Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia-Reference-Cited by-同舟云学术

Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia

Published:2018-12 Issue: Volume:11 Page:9-28
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Lidonnici Maria Rosa,Paleari Ylenia,Tiboni Francesca,Mandelli Giacomo,Rossi Claudia,Vezzoli Michela,Aprile Annamaria,Lederer Carsten Werner,Ambrosi Alessandro,Chanut Franck,Sanvito Francesca,Calabria Andrea,Poletti Valentina,Mavilio Fulvio,Montini Eugenio,Naldini Luigi,Cristofori Patrizia,Ferrari Giuliana

Funder

Telethon SR-Tiget

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference71 articles.

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3. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome;Aiuti;Science,2013

4. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy;Biffi;Science,2013

5. Gene Therapy in a Patient with Sickle Cell Disease;Ribeil;N. Engl. J. Med.,2017

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