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Journal of Neurology, Neurosurgery, and Psychiatry|November 1, 2011
Adult-onset cerebellar ataxia due to mutations in CABC1/ADCK3Rita Horvath, Birgit Czermin, Sweena Gulati, et al.
Plos Currents|January 19, 2013
Guidance in social and ethical issues related to clinical, diagnostic care and novel therapies for hereditary neuromuscular rare diseases: "translating" the translationalPauline McCormack, Simon Woods, Annemieke Aartsma-Rus, et al.
Molecular Therapy. Methods & Clinical Development|December 24, 2025
Targeted inhibition of ELANE expression using adenine base editing to treat severe congenital neutropeniaBetül Findik, Benjamin Dannenmann, Franka Bernhard, et al.
Journal of Neuromuscular Diseases|February 11, 2020
RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study DesignRichard S Finkel, John W Day, Darryl C De Vivo, et al.
The Lancet. Neurology|August 31, 2010
Treatment of Duchenne muscular dystrophy with ciclosporin A: a randomised, double-blind, placebo-controlled multicentre trialJanbernd Kirschner, Joachim Schessl, Ulrike Schara, et al.
The Lancet. Neurology|May 3, 2017
Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trialEnrico Bertini, Eric Dessaud, Eugenio Mercuri, et al.
Molecular & Cellular Proteomics : MCP|November 2, 2012
A combined laser microdissection and mass spectrometry approach reveals new disease relevant proteins accumulating in aggregates of filaminopathy patientsRudolf A Kley, Alexandra Maerkens, Yvonne Leber, et al.
Small (Weinheim an Der Bergstrasse, Germany)|December 17, 2019
Biodegradable Nanocarriers Resembling Extracellular Vesicles Deliver Genetic Material with the Highest Efficiency to Various Cell TypesYana Tarakanchikova, Jamal Alzubi, Valentina Pennucci, et al.
International Journal of Molecular Sciences|January 23, 2024
CRISPR-Cas12a for Highly Efficient and Marker-Free Targeted Integration in Human Pluripotent Stem CellsRuba Hammad, Jamal Alzubi, Manuel Rhiel, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|May 16, 2024
Engineering of potent CAR NK cells using non-viral Sleeping Beauty transposition from minimalistic DNA vectorsTobias Bexte, Lacramioara Botezatu, Csaba Miskey, et al.
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