Combining stem cells and exon skipping strategy to treat muscular dystrophy

Research output: Contribution to journalArticle

13 Citations (Scopus)

Abstract

Background: Muscular dystrophies are characterized by primary wasting of skeletal muscle. Mutations in the dystrophin gene cause hereditary muscular diseases such as Becker muscular dystrophy (BMD) and Duchenne muscular dystrophy (DMD), the most severe form. Characterization of the dystrophin gene and evidence that different types of adult stem cells are capable of muscle regeneration has lead to the development of potential gene therapy and stem cell treatments for DMD. Objectives: The main goal is to combine gene modification strategies with cell-mediated therapies. This approach could permit autologous transplantation of cells, minimizing the risk of implant rejection. Results/conclusion: The combination of gene and stem cell approaches seems to be most promising, particularly intra-arterial injections of the patient's own stem cells transduced by antisense oligonucleotide technology. This approach should offer the chance to distribute the autologous corrected stem cells to the whole body musculature providing a clinical benefit for dystrophic patients.

Original languageEnglish
Pages (from-to)1051-1061
Number of pages11
JournalExpert Opinion on Biological Therapy
Volume8
Issue number8
DOIs
Publication statusPublished - Aug 2008

Fingerprint

Muscular Dystrophies
Stem cells
Duchenne Muscular Dystrophy
Exons
Stem Cells
Dystrophin
Genes
Muscle
Intra-Arterial Injections
Inborn Genetic Diseases
Adult Stem Cells
Autologous Transplantation
Antisense Oligonucleotides
Muscular Diseases
Cell- and Tissue-Based Therapy
Gene therapy
Genetic Therapy
Regeneration
Skeletal Muscle
Technology

Keywords

  • Adult stem cells
  • AON
  • DMD
  • Exon skipping
  • Muscular dystrophies
  • Viral vectors

ASJC Scopus subject areas

  • Pharmacology
  • Biochemistry, Genetics and Molecular Biology(all)
  • Genetics
  • Immunology

Cite this

Combining stem cells and exon skipping strategy to treat muscular dystrophy. / Meregalli, Mirella; Farini, Andrea; Torrente, Yvan.

In: Expert Opinion on Biological Therapy, Vol. 8, No. 8, 08.2008, p. 1051-1061.

Research output: Contribution to journalArticle

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