Gene Therapy for X-linked agammaglobulinemia (XLA)

Start project: 2021
Involved institutes: LUMC
Stage: Preclinical
Status: Ongoing

Description: X-linked agammaglobulinemia (XLA) is the 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 a potentially curative treatment for XLA.

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