Abstract
Deficiency of adenosine deaminase (ADA) activity causes an autosomally inherited form of severe combined immunodeficiency (ADA¯SC1D) disease (1, 2). It has been suggested that this form of SCID is caused by a defect in T- and B-cell differentiation due to the accumulation of adenine nucleosides in the absence of functional ADA (2). The cloning of sequences encoding human ADA (3, 4, 5) opened new ways to investigate the molecular basis of ADA¯SCID disease (6–10) and allowed studies aimed at the development of gene therapy protocols for ADA¯SCID patients (11–15).
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Valerio, D. et al. (1989). Towards Gene Therapy for Adenosine Deaminase Deficiency. In: Baum, S.J., Dicke, K.A., Lotzová, E., Pluznik, D.H. (eds) Experimental Hematology Today—1988. Experimental Hematology Today—1988, vol 1988. Springer, New York, NY. https://doi.org/10.1007/978-1-4613-8862-3_14
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DOI: https://doi.org/10.1007/978-1-4613-8862-3_14
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