Evidence map›Paper›PMID 12235112›Full record

ArticleThe Journal of clinical investigation2002

Long-term persistence of donor nuclei in a Duchenne muscular dystrophy patient receiving bone marrow transplantation.

Emanuela Gussoni, Richard R Bennett, Kristina R Muskiewicz, Todd Meyerrose, Jan A Nolta, Irene Gilgoff, James Stein, Yiu-Mo Chan, Hart G Lidov, Carsten G Bönnemann and 6 more

Open access · greenAbstract readCase Reports
In one paragraph

Article in The Journal of clinical investigation, 2002. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 61 papers.

0numbers the graph read from it
0cells of the map it votes in
61citing papers in PubMed
7.3field-weighted citation impact, top 2% 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.

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

61 citing papers in PubMed, 197 citations in OpenAlex.

  1. Article
  2. Cell Fusion in Reprogramming and Regeneration.Advances in experimental medicine and biology · 2026
    Review
  3. Muscular Dystrophies.Advances in experimental medicine and biology · 2025
    Review
  4. Developing Advanced Chimeric Cell Therapy for Duchenne Muscular Dystrophy.International journal of molecular sciences · 2024
    Article
  5. Article
  6. Article
  7. Intraosseous transplant of dystrophin expressing chimeric (DEC) cells improves skeletal muscle function inPostepy w kardiologii interwencyjnej = Advances in interventional cardiology · 2022
    Article
  8. The Role of MSCs and Cell Fusion in Tissue Regeneration.International journal of molecular sciences · 2021
    Review
  9. Article
  10. Review
  11. Stem cell therapy for muscular dystrophies.The Journal of clinical investigation · 2020
    Review
  12. Article
  13. Review
  14. Article
  15. Article
  16. Article
  17. Molecular Therapies for Muscular Dystrophies.Current treatment options in neurology · 2018
    Review
  18. Article
  19. Article
  20. Skeletal Muscle Regenerative Potential of Human MuStem Cells following Transplantation into Injured Mice Muscle.Molecular therapy : the journal of the American Society of Gene Therapy · 2018
    Article

1 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

16 authors at 9 institutions in 4 countries.

Emanuela GussoniDivision of Genetics, Children's Hospital, Boston, Massachusetts 02115, USA.
Richard R Bennett
Kristina R Muskiewicz
Todd Meyerrose
Jan A Nolta
Irene Gilgoff
James Stein
Yiu-Mo Chan
Hart G Lidov
Carsten G Bönnemann
Arpad Von Moers
Glenn E Morris
Johan T Den Dunnen
Jeffrey S Chamberlain
Louis M Kunkel
Kenneth Weinberg
Boston Children's Hospital · USChildren's Hospital of Los Angeles · USCharité - Universitätsmedizin Berlin · DEChildren's Hospital of Philadelphia · USGeisinger Medical Center · USLeiden University Medical Center · NLRancho Los Amigos National Rehabilitation Center · USUniversity of Washington · USWrexham University · GB

Funding

ZOPOLRESTAT IN NORMOTENSIVE, TYPE 1 DIA WITH INCIPIENT NEPHROPATHYM01RR000043 · NCRR · UNIVERSITY OF SOUTHERN CALIFORNIA · PI ZAIA, JOHN A · 1985 to 2010
$76.4M
Progenitor Cells inHuman Skeletal MuscleP01NS040828 · NINDS · CHILDREN'S HOSPITAL BOSTON · PI KUNKEL, LOUIS M · 2001 to 2007
$8.6M
REGULATION OF LYMPHOPOIESIS FROM PLURIPOTENT STEM CELLSP50HL054850 · NHLBI · CHILDREN'S HOSPITAL LOS ANGELES · PI PARKMAN, ROBERTSON · 1995 to 2004
$7.5M
Immunodeficient Mouse Model of Stem Cell PlasticityR01DK061848 · NIDDK · WASHINGTON UNIVERSITY · PI NOLTA, JAN A. · 2001 to 2009
$2.7M
NCRR NIH HHS M01 RR000043NCRR NIH HHS M01 RR00043NHLBI NIH HHS P50 HL054850NHLBI NIH HHS P50 HL-54850NIDDK NIH HHS R01 DK061848NINDS NIH HHS 1P01NS40828-01NINDS NIH HHS P01 NS040828
6 · The paper itself

Abstract

Duchenne muscular dystrophy (DMD) is a severe progressive muscle-wasting disorder caused by mutations in the dystrophin gene. Studies have shown that bone marrow cells transplanted into lethally irradiated mdx mice, the mouse model of DMD, can become part of skeletal muscle myofibers. Whether human marrow cells also have this ability is unknown. Here we report the analysis of muscle biopsies from a DMD patient (DMD-BMT1) who received bone marrow transplantation at age 1 year for X-linked severe combined immune deficiency and who was diagnosed with DMD at age 12 years. Analysis of muscle biopsies from DMD-BMT1 revealed the presence of donor nuclei within a small number of muscle myofibers (0.5-0.9%). The majority of the myofibers produce a truncated, in-frame isoform of dystrophin lacking exons 44 and 45 (not wild-type). The presence of bone marrow-derived donor nuclei in the muscle of this patient documents the ability of exogenous human bone marrow cells to fuse into skeletal muscle and persist up to 13 years after transplantation.

Indexed as

Bone Marrow TransplantationAdolescentAnimalsBiopsyBone Marrow CellsCell NucleusChildDystrophinExonsFemaleHumansImmunohistochemistryInfantIn Situ Hybridization, FluorescenceMaleMuscle, SkeletalDystrophin

Identifiers

PMID12235112
PMCPMC151133
OpenAlexW2160899676

What OpenQuestion 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 OpenQuestion graph.