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Related Experiment Videos

Gene therapy for hemophilia.

C M Lynch1

  • 1Targeted Genetics Corporation, Seattle, WA 98101 USA. lynchc@targen.com

Current Opinion in Molecular Therapeutics
|November 21, 2001
PubMed
Summary

Gene therapy offers new hope for hemophilia (a bleeding disorder). Adeno-associated virus vectors show promise for sustained clotting factor expression, potentially leading to a cure.

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Area of Science:

  • Hematology
  • Gene Therapy
  • Genetic Disorders

Background:

  • Hemophilia (A and B) is a serious inherited bleeding disorder due to clotting factor deficiencies.
  • Current treatments involve factor infusions, which are problematic and lack prophylactic options.
  • Gene therapy development for hemophilia has been ongoing for a decade.

Purpose of the Study:

  • To review the progress of gene therapy for hemophilia.
  • To discuss various gene delivery strategies and vector types.
  • To evaluate the potential for a curative treatment.

Main Methods:

  • Overview of early retroviral and adenoviral vector efforts for ex vivo and in vivo gene delivery.
  • Review of recent adeno-associated virus (AAV) vector development for FIX and FVIII.
  • Assessment of preclinical data in murine and canine hemophilia models.

Main Results:

  • Sustained therapeutic levels of FIX and FVIII expression achieved in mice.
  • Phenotypic correction of hemophilia B demonstrated in animal models.
  • Initiation of a Phase I human clinical trial for FIX gene therapy.

Conclusions:

  • Adeno-associated virus vectors show significant promise for hemophilia gene therapy.
  • Preclinical success suggests a potential for sustained therapeutic effects.
  • Hemophilia gene therapy is transitioning from possible to probable for a cure.

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