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Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS

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Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS

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The role of the NF-kappaB pathway in Amyotrophic lateral sclerosis pathogenesis

Core-shell Structured Chitosan-polyethylenimine Nanoparticles for Gene Delivery: Improved Stability, Cellular Uptake, and Transfection Efficiency

Astrocyte-targeting RNA interference against mutated superoxide dismutase 1 induces motoneuron plasticity and protects fast-fatigable motor units in a mouse model of amyotrophic lateral sclerosis

Efficient viral transduction in mouse inner ear hair cells with utricle injection and AAV9-PHP.B

Blood Flow to the Spleen is Altered in a Mouse Model of Spinal Muscular Atrophy

AAV based gene therapy for rare diseases

Machine learning-based tools to model and to remove the off-target effect

Intracerebroventricular Injection of Adeno-Associated Virus 6 and 9 Vectors for Cell Type-Specific Transgene Expression in the Spinal Cord

MAR Elements and Transposons for Improved Transgene Integration and Expression

MAR-mediated integration of plasmid vectors for in vivo gene transfer and regulation