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Biomaterials|August 22, 2015
TALEN-mediated functional correction of X-linked chronic granulomatous disease in patient-derived induced pluripotent stem cellsAnne-Kathrin Dreyer, Dirk Hoffmann, Nico Lachmann, et al.World Journal of Hepatology|March 13, 2018
Homologous recombination mediates stable Fah gene integration and phenotypic correction in tyrosinaemia mouse-modelNorman Junge, Qinggong Yuan, Thu Huong Vu, et al.Neuropediatrics|December 1, 2021
Clinical and Genetic Aspects of Juvenile Onset Pompe DiseaseJohanna Holzwarth, Nadja Minopoli, Charlotte Pfrimmer, et al.Cell Stem Cell|February 24, 2021
Quantitative evaluation of chromosomal rearrangements in gene-edited human stem cells by CAST-SeqGiandomenico Turchiano, Geoffroy Andrieux, Julia Klermund, et al.Hepatology (Baltimore, Md.)|July 24, 2012
MicroRNA-221 overexpression accelerates hepatocyte proliferation during liver regenerationQinggong Yuan, Komal Loya, Bhavna Rani, et al.Communications Biology|July 1, 2026
Base editing-derived models of human WDR34 and WDR60 disease alleles replicate retrograde intraflagellar transport (IFT) and hedgehog signaling defectsDinu Antony, Elif Yilmaz Güleç, Anna Klawonn, et al.Genome Biology|February 3, 2026
Functional correction and genome integrity with duplex base editing of β-thalassemic hematopoietic stem cellsNikoletta Y Papaioannou, Petros Patsali, Julia Klermund, et al.Der Nervenarzt|January 9, 2019
[Treatment evaluation in patients with 5q-associated spinal muscular atrophy : Real-world experience]Maggie C Walter, Bianca Dräger, Rene Günther, et al.Journal of Neuromuscular Diseases|April 26, 2018
Evaluation of Children with SMA Type 1 Under Treatment with Nusinersen within the Expanded Access Program in GermanyAstrid Pechmann, Thorsten Langer, David Schorling, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|March 9, 2024
On- and off-target effects of paired CRISPR-Cas nickase in primary human cellsJulia Klermund, Manuel Rhiel, Thomas Kocher, et al.Pageof 32