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La beta-talasemia es un trastorno congénito causado generalmente por mutaciones puntuales en el gen que codifica para la síntesis de cadenas beta de la globina, lo que produce un fenómeno de eritropoyesis ineficaz, y reducción de la vida media de los eritrocitos en las formas más severas. Dentro de la clasificación se engloban formas de talasemia de diversos comportamientos clínicos; desde formas severas hasta formas asintomáticas. Las complicaciones de las talasemias muchas veces se asocian al tratamiento crónico de la misma. El tratamiento actual conlleva transfusiones sanguíneas a repetición, entre otros. El desarrollo de nuevas terapias dirigidas mitiga los efectos adversos asociados a las transfusiones, además de intervenir directamente en la parte genética.
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