Work

103. Development of an effective and safe hematopoietic stem cell gene therapy for globoid cell leukodystrophy: The unexpected issue of GALC toxicity

Year: 2008

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2007.10.115

Authors: Ilaria Visigalli, Martina Cesani, Margherita Neri, Silvia Ungari, Laura Tononi, Sergio Marchesini, Manuel Grez, Angela Gritti, Luigi Naldini, Alessandra Biffi

Institutions: The San Raffaele Telethon Institute for Gene Therapy, Vita-Salute San Raffaele University, University of Brescia, Georg Speyer Haus

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology (medical)

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W2015444807

FWCI:

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