Will gene therapy trump factor treatment in hemophilia?

Savita Rangarajan, Louis Aledort

Research output: Contribution to journalReview articlepeer-review

2 Scopus citations

Abstract

Hemophilia treatment is entering a new phase, with the exciting possibility of gene therapy promising a cure. Novel gene transfer strategies are being considered for patients with inhibitors.Improvement of factor-replacement therapy is being aggressively pursued with long-acting factor concentrates, many of which are in clinical trials. Whether gene therapy will be safe and cost effective to eventually supersede factor-replacement therapy is yet to be determined. It is hoped that with the profusion of clinical trial programs in hemophilia care, it will eventually provide affordable treatment to many patients who currently cannot access adequate treatment in the developing countries.

Original languageEnglish
Pages (from-to)43-48
Number of pages6
JournalExpert Review of Hematology
Volume6
Issue number1
DOIs
StatePublished - Feb 2013

Keywords

  • ex vivo gene therapy
  • gene therapy
  • hemophilia
  • inhibitors
  • long-acting factor VIII/IX

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