Towards gene therapy of sickle cell disease

Eric E. Bouhassira, A. S. Acharya

Research output: Contribution to journalArticle

Abstract

Sickle cell disease (SCD) is a significant health problem in the United States, in part of Europe and in many developing countries. Progress has been made in the management of the disease but a cure is still elusive except in the rare cases where bone marrow transplant is a practical option. Hopes for a complete universal cure rest mostly on two gene therapy approaches: correcting the mutation that causes the disease, or adding a corrective gene to the genome of the patient. The former approach is extremely attractive but is in its early stages of development. One of the most important remaining obstacles for the latter approach is the control of expression of a transgene integrated at ectopic chromosomal sites. Animal models for SCD should help in the development of the gene therapy methods discussed above, as well as the pharmacological approaches that aim at ameliorating or eliminating the symptoms of SCD.

Original languageEnglish (US)
Pages (from-to)1081-1093
Number of pages13
JournalExpert Opinion on Therapeutic Patents
Volume10
Issue number7
StatePublished - 2000

Fingerprint

gene therapy
Sickle Cell Anemia
Genetic Therapy
Disease
Disease Management
Transgenes
Developing Countries
Animal Models
Bone Marrow
Genome
Pharmacology
Transplants
Mutation
Health
Genes
animal
developing country
cause
health
management

Keywords

  • Anti-sickling globins
  • Cre recombinase
  • Gene therapy
  • Insulator
  • Recombinase-mediated cassette exchange
  • Sickle cell disease

ASJC Scopus subject areas

  • Medicine (miscellaneous)
  • Pharmacology
  • Law

Cite this

Towards gene therapy of sickle cell disease. / Bouhassira, Eric E.; Acharya, A. S.

In: Expert Opinion on Therapeutic Patents, Vol. 10, No. 7, 2000, p. 1081-1093.

Research output: Contribution to journalArticle

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