Preclinical Testing of the Safety and Tolerability of Lentiviral Vector–Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies-Reference-Cited by-同舟云学术

Preclinical Testing of the Safety and Tolerability of Lentiviral Vector–Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies

Published:2016-10 Issue:10 Volume:27 Page:813-829
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Visigalli Ilaria¹,Delai Stefania¹,Ferro Francesca¹,Cecere Francesca¹,Vezzoli Michela¹,Sanvito Francesca¹²,Chanut Franck³⁴,Benedicenti Fabrizio¹,Spinozzi Giulio¹,Wynn Rob⁵,Calabria Andrea¹,Naldini Luigi¹⁶,Montini Eugenio¹,Cristofori Patrizia¹³,Biffi Alessandra¹

Affiliation:

1. San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Division of Regenerative Medicine, Stem Cells and Gene Therapy, Milan, Italy

2. Department of Pathology, IRCCS San Raffaele Scientific Institute, Milan, Italy

3. Glaxo Smith Kline R&D UK Safety Assessment, Stevenage, United Kingdom

4. Sanofi, Montpellier, France

5. Royal Manchester Children's Hospital, Manchester, United Kingdom.

6. Vita Salute San Raffaele University, Milan, Italy

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2016.068

Reference35 articles.

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2. ALLELISM, NON-ALLELISM, AND GENETIC COMPOUNDS AMONG THE MUCOPOLYSACCHARIDOSES

3. Transplantation of Umbilical-Cord Blood in Babies with Infantile Krabbe's Disease

4. Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study

5. Hematopoietic Cell Transplantation for MPS Patients Is Safe and Effective: Results after Implementation of International Guidelines

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