Pinedo AMT, Rodríguez LD, Arellanos IEA, Mendoza EDM, Gómez CF, Correa BMD

Language: Spanish
References: 5
Page: 34-42
PDF size: 903.88 Kb.

ABSTRACT

A few weeks ago, an article was published in the journal Nature Medicine demonstrating the success of cell-gene therapy (CGT) for the treatment of a serious disease, Wiskott Aldrich Syndrome. This is a Primary Immunodeficiency or Inborn Error of Immunity due to an alteration in hematopoiesis. This affects coagulation and innate and acquired (adaptive) immune responses. In this work, we describe the contribution of molecular, cellular, and immunological technology to offer this quasi-curative therapy, and to monitor the reestablished functions of platelets and immune cells. We present a brief description of WAS and CGT outcomes in nine patients reported in the aforementioned article who were followed up for four to nine years. One patient died, but not as consequence of the treatment. In the others, coagulation was fully or partially restored, so thrombocytopenia disappeared or markedly improved. And in all eight survivors, both blood cell counts and functions, including antibody production, generation of an adecuate TcR repertoire, and immune synapse formation, were restored. Finally, we discuss some perspectives on the long-term efficacy and safety of CGT, its limitations and possible ethical aspects to consider for its use.

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