Article
Potential application of gene therapy to X-linked agammaglobulinemia.
Current gene therapy - 1 Aug 2007
Moreau Thomas, Calmels Boris, Barlogis Vincent, Michel Gérard, Tonnelle Cécile, Chabannon Christian
Abstract excerpt
X-linked agammaglobulinemia (XLA), or Bruton's disease, is the most common human primary humoral immunodeficiency. XLA is caused by mutations of the Bruton's tyrosine kinase (BTK), a key regulator of B-cell physiology. Since the mid 80's, substitutive therapy by intravenous gammaglobulin infusions has significantly improved XLA patient survival and quality of life. Nevertheless, some frequent affections persist...
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