Gene therapy progress and prospects: Gene therapy for the hemophilias

Research output: Contribution to journalReview articlepeer-review

40 Scopus citations

Abstract

Recent gene transfer trials for hemophilia A and B, bleeding disorders lacking either functional factor VIII or IX, respectively, have produced tantalizing results, suggesting that the potential to correct these bleeding disorders at a molecular level may be at hand. Genetic correction of the hemophilias represents a model system to develop a basic understanding of how gene therapy will be achieved. The goals for hemophilia gene transfer require the long-term therapeutic production of the coagulant protein without stimulating an immune response to the transgene product or the vector. Based on a scientific understanding of the molecular and cellular defects, leading to the bleeding phenotype, impressive strides have been made in the last 2 years.

Original languageEnglish
Pages (from-to)999-1003
Number of pages5
JournalGene Therapy
Volume10
Issue number12
DOIs
StatePublished - Jun 2003

Keywords

  • Gene repair
  • Gene transfer
  • Hemophilia
  • Stem cells
  • Viral vectors

Fingerprint

Dive into the research topics of 'Gene therapy progress and prospects: Gene therapy for the hemophilias'. Together they form a unique fingerprint.

Cite this