initiative, Nova Scotia Fabry Disease Program, Halifax, NS, Canada.
Shelly JelinskiAlberta Children's Hospital and Foothills Medical Centre, Calgary, AB, Canada.
Ahsan ChaudhryDepartments of Oncology and Medicine, Alberta Blood and Marrow Transplant Program, University of Calgary, Calgary, AB, Canada.
Nicole ProkopishynDepartment of Pathology and Laboratory Medicine, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada.
Chantal F MorelFred A. Litwin Family Centre in Genetic Medicine, Department of Medicine, University Health Network, Toronto, ON, Canada.
Stephen CoubanDivision of Hematology, Department of Medicine, Dalhousie University, Halifax, NS, Canada.
Peter R DugganCumming School of Medicine, University of Calgary, Calgary, AB, Canada.ORCID 0000-0002-1615-2274
Daniel H FowlerRapa Therapeutics, Rockville, MD, USA.
Armand KeatingUniversity Health Network, Toronto, ON, Canada.
Michael L WestDivision of Nephrology, Department of Medicine, Dalhousie University, Halifax, NS, Canada.
Ronan FoleyDepartment of Pathology and Molecular Medicine, McMaster University and Juravinski Hospital and Cancer Centre, Hamilton, ON, Canada.
Jeffrey A MedinUniversity Health Network, Toronto, ON, Canada. jmedin@mcw.edu.ORCID 0000-0001-8165-8995
University Health Network · CAUniversity of Calgary · CAJuravinski Hospital · CADalhousie University · CAMedical College of Wisconsin · USUniversité de Sherbrooke · CAWestern University · CAAlberta Health Services · CANova Scotia Health Authority · CAPrincess Margaret Cancer Centre · CA

Funding

CIHR 119187
6 · The paper itself

Abstract

Enzyme and chaperone therapies are used to treat Fabry disease. Such treatments are expensive and require intrusive biweekly infusions; they are also not particularly efficacious. In this pilot, single-arm study (NCT02800070), five adult males with Type 1 (classical) phenotype Fabry disease were infused with autologous lentivirus-transduced, CD34

Indexed as

Adultalpha-GalactosidaseAntigens, CD34Bone Marrow CellsFabry DiseaseGenetic TherapyGenetic VectorsHematopoietic Stem CellsHumansLentivirusLeukocytesMaleMiddle AgedTrihexosylceramidesalpha-GalactosidaseAntigens, CD34GLA protein, humanglobotriaosylceramideTrihexosylceramides

Identifiers

PMID33633114
PMCPMC7907075
OpenAlexW3130363432

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

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.