Gene Therapy for X-linked agammaglobulinemia (XLA)

Gene Therapy for X-linked agammaglobulinemia (XLA)

X-linked agammaglobulinemia most common primary immunodeficiency in man and caused by mutations in the BTK gene. XLA is characterized by a B-cell differentiation arrest and recurrent bacterial infections that can be fatal. The aim of this project is to develop a lentiviral gene therapy to correct the BTK gene, as potentially curative treatment for XLA.