Preclinical alternative drug discovery programs for monogenic rare diseases. Should small molecules or gene therapy be used? The case of hereditary spastic paraplegias

Abstract excerpt

• We provide guidelines for drug discovery campaigns for rare Hereditary spastic paraplegia (HSP). • Alternative models beyond traditional drug discovery paradigms are required. • Gene therapy and drug repurposing have different application domains in the delivery of targeted therapies. • In silico and specific experimental methods are suggested. • We provide concrete examples of the application of gene therapy...

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