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Orphanet Journal of Rare Diseases|October 24, 2013
The TREAT-NMD care and trial site registry: an online registry to facilitate clinical research for neuromuscular diseasesSunil Rodger, Hanns Lochmüller, Adrian Tassoni, et al.
Biochemistry|May 3, 2011
Adding fingers to an engineered zinc finger nuclease can reduce activityYuka Shimizu, Cem Şöllü, Joshua F Meckler, et al.
The Lancet. Neurology|May 30, 2020
Advances in the diagnosis of inherited neuromuscular diseases and implications for therapy developmentRachel Thompson, Sally Spendiff, Andreas Roos, et al.
Biophysical Journal|April 23, 2009
Ultramicroscopy reveals axonal transport impairments in cortical motor neurons at prion diseaseVladimir Ermolayev, Mike Friedrich, Revaz Nozadze, et al.
Molecular Therapy. Nucleic Acids|July 21, 2025
Sustained and specific multiplexed immune checkpoint modulation in CAR T cells induced by targeted epigenome editingMaria Silvia Roman Azcona, Gianni Monaco, Melissa Whitehead, et al.
Human Gene Therapy Methods|September 25, 2013
Histone deacetylase inhibition rescues gene knockout levels achieved with integrase-defective lentiviral vectors encoding zinc-finger nucleasesLaetitia P L Pelascini, Ignazio Maggio, Jin Liu, et al.
Muscle & Nerve|August 23, 2008
Predominant fiber atrophy and fiber type disproportion in early ullrich diseaseJoachim Schessl, Nathalie M Goemans, Alexandra I Magold, et al.
Frontiers in Immunology|May 21, 2021
Natural Killer Cells Generated From Human Induced Pluripotent Stem Cells Mature to CD56brightCD16+NKp80+/-In-Vitro and Express KIR2DL2/DL3 and KIR3DL1Johanna Euchner, Jasmin Sprissler, Toni Cathomen, et al.
EMBO Molecular Medicine|October 24, 2022
Adoptive T cell therapy cures mice from active hemophagocytic lymphohistiocytosis (HLH)Kristoffer Weißert, Sandra Ammann, Tamara Kögl, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 9, 2017
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.
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