Article

Development of novel targeted lentiviral vectors for gene therapy of motor neuron diseases

2013-01-01

Abstract excerpt

Motor Neuron Diseases (MND) including Amyotrophic Lateral Sclerosis (ALS) and Spinal Muscular Atrophy (SMA) are neurodegenerative diseases that can cause progressive paralysis and premature death, while there are no treatments available up to date. Gene therapy using lentiviruses has been successful at alleviating symptoms and extending survival in murine models of both. Restricting transduction to specific cells,...

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Literature Corpus work: 8209afee-cd82-5231-9098-46e4b1ed744d
DOI: 10.25560/27243

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