Novel candidate disease for gene therapy: Metachromatic leukodystrophy

Alessandra Biffi, Luigi Naldini

Research output: Contribution to journalArticle

Abstract

Metachromatic leukodystrophy (MLD) is a rare, fatal, inherited, autosomal recessive, lysosomal storage disorder, characterized by severe and progressive demyelination affecting the central and peripheral nervous systems. Despite some initial expectations in hematopoietic stem cell transplantation, and despite the ameliorated supportive therapy, MLD remains a life-threatening disease, with an extremely poor quality of life and a severe prognosis for all affected patients. Prospectively, in children affected by MLD, who have no other therapeutic option and an extremely poor prognosis, the potential risks associated with the use of a novel technology, such as gene therapy, might be well balanced by the potential benefit of a positive outcome. Thus, MLD might be considered an optimal candidate disease for testing innovative and potentially efficacious therapeutic approaches. Some of the gene therapy approaches discussed here, such as hematopoietic stem cells gene therapy, are likely to enter clinical testing in the near future.

Original languageEnglish
Pages (from-to)1193-1205
Number of pages13
JournalExpert Opinion on Biological Therapy
Volume7
Issue number8
DOIs
Publication statusPublished - Aug 2007

Fingerprint

Metachromatic Leukodystrophy
Gene therapy
Genetic Therapy
Stem cells
Neurology
Testing
Hematopoietic Stem Cell Transplantation
Peripheral Nervous System
Demyelinating Diseases
Cell- and Tissue-Based Therapy
Hematopoietic Stem Cells
Therapeutics
Central Nervous System
Quality of Life
Technology

Keywords

  • Gene therapy
  • Metachromatic leukodystrophy

ASJC Scopus subject areas

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

Cite this

Novel candidate disease for gene therapy : Metachromatic leukodystrophy. / Biffi, Alessandra; Naldini, Luigi.

In: Expert Opinion on Biological Therapy, Vol. 7, No. 8, 08.2007, p. 1193-1205.

Research output: Contribution to journalArticle

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