Preclinical Development of Autologous Hematopoietic Stem Cell-Based Gene Therapy for Immune Deficiencies: A Journey from Mouse Cage to Bed Side

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Authors: Laura Garcia-Perez, Anita Ordas, Kirsten Canté-Barrett, Pauline Meij, Karin Pike-Overzet, Arjan Lankester, Frank J. T. Staal

Journal title: Pharmaceutics

Journal number: 12/6

Journal publisher: Multidisciplinary Digital Publishing Institute (MDPI)

Published year: 2020

Published pages: 549

DOI identifier: 10.3390/pharmaceutics12060549

ISSN: 1999-4923