How might somatic-cell gene therapy potentially be used to treat or cure this disease?
Somatic gene therapy is the insertion of a functional gene into a cell, replacing a mutant allele which is defective. This correction of dysfunctional genes would allow for the body to produce its own antibodies. Replacing the mutant alleles involves using a vector, often a virus, to inject the new gene. The use of a viral vector is a potential problem in an immunocompromised patient who may not be able to withstand an infection. Billy’s Bruton’s tyrosine kinase gene, which is responsible for B cell production, can be replaced by an efficient protein. This will allow for healthy B cell maturation and the manufacturing of antibodies. Billy would also be able to generate immune memory obtained by vaccines. IVIG would not allow for Billy to vaccine, the chance of infection from viral components being too great. Ultimately, a gene therapy which replaced the need for IVIG, without the risks of allogenic bone marrow transplants is the best potential cure for X-linked agammaglobulinemia36). R
