MicroRNA-Directed Neuronal Reprogramming as a Therapeutic Strategy for Neurological Diseases.
Therapeutic Strategies Under Development Targeting Inflammatory Mechanisms in Amyotrophic Lateral Sclerosis.
MicroRNA Metabolism and Dysregulation in Amyotrophic Lateral Sclerosis.
Genome-wide RNA-seq of iPSC-derived motor neurons indicates selective cytoskeletal perturbation in Brown-Vialetto disease that is partially rescued by riboflavin.
iPSC-derived LewisX+CXCR4+β1-integrin+ neural stem cells improve the amyotrophic lateral sclerosis phenotype by preserving motor neurons and muscle innervation in human and rodent models.
Development of Therapeutics for C9ORF72 ALS/FTD-Related Disorders.
Differential neuronal vulnerability identifies IGF-2 as a protective factor in ALS.
Experimental Advances Towards Neural Regeneration from Induced Stem Cells to Direct In Vivo Reprogramming.
Autophagy in motor neuron disease: Key pathogenetic mechanisms and therapeutic targets.
Morpholino-mediated SOD1 reduction ameliorates an amyotrophic lateral sclerosis disease phenotype.
Clinical and molecular features and therapeutic perspectives of spinal muscular atrophy with respiratory distress type 1.
MFN2-related neuropathies: Clinical features, molecular pathogenesis and therapeutic perspectives.
SOD1 misplacing and mitochondrial dysfunction in amyotrophic lateral sclerosis pathogenesis.
Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches.
Human pluripotent stem cells as tools for neurodegenerative and neurodevelopmental disease modeling and drug discovery.
Spinal muscular atrophy–recent therapeutic advances for an old challenge.
Motor neurons with differential vulnerability to degeneration show distinct protein signatures in health and ALS.
Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model.
Therapeutic development in amyotrophic lateral sclerosis.
Pluripotent stem cell-based models of spinal muscular atrophy
Therapeutic applications of the cell-penetrating HIV-1 Tat peptide
Antisense oligonucleotide therapy for the treatment of C9ORF72 ALS/FTD diseases
The wide spectrum of clinical phenotypes of spinal muscular atrophy with respiratory distress type 1: a systematic review
iPSC-Based Models to Unravel Key Pathogenetic Processes Underlying Motor Neuron Disease Development
Stem cell transplantation for amyotrophic lateral sclerosis: therapeutic potential and perspectives on clinical translation.
iPSC-Derived neural stem cells act via kinase inhibition to exert neuroprotective effects in spinal muscular atrophy with respiratory distress type 1.
Motor neuron derivation from human embryonic and induced pluripotent stem cells: experimental approaches and clinical perspectives.
In vitro neurogenesis: development and functional implications of iPSC technology.
Induced neural stem cells: methods of reprogramming and potential therapeutic applications.
Cellular therapy to target neuroinflammation in amyotrophic lateral sclerosis.
Effect of combined systemic and local morpholino treatment on the spinal muscular atrophy Δ7 mouse model phenotype.
Molecular therapeutic strategies for spinal muscular atrophies: current and future clinical trials.