| Literature DB >> 29423566 |
Francesco Mari1, Beatrice Berti1, Alessandro Romano2, Jacopo Baldacci3, Riccardo Rizzi1, M Grazia Alessandrì3, Alessandra Tessa3, Elena Procopio1, Anna Rubegni3, Charles Marques Lourenḉo4, Alessandro Simonati5, Renzo Guerrini6,7, Filippo Maria Santorelli8.
Abstract
Spastic paraplegia 35 (SPG35) is a recessive condition characterized by childhood onset, progressive course, complicated by dystonia, dysarthria, cognitive impairment, and epilepsy. Mutations in the FA2H gene have been described in several families, leading to the proposal of a single entity, named fatty acid hydrolase-associated neurodegeneration (FAHN). Several reports have described a polymorphic radiological picture with white matter lesions of various degrees and a distinct form of neurodegeneration with brain iron accumulation. While we reviewed the pertinent literature, we also report three new patients with SPG35, highlighting the possible absence of white matter lesions even after a long neuroimaging follow-up. Three-dimensional modeling of the mutated proteins was helpful to elucidate the role of the site of mutations and the correlation with the residual enzyme activity as determined in cultured skin fibroblasts.Entities:
Keywords: Complicated hereditary spastic paraplegia; FA2H; SPG35
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Year: 2018 PMID: 29423566 DOI: 10.1007/s10048-018-0538-8
Source DB: PubMed Journal: Neurogenetics ISSN: 1364-6745 Impact factor: 2.660