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Journal of Neurology|May 11, 2024
JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplamClaudia A Chiriboga, Claudio Bruno, Tina Duong, et al.
Neurology|November 2, 2014
A homozygous splice-site mutation in CARS2 is associated with progressive myoclonic epilepsyKerstin Hallmann, Gábor Zsurka, Susanna Moskau-Hartmann, et al.
Biotechnology Journal|October 26, 2020
Preclinical Evaluation of a Novel TALEN Targeting CCR5 Confirms Efficacy and Safety in Conferring Resistance to HIV-1 InfectionMarianna Romito, Alexandre Juillerat, Yik Lim Kok, et al.
Plos Genetics|May 23, 2015
Rescue of DNA-PK Signaling and T-Cell Differentiation by Targeted Genome Editing in a prkdc Deficient iPSC Disease ModelShamim H Rahman, Johannes Kuehle, Christian Reimann, et al.
Scientific Reports|April 26, 2020
TALEN mediated gene editing in a mouse model of Fanconi anemiaMaria José Pino-Barrio, Yari Giménez, Mariela Villanueva, et al.
Annals of Clinical and Translational Neurology|September 11, 2023
Combination disease-modifying treatment in spinal muscular atrophy: A proposed classificationCrystal M Proud, Eugenio Mercuri, Richard S Finkel, et al.
Human Molecular Genetics|January 24, 2018
A novel mechanism causing imbalance of mitochondrial fusion and fission in human myopathiesMarina Bartsakoulia, Angela Pyle, Diego Troncoso-Chandía, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 1, 2024
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropeniaMasoud Nasri, Malte U Ritter, Perihan Mir, et al.
Nature Communications|January 3, 2024
Transient inhibition of 53BP1 increases the frequency of targeted integration in human hematopoietic stem and progenitor cellsRon Baik, M Kyle Cromer, Steve E Glenn, et al.
Neuromuscular Disorders : NMD|November 28, 2020
Long-term follow-up of patients with type 2 and non-ambulant type 3 spinal muscular atrophy (SMA) treated with olesoxime in the OLEOS trialFrancesco Muntoni, Enrico Bertini, Giacomo Comi, et al.
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