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US 2022/0177527 A1Published application
Methods of detecting and treating hereditary spastic paraplegia
AssigneeUniversity of Miami
InventorsStephan L. Zuchner, Mohammad A. Faghihi
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Abstract
The present disclosure relates to methods of detecting and, optionally, treating Hereditary Spastic Paraplegia and other neurological diseases.
Claims10
A method of treating neurological disease in a mammalian subject, the method comprising detecting the presence of a truncating mutation in a Ubiquitin Associated Protein 1 (UBAP1) gene in a biological sample from the subject;
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- administering to the subject a composition that comprises an agent selected from the group consisting of a UBAP1 peptide;
- polynucleotide that encodes a UBAP1 peptide;
- an agent that induces expression of an endogenous nucleotide sequence that encodes a UBAP1 peptide;
- an agent that blocks expression of a mutant UBAP1 gene;
- and an agent that corrects the mutation in UBAP1 gene.
3 more elements
Dependencies are inferred from the claim text.