Will gene therapy trump factor treatment in hemophilia?

Savita Rangarajan*, Louis Aledort

*Corresponding author for this work

    Research output: Contribution to journalLiterature reviewpeer-review

    2 Citations (Scopus)

    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
    Publication statusPublished - Feb 2013

    Keywords

    • ex vivo gene therapy
    • gene therapy
    • hemophilia
    • inhibitors
    • long-acting factor VIII/IX
    • RECOMBINANT FACTOR-VIII
    • FC FUSION PROTEIN
    • FACTOR-IX
    • PEGYLATED LIPOSOMES
    • PROLONGED ACTIVITY
    • ENDOTHELIAL-CELLS
    • LONG
    • EXPRESSION
    • ANTIBODIES
    • PHARMACOKINETICS

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