Polyglutamine Disorders

Provides a cutting-edge review of polyglutamine diseases Includes chapters that explore each disorder in depth, as well as gene and cell therapies Shows the involvement of leading global researchers in the field of polyglutamine disorders

Autorentext
Clevio Nobrega Ph.D. and Luis Pereira de Almeida Ph.D.University of Coimbra, Center for Neurosciences and Cell Biology, FMUC, Coimbra, Portugal

Zusammenfassung

Inhalt
Clinical features of Huntington's disease.- Genetic rodent models of Huntington's disease.- Mitochondrial dysfunction in Huntington's disease.- RNA related pathology in Huntington's disease.- Spinal and bulbar muscular atrophy: from clinical genetic features and molecular pathology to mechanisms underlying disease toxicity.- Spinocerebellar Ataxia Type 1: Molecular Mechanisms Of Neurodegeneration And Preclinical Studies.- Spinocerebellar Ataxia, type 6.- Spinocerebellar ataxia type 2.- Molecular mechanisms and therapeutic strategies in Spinocerebellar ataxia type 7.- Spinocerebellar ataxia type 17 (SCA17).- The neuropatholy of spinocerebellar ataxia type 3/Machado-Joseph disease.- Origins and spread of Machado-Joseph disease ancestral mutational events.- Clinical features in Machado-Joseph disease.- Polyglutamine-independent features in ataxin-3 aggregation and pathogenesis of Machado-Joseph disease.- Animal models in Machado-Joseph disease.- Towards the identification of molecular biomarkers of Spinocerebellar ataxia type 3/Machado-Joseph disease.- Planning future clinical trials for Machado Joseph Disease.- Molecular mechanisms and cellular pathways implicated in Machado-Joseph disease pathogenesis.- Pharmacological therapies for Machado-Joseph disease.- Gene therapy for Polyglutamine diseases.- Stem cell-based therapies for Polyglutamine diseases.