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ALS Genetics, Mechanisms, and Therapeutics: Where Are We Now?
Mejzini, Rita; Flynn, Loren L; Pitout, Ianthe L; Fletcher, Sue; Wilton, Steve D; Akkari, P Anthony.
Afiliación
  • Mejzini R; Centre for Molecular Medicine and Innovative Therapeutics, Murdoch University, Perth, WA, Australia.
  • Flynn LL; The Perron Institute for Neurological and Translational Science, Perth, WA, Australia.
  • Pitout IL; Centre for Molecular Medicine and Innovative Therapeutics, Murdoch University, Perth, WA, Australia.
  • Fletcher S; The Perron Institute for Neurological and Translational Science, Perth, WA, Australia.
  • Wilton SD; Centre for Neuromuscular and Neurological Disorders, The University of Western Australia, Perth, WA, Australia.
  • Akkari PA; Centre for Molecular Medicine and Innovative Therapeutics, Murdoch University, Perth, WA, Australia.
Front Neurosci ; 13: 1310, 2019.
Article en En | MEDLINE | ID: mdl-31866818
The scientific landscape surrounding amyotrophic lateral sclerosis (ALS) continues to shift as the number of genes associated with the disease risk and pathogenesis, and the cellular processes involved, continues to grow. Despite decades of intense research and over 50 potentially causative or disease-modifying genes identified, etiology remains unexplained and treatment options remain limited for the majority of ALS patients. Various factors have contributed to the slow progress in understanding and developing therapeutics for this disease. Here, we review the genetic basis of ALS, highlighting factors that have contributed to the elusiveness of genetic heritability. The most commonly mutated ALS-linked genes are reviewed with an emphasis on disease-causing mechanisms. The cellular processes involved in ALS pathogenesis are discussed, with evidence implicating their involvement in ALS summarized. Past and present therapeutic strategies and the benefits and limitations of the model systems available to ALS researchers are discussed with future directions for research that may lead to effective treatment strategies outlined.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Tipo de estudio: Prognostic_studies Idioma: En Revista: Front Neurosci Año: 2019 Tipo del documento: Article País de afiliación: Australia Pais de publicación: Suiza
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Tipo de estudio: Prognostic_studies Idioma: En Revista: Front Neurosci Año: 2019 Tipo del documento: Article País de afiliación: Australia Pais de publicación: Suiza