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Plos Pathogens|July 15, 2010
Integration preferences of wildtype AAV-2 for consensus rep-binding sites at numerous loci in the human genomeDaniela Hüser, Andreas Gogol-Döring, Timo Lutter, et al.Nucleic Acids Research|August 19, 2010
Autonomous zinc-finger nuclease pairs for targeted chromosomal deletionCem Söllü, Kaweh Pars, Tatjana I Cornu, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|July 26, 2005
Custom zinc-finger nucleases for use in human cellsStephen Alwin, Maja B Gere, Eva Guhl, et al.Neuromuscular Disorders : NMD|May 18, 2004
A case of childhood Pompe disease demonstrating phenotypic variability of p.Asp645AsnMarian A Kroos, Janbernd Kirschner, Frank N Gellerich, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|November 21, 2007
DNA-binding specificity is a major determinant of the activity and toxicity of zinc-finger nucleasesTatjana I Cornu, Stacey Thibodeau-Beganny, Eva Guhl, et al.Annals of Neurology|December 29, 2004
p.S143F mutation in lamin A/C: a new phenotype combining myopathy and progeriaJanbernd Kirschner, Thomas Brune, Manfred Wehnert, et al.Journal of Neuromuscular Diseases|October 10, 2019
Safety and Treatment Effects of Nusinersen in Longstanding Adult 5q-SMA Type 3 - A Prospective Observational StudyMaggie C Walter, Stephan Wenninger, Simone Thiele, et al.Journal of the Neurological Sciences|February 2, 2010
Late-onset autosomal dominant limb girdle muscular dystrophy and Paget's disease of bone unlinked to the VCP gene locusMichael Kottlors, Olaf Moske-Eick, Angela Huebner, et al.Oncoimmunology|June 13, 2022
CRISPR-Cas9 based gene editing of the immune checkpoint NKG2A enhances NK cell mediated cytotoxicity against multiple myelomaTobias Bexte, Jamal Alzubi, Lisa Marie Reindl, et al.Nucleic Acids Research|March 15, 2018
Designer epigenome modifiers enable robust and sustained gene silencing in clinically relevant human cellsTafadzwa Mlambo, Sandra Nitsch, Markus Hildenbeutel, et al.Pageof 32