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Gene therapy for leukocyte adhesion deficiency.
1Department of Medicine, University of Washington, VA Puget Sound Health Care System, 1660 S Columbian Way (151), Seattle, WA 98108, USA. tbauer@u.washington.edu
Current Opinion in Molecular Therapeutics
|March 16, 2001
Summary
Gene transfer of the CD18 subunit offers a promising new treatment for Leukocyte Adhesion Deficiency (LAD), an immunodeficiency disease. This approach aims to correct the underlying molecular defect, improving leukocyte function and potentially offering a curative option beyond transplantation.
Area of Science:
- Immunology
- Genetics
- Hematology
Background:
- Leukocyte Adhesion Deficiency (LAD) is a severe inherited immunodeficiency.
- It stems from defects in the CD18 integrin, impairing leukocyte adhesion and migration.
- Current treatments like stem cell transplantation have significant limitations.
Purpose of the Study:
- To review Leukocyte Adhesion Deficiency (LAD).
- To discuss preclinical studies of CD18 gene transfer.
- To present initial results from a clinical trial using CD18 gene therapy.
Main Methods:
- Experimental studies on CD18 gene transfer in LAD leukocytes.
- Retroviral-mediated gene transfer of CD18.
- Clinical trial in LAD patients with severe deficiency.
Main Results:
- Preclinical studies demonstrated CD18 gene transfer corrects LAD leukocyte defects.
- Initial clinical trial results are presented for two LAD patients.
Conclusions:
- CD18 gene transfer shows potential as a curative treatment for LAD.
- This approach may overcome limitations associated with hematopoietic stem cell transplantation.