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European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society|April 10, 2019
Quality of life of patients with spinal muscular atrophy: A systematic reviewErik Landfeldt, Josefin Edström, Thomas Sejersen, et al.
Methods (San Diego, Calif.)|December 28, 2010
Targeted genome editing in pluripotent stem cells using zinc-finger nucleasesSylwia Bobis-Wozowicz, Anna Osiak, Shamim H Rahman, et al.
Human Gene Therapy|August 16, 2017
Genome and Epigenome Editing to Treat Disorders of the Hematopoietic SystemClaudio Mussolino, Jamal Alzubi, Valentina Pennucci, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 26, 2013
Inactivation of hepatitis B virus replication in cultured cells and in vivo with engineered transcription activator-like effector nucleasesKristie Bloom, Abdullah Ely, Claudio Mussolino, et al.
Methods in Molecular Biology (Clifton, N.J.)|May 13, 2008
Generation and functional analysis of zinc finger nucleasesToni Cathomen, David J Segal, Vincent Brondani, et al.
Biometrical Journal. Biometrische Zeitschrift|December 19, 2024
Investigating a Domain Adaptation Approach for Integrating Different Measurement Instruments in a Longitudinal Clinical RegistryMaren Hackenberg, Michelle Pfaffenlehner, Max Behrens, et al.
FEBS Letters|December 14, 2017
Mitochondrial fatty acid biosynthesis and muscle fiber plasticity in very long-chain acyl-CoA dehydrogenase-deficient miceSara Tucci, Nadja Mingirulli, Zeinab Wehbe, et al.
Human Gene Therapy|June 3, 2011
Zinc-finger nucleases for somatic gene therapy: the next frontierShamim H Rahman, Morgan L Maeder, J Keith Joung, et al.
Trends in Biotechnology|July 19, 2018
The Human Genome Editing Race: Loosening Regulatory Standards for Commercial Advantage?Toni Cathomen, Silke Schüle, Martina Schüßler-Lenz, et al.
Hepatology (Baltimore, Md.)|December 26, 2006
Uridine supplementation antagonizes zalcitabine-induced microvesicular steatohepatitis in miceDirk Lebrecht, Yetlanezi A Vargas-Infante, Bernhard Setzer, et al.
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