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American Journal of Medical Genetics. Part A|February 4, 2005
Ullrich congenital muscular dystrophy: connective tissue abnormalities in the skin support overlap with Ehlers-Danlos syndromesJanbernd Kirschner, Ingrid Hausser, Yaqun Zou, et al.European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society|May 31, 2018
CD59 deficiency presenting as polyneuropathy and Moyamoya syndrome with endothelial abnormalities of small brain vesselsChristian Klemann, Janbernd Kirschner, Sandra Ammann, et al.Cells|December 11, 2022
Transcriptomic Landscape and Functional Characterization of Human Induced Pluripotent Stem Cell-Derived Limbal Epithelial Progenitor CellsNaresh Polisetti, Julian Rapp, Paula Liang, et al.European Journal of Neurology|March 23, 2022
Cathepsin D as biomarker in cerebrospinal fluid of nusinersen-treated patients with spinal muscular atrophyDavid C Schorling, Heike Kölbel, Andreas Hentschel, et al.Journal of Inherited Metabolic Disease|January 26, 2018
A mild case of molybdenum cofactor deficiency defines an alternative route of MOCS1 protein maturationSimon Julius Mayr, Jörn Oliver Sass, Julia Vry, et al.Stem Cell Reports|March 29, 2014
Efficient designer nuclease-based homologous recombination enables direct PCR screening for footprintless targeted human pluripotent stem cellsSylvia Merkert, Stephanie Wunderlich, Christien Bednarski, et al.Molecular Therapy Oncolytics|July 31, 2020
PSMA-Directed CAR T Cells Combined with Low-Dose Docetaxel Treatment Induce Tumor Regression in a Prostate Cancer Xenograft ModelJamal Alzubi, Viviane Dettmer-Monaco, Johannes Kuehle, et al.Neuromuscular Disorders : NMD|March 30, 2021
Population pharmacokinetics-based recommendations for a single delayed or missed dose of nusinersenDrew MacCannell, Zdenek Berger, Lilly East, et al.International Journal of Molecular Sciences|April 9, 2020
Targeted Integration of Inducible Caspase-9 in Human iPSCs Allows Efficient in vitro Clearance of iPSCs and iPSC-MacrophagesAlexandra Lipus, Ewa Janosz, Mania Ackermann, et al.Hepatology (Baltimore, Md.)|March 15, 2011
MicroRNA-221 regulates FAS-induced fulminant liver failureAmar Deep Sharma, Nidhi Narain, Eva-Maria Händel, et al.Pageof 32