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Frontiers in Medicine|January 26, 2023
Clinical genome editing to treat sickle cell disease-A brief updateParinaz Zarghamian, Julia Klermund, Toni CathomenCurrent Opinion in Pharmacology|September 7, 2015
Proven and novel strategies for efficient editing of the human genomeClaudio Mussolino, Tafadzwa Mlambo, Toni CathomenEuropean Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society|December 7, 2017
Single-center experience with intrathecal administration of Nusinersen in children with spinal muscular atrophy type 1Astrid Pechmann, Thorsten Langer, Sabine Wider, et al.Orphanet Journal of Rare Diseases|July 31, 2020
Experiences of caregivers of children with spinal muscular atrophy participating in the expanded access program for nusinersen: a longitudinal qualitative studyPetra Kiefer, Janbernd Kirschner, Astrid Pechmann, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|November 13, 2008
Expanding or restricting the target site repertoire of zinc-finger nucleases: the inter-domain linker as a major determinant of target site selectivityEva-Maria Händel, Stephen Alwin, Toni CathomenNature Medicine|April 8, 2017
Refining strategies to translate genome editing to the clinicTatjana I Cornu, Claudio Mussolino, Toni CathomenHamostaseologie|January 11, 2017
Therapeutic genome editing with engineered nucleasesSimone A Haas, Viviane Dettmer, Toni CathomenNature Communications|August 5, 2025
The hidden risks of CRISPR/Cas: structural variations and genome integrityClotilde Aussel, Toni Cathomen, Carla Fuster-GarcíaPediatrics|July 5, 2005
Intravenously administered immunoglobulin in the treatment of childhood Guillain-Barré syndrome: a randomized trialRudolf Korinthenberg, Joachim Schessl, Janbernd Kirschner, et al.Joint Bone Spine|June 16, 2009
Muscle hypertrophy of the lower leg caused by L5 radiculopathyMichael Kottlors, Klaus Mueller, Janbernd Kirschner, et al.Pageof 32