Autor/es reacciones
Mercè Pineda Marfa
Paediatric neurologist at the Hospital Sant Joan de Déu and the Centro Médico Teknon
This study, in which bone illness is improved, is of unbeatable quality.
The treatment really improves the disease greatly, something that does not occur with bone marrow transplantation. The limitations, as in all gene therapy, are immune problems both in humoral and cellular immunity.
Ideally, we would have a follow-up of 10 or 15 years, but we should be satisfied with what we have now.
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