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Human Gene Therapy|April 30, 2019
Retroviral UNC13D Gene Transfer Restores Cytotoxic Activity of T Cells Derived from Familial Hemophagocytic Lymphohistiocytosis Type 3 Patients In VitroViviane Dettmer, Kristie Bloom, Miriam Gross, et al.Scientific Reports|September 19, 2014
Novel lentiviral vectors with mutated reverse transcriptase for mRNA delivery of TALE nucleasesUlrike Mock, Kristoffer Riecken, Belinda Berdien, et al.Gene Therapy|February 2, 2021
Targeted multi-epitope switching enables straightforward positive/negative selection of CAR T cellsLaura Mosti, Lukas M Langner, Kay O Chmielewski, et al.Journal of Neuromuscular Diseases|January 11, 2021
Safety Monitoring of Gene Therapy for Spinal Muscular Atrophy with Onasemnogene Abeparvovec -A Single Centre ExperienceJohannes Friese, Stephanie Geitmann, Dorothea Holzwarth, et al.Scientific Reports|October 1, 2017
Targeted genome editing restores T cell differentiation in a humanized X-SCID pluripotent stem cell disease modelJamal Alzubi, Celeste Pallant, Claudio Mussolino, et al.Human Gene Therapy|July 27, 2026
Systematic Benchmarking of CRISPR-Cas9 Off-Target Prediction Tools Reveals Limitations and Implications for Preclinical AssessmentMasako M Kaufmann, Maren Hackenberg, William Jobson Pargeter, et al.The Patient|February 5, 2019
Health-Related Quality of Life in Patients with Adult-Onset Myotonic Dystrophy Type 1: A Systematic ReviewErik Landfeldt, Josefin Edström, Cecilia Jimenez-Moreno, et al.Arthritis Research & Therapy|October 31, 2012
Muscle-fiber transdifferentiation in an experimental model of respiratory chain myopathyNils Venhoff, Dirk Lebrecht, Dietmar Pfeifer, et al.European Journal of Immunology|January 31, 2022
Immunopathology caused by impaired CD8+ T-cell responsesPeter Aichele, Christoph Neumann-Haefelin, Stephan Ehl, et al.Human Gene Therapy|December 22, 2020
HIV Gene Therapy: An UpdateTatjana I Cornu, Claudio Mussolino, Matthias C Müller, et al.Pageof 32