Lack of mRNA and dystrophin expression in DMD patients three months after myoblast transfer

L. Morandi, P. Bernasconi, M. Gebbia, M. Mora, F. Crosti, R. Mantegazza, F. Cornelio

Research output: Contribution to journalArticle

Abstract

We report our experience on myoblast transplantation in three Duchenne muscular dystrophy patients. Pure myoblasts (55 × 106 per patient) from HLA-matched donors, were injected into a tibialis anterior and the controlateral muscle was sham injected. Three months after transplantation, biopsies from the injected muscles were negative for dystrophin expression by immunocytochemistry. Reverse transcriptase-PCR (RT-PCR) failed to amplify any fragments of the deleted regions. This result confirms that myoblast transplantation is feasible, although the efficacy of this therapeutic approach is poor.

Original languageEnglish
Pages (from-to)291-295
Number of pages5
JournalNeuromuscular Disorders
Volume5
Issue number4
DOIs
Publication statusPublished - 1995

Fingerprint

Dystrophin
Myoblasts
Transplantation
Messenger RNA
Muscles
Duchenne Muscular Dystrophy
Reverse Transcriptase Polymerase Chain Reaction
Immunohistochemistry
Tissue Donors
Biopsy
Therapeutics

Keywords

  • DMD
  • dystrophin mRNA
  • myoblast transfer

ASJC Scopus subject areas

  • Genetics(clinical)
  • Clinical Neurology
  • Pediatrics, Perinatology, and Child Health
  • Neurology
  • Developmental Neuroscience

Cite this

Lack of mRNA and dystrophin expression in DMD patients three months after myoblast transfer. / Morandi, L.; Bernasconi, P.; Gebbia, M.; Mora, M.; Crosti, F.; Mantegazza, R.; Cornelio, F.

In: Neuromuscular Disorders, Vol. 5, No. 4, 1995, p. 291-295.

Research output: Contribution to journalArticle

@article{31bdc2488f464795b1118590b8474d1f,
title = "Lack of mRNA and dystrophin expression in DMD patients three months after myoblast transfer",
abstract = "We report our experience on myoblast transplantation in three Duchenne muscular dystrophy patients. Pure myoblasts (55 × 106 per patient) from HLA-matched donors, were injected into a tibialis anterior and the controlateral muscle was sham injected. Three months after transplantation, biopsies from the injected muscles were negative for dystrophin expression by immunocytochemistry. Reverse transcriptase-PCR (RT-PCR) failed to amplify any fragments of the deleted regions. This result confirms that myoblast transplantation is feasible, although the efficacy of this therapeutic approach is poor.",
keywords = "DMD, dystrophin mRNA, myoblast transfer",
author = "L. Morandi and P. Bernasconi and M. Gebbia and M. Mora and F. Crosti and R. Mantegazza and F. Cornelio",
year = "1995",
doi = "10.1016/0960-8966(94)00070-P",
language = "English",
volume = "5",
pages = "291--295",
journal = "Neuromuscular Disorders",
issn = "0960-8966",
publisher = "Elsevier Ltd",
number = "4",

}

TY - JOUR

T1 - Lack of mRNA and dystrophin expression in DMD patients three months after myoblast transfer

AU - Morandi, L.

AU - Bernasconi, P.

AU - Gebbia, M.

AU - Mora, M.

AU - Crosti, F.

AU - Mantegazza, R.

AU - Cornelio, F.

PY - 1995

Y1 - 1995

N2 - We report our experience on myoblast transplantation in three Duchenne muscular dystrophy patients. Pure myoblasts (55 × 106 per patient) from HLA-matched donors, were injected into a tibialis anterior and the controlateral muscle was sham injected. Three months after transplantation, biopsies from the injected muscles were negative for dystrophin expression by immunocytochemistry. Reverse transcriptase-PCR (RT-PCR) failed to amplify any fragments of the deleted regions. This result confirms that myoblast transplantation is feasible, although the efficacy of this therapeutic approach is poor.

AB - We report our experience on myoblast transplantation in three Duchenne muscular dystrophy patients. Pure myoblasts (55 × 106 per patient) from HLA-matched donors, were injected into a tibialis anterior and the controlateral muscle was sham injected. Three months after transplantation, biopsies from the injected muscles were negative for dystrophin expression by immunocytochemistry. Reverse transcriptase-PCR (RT-PCR) failed to amplify any fragments of the deleted regions. This result confirms that myoblast transplantation is feasible, although the efficacy of this therapeutic approach is poor.

KW - DMD

KW - dystrophin mRNA

KW - myoblast transfer

UR - http://www.scopus.com/inward/record.url?scp=0029027377&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=0029027377&partnerID=8YFLogxK

U2 - 10.1016/0960-8966(94)00070-P

DO - 10.1016/0960-8966(94)00070-P

M3 - Article

C2 - 7580241

AN - SCOPUS:0029027377

VL - 5

SP - 291

EP - 295

JO - Neuromuscular Disorders

JF - Neuromuscular Disorders

SN - 0960-8966

IS - 4

ER -