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CD18 foamy virus gene therapy

Development stage
Preclinical
Lead developer
National Institutes of Health
Modality
Gene Therapies, Stem Cell Therapies → Cell Therapies
Administration
Intravenous
01

Overview

CD18 foamy virus gene therapy is an experimental ex vivo gene therapy designed to treat Leukocyte Adhesion Deficiency type 1 (LAD-1), a rare genetic immunodeficiency. The therapy involves the transduction of autologous CD34+ hematopoietic stem cells with a foamy virus (FV) vector carrying a functional copy of the ITGB2 gene, which encodes the CD18 protein (the beta-2 subunit of integrins). Foamy virus vectors are utilized due to their large transgene capacity, stable integration, and lack of known pathogenicity in humans. In preclinical and veterinary studies, particularly in dogs with canine leukocyte adhesion deficiency (CLAD), this approach has demonstrated long-term (4-7 years) restoration of CD18 expression and reversal of the disease phenotype following nonmyeloablative conditioning.

Other names
FV-CD18FV-CD-18FV-CD 18Foamy virus-mediated CD18 gene therapyITGB2 foamy virus gene therapyITGB-2 foamy virus gene therapyITGB 2 foamy virus gene therapy
02

Targets

ITGB2 (Complement receptor 4)

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