Gene therapy in the treatment of Fanconi anemia, a progressive bone marrow failure syndrome

David A. Williams, James Croop, Patrick Kelly

Research output: Contribution to journalArticle

12 Citations (Scopus)

Abstract

Fanconi anemia (FA) is a genetic disease characterized by progressive, fatal bone marrow failure, congenital anomalies and predisposition to cancer. Although stem cell transplantation is therapeutic, human leukocyte antigen-identical sibling donors are available to a minority of patients. In murine models and human cells in vitro, gene transfer corrects the FA cellular phenotype of chromosomal breakage in response to DNA-damaging agents, suggesting therapeutic use of gene transfer is possible. However, disease-specific characteristics make application of viral vector technology difficult. Multiple studies are currently underway to develop a gene therapy approach for treating this disease, including phase I trials.

Original languageEnglish
Pages (from-to)461-466
Number of pages6
JournalCurrent Opinion in Molecular Therapeutics
Volume7
Issue number5
StatePublished - Oct 2005

Fingerprint

Fanconi Anemia
Genetic Therapy
Chromosome Breakage
Inborn Genetic Diseases
Stem Cell Transplantation
Therapeutic Uses
HLA Antigens
Genes
Siblings
Bone Marrow
Tissue Donors
Technology
Phenotype
DNA
Therapeutics
Neoplasms
Bone Marrow failure syndromes
In Vitro Techniques

Keywords

  • Aplastic anemia
  • Chromosomal instability
  • DNA-damaging agent
  • Fanconi anemia
  • Gene transfer
  • Stem cell

ASJC Scopus subject areas

  • Genetics
  • Genetics(clinical)
  • Pharmacology, Toxicology and Pharmaceutics(all)

Cite this

Gene therapy in the treatment of Fanconi anemia, a progressive bone marrow failure syndrome. / Williams, David A.; Croop, James; Kelly, Patrick.

In: Current Opinion in Molecular Therapeutics, Vol. 7, No. 5, 10.2005, p. 461-466.

Research output: Contribution to journalArticle

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