An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype-Reference-Cited by-同舟云学术

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

Published:2018-09 Issue: Volume:10 Page:268-280
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Weber Leslie,Poletti Valentina,Magrin Elisa,Antoniani Chiara,Martin Samia,Bayard Charles,Sadek Hanem,Felix Tristan,Meneghini Vasco,Antoniou Michael N.,El-Nemer Wassim,Mavilio Fulvio,Cavazzana Marina,Andre-Schmutz Isabelle,Miccio Annarita

Funder

AFM-Telethon

European Research Council

Agence Nationale de la Recherche

EU Marie Curie-COFUND

BioMarin Pharmaceutical Inc

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

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4. Correction of sickle cell disease in transgenic mouse models by gene therapy;Pawliuk;Science,2001

5. A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction;Perumbeti;Blood,2009

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