Gene therapy for hemophilia

G. Hortelano, Patricia L. Chang

Research output: Contribution to journalArticle

13 Citations (Scopus)

Abstract

Hemophilia A and B are X-linked genetic disorders caused by deficiency of the coagulation factors VIII and IX, respectively. Because of the health hazards and costs of current product replacement therapy, much effort is devoted to the development of gene therapy for these disorders. Approaches to gene therapy for the hemophilias include: ex vivo gene therapy in which cells from the intended recipients are explanted, genetically modified to secrete Factor VIII or IX, and reimplanted into the donor; in vivo gene therapy in which Factor VIII or IX encoding vectors are directly injected into the recipient; and non-autologous gene therapy in which universal cell lines engineered to secrete Factor VIII or IX are enclosed in immuno-protective devices before implantation into recipients. Research into these approaches is aided by the many murine and canine models available. While problems of achieving high and sustained levels of factor delivery, and issues related to efficacy, safety and cost are still to be resolved, progress in gene therapy for the hemophilias has been encouraging and is likely to reach human clinical trial in the foreseeable future.

Original languageEnglish
Pages (from-to)1-24
Number of pages24
JournalArtificial Cells, Blood Substitutes, and Immobilization Biotechnology
Volume28
Issue number1
Publication statusPublished - Jan 2000
Externally publishedYes

Fingerprint

Gene therapy
Hemophilia A
Genetic Therapy
Factor IX
Factor VIII
Protective Devices
Hemophilia B
Health hazards
Inborn Genetic Diseases
Coagulation
Health Care Costs
Canidae
Costs
Cells
Clinical Trials
Safety
Costs and Cost Analysis
Cell Line
Research

Keywords

  • Adenoassociated virus
  • Adenovirus
  • Alginate
  • Factor IX
  • Factor VIII
  • Immuno-isolation
  • Microcapsule
  • Retrovirus

ASJC Scopus subject areas

  • Biomedical Engineering
  • Hematology
  • Biotechnology
  • Biomaterials

Cite this

Gene therapy for hemophilia. / Hortelano, G.; Chang, Patricia L.

In: Artificial Cells, Blood Substitutes, and Immobilization Biotechnology, Vol. 28, No. 1, 01.2000, p. 1-24.

Research output: Contribution to journalArticle

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