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Gene therapy approaches for wiskott-aldrich syndrome

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We will develop gene therapy approaches for wiskott-Aldrich syndrome, based on ex vivo transduction of T lymphocytes or hematopoietic progenitors with retroviral vectors. Efficacy and safety will be assessed in preclinical studies.

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Koordynator

FONDAZIONE CENTRO SAN RAFFAELE DEL MONTE TABOR - ISTITUTO DI RICOVERO E CURA A CARATTERE SCIENTIFICO
Wkład UE
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Adres
Via Olgettina 58
20132 MILANO
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