Resumo

MARTINEZ-MORGA, Marta et al. Mecanismo de acción de la terapia celular en enfermedades hereditarias. Medicina (B. Aires) [online]. 2020, vol.80, suppl.2, pp.2-6. ISSN 0025-7680.

Inherited metabolism disorders are serious childhood diseases that lead to significant cognitive impairment and regression of psychomotor development. The pathophysiology of the neural progressive deterioration is usually associated with severe neuroinflammation and demyelination, and as a consequence, neurodegeneration. At the moment they have no adequate treatment and require early and aggressive therapeutic approaches, which entail high mortality rates and, very frequently, low degrees of functional improvement and survival. Bone marrow transplantation and bone marrow mesenchymal cells grafts are therapeutic and experimental therapies that improve the course of these diseases through different mechanisms of action: enzyme replacement, membrane exchange and regulation of the inflammatory process.

Palavras-chave : Deposit lysosomal diseases; Cell therapy; Mesenchymal cells; Replacement therapy; Cross-correction.

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