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Human Gene Therapy|July 20, 2019
Challenges of Gene Therapy for the Treatment of Glycogen Storage Diseases Type I and Type IIILouisa Jauze, Laure Monteillet, Gilles Mithieux, et al.Human Gene Therapy|February 23, 2019
New Human Chromosomal Sites with "Safe Harbor" Potential for Targeted Transgene InsertionStefan Pellenz, Michael Phelps, Weiliang Tang, et al.Human Gene Therapy|February 28, 2019
Dysregulated Expression of microRNA-21 and Disease-Related Genes in Human Patients and in a Mouse Model of Alport SyndromeJifan Guo, Wenping Song, Joseph Boulanger, et al.Human Gene Therapy|November 21, 2018
Synergistic Antitumor Effect on Bladder Cancer by Rational Combination of Programmed Cell Death 1 Blockade and CRISPR-Cas9-Mediated Long Non-Coding RNA Urothelial Carcinoma Associated 1 KnockoutShuai Zhen, Jiaojiao Lu, Wei Chen, et al.Human Gene Therapy|February 19, 2019
Lentiviral Gene Therapy for Bone Repair Using Human Umbilical Cord Blood-Derived Mesenchymal Stem CellsSofia Bougioukli, Biagio Saitta, Osamu Sugiyama, et al.Human Gene Therapy|March 9, 2019
Enhanced Transduction of Macaca fascicularis Hematopoietic Cells with Chimeric Lentiviral VectorsKarine Sii-Felice, Javier Castillo Padilla, Francis Relouzat, et al.Human Gene Therapy|February 12, 2021
Adeno-Associated Virus-Mediated Gene Therapy in the Mashlool, Atp1a3, Mouse Model of Alternating Hemiplegia of ChildhoodArsen S Hunanyan, Boris Kantor, Ram S Puranam, et al.Human Gene Therapy|February 8, 2021
Characterization and Functional Analysis of CD44v6.CAR T Cells Endowed with a New Low-Affinity Nerve Growth Factor Receptor-Based SpacerAnna Stornaiuolo, Barbara Valentinis, Camilla Sirini, et al.Human Gene Therapy|March 8, 2017
CRISPR-Mediated Knockout of Cybb in NSG Mice Establishes a Model of Chronic Granulomatous Disease for Human Stem-Cell Gene Therapy TransplantsColin L Sweeney, Uimook Choi, Chengyu Liu, et al.Human Gene Therapy|February 11, 2005
Tumor-specific gene delivery mediated by a novel peptide-polyethylenimine-DNA polyplex targeting aminopeptidase N/CD13Stanley Moffatt, Sandra Wiehle, Richard J CristianoPageof 414