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Therapeutic Strategies for Mutant SPAST-Based Hereditary Spastic Paraplegia
Journal article   Open access   Peer reviewed

Therapeutic Strategies for Mutant SPAST-Based Hereditary Spastic Paraplegia

Neha Mohan, Liang Qiang, Gerardo Morfini and Peter W. Baas
Brain sciences, v 11(8), p1081
01 Aug 2021
PMID: 34439700
url
https://doi.org/10.3390/brainsci11081081View
Published, Version of Record (VoR)CC BY V4.0 Open

Abstract

Life Sciences & Biomedicine Neurosciences Neurosciences & Neurology Science & Technology
Mutations of the SPAST gene that encodes the microtubule-severing enzyme called spastin are the chief cause of Hereditary Spastic Paraplegia. Growing evidence indicates that pathogenic mutations functionally compromise the spastin protein and endow it with toxic gain-of-function properties. With each of these two factors potentially relevant to disease etiology, the present article discusses possible therapeutic strategies that may ameliorate symptoms in patients suffering from SPAST-based Hereditary Spastic Paraplegia, which is usually termed SPG4-HSP.

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Collaboration types
Domestic collaboration
Web of Science research areas
Neurosciences
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