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Journal of Clinical Medicine|November 27, 2019
The Scope for Thalassemia Gene Therapy by Disruption of Aberrant Regulatory ElementsPetros Patsali, Claudio Mussolino, Petros Ladas, et al.
Molecular Therapy. Nucleic Acids|August 8, 2025
Off-target effects in CRISPR-Cas genome editing for human therapeutics: Progress and challengesNechama Kalter, Carla Fuster-García, Alfredo Silva, et al.
Orphanet Journal of Rare Diseases|January 23, 2019
SMArtCARE - A platform to collect real-life outcome data of patients with spinal muscular atrophyAstrid Pechmann, Kirsten König, Günther Bernert, et al.
Journal of Neuromuscular Diseases|March 13, 2023
The Dilemma of Choice for Duchenne Patients Eligible for Exon 51 Skipping The European ExperienceAnnemieke Aartsma-Rus, Liesbeth De Waele, Saskia Houwen-Opstal, et al.
Brain : a Journal of Neurology|December 23, 2025
Treatment evolution in spinal muscular atrophy: insights from the SMArtCARE registryCornelia Voigt-Müller, Michelle Pfaffenlehner, Günther Bernert, et al.
Neural Regeneration Research|September 4, 2025
ID3-depleted human induced pluripotent stem cell-derived neural stem/progenitor cells promote neurorepairJia-Di Lin, Ruba Hammad, Prateek Kumar, et al.
Frontiers in Genome Editing|June 9, 2022
Genome Editing With TALEN, CRISPR-Cas9 and CRISPR-Cas12a in Combination With AAV6 Homology Donor Restores T Cell Function for XLPBenjamin C Houghton, Neelam Panchal, Simone A Haas, et al.
Plos One|December 24, 2011
Selection-independent generation of gene knockout mouse embryonic stem cells using zinc-finger nucleasesAnna Osiak, Frank Radecke, Eva Guhl, et al.
Nucleic Acids Research|March 1, 2012
Engineered zinc finger nickases induce homology-directed repair with reduced mutagenic effectsCherie L Ramirez, Michael T Certo, Claudio Mussolino, et al.
Human Gene Therapy|October 18, 2021
Gene Therapy "Made in Germany": A Historical Perspective, Analysis of the Status Quo, and Recommendations for Action by the German Society for Gene TherapyHildegard Büning, Boris Fehse, Zoltán Ivics, et al.
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