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National Ataxia Foundation

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Drug Discovery Research

Identifying serotonin receptors as a therapeutic target for SCA3

Written by Dr. Hannah K Shorrock Edited by Dr. Hayley McLoughlin A C. elegans model of SCA3 helps to identify the serotonin receptor agonist befiradol as a potential therapeutic for SCA3 To move toward treatments for ataxia patients, it is important to understand what cellular and molecular pathways are dysfunctional Read More…

Snapshot: What is N-acetylcysteine?

What is N-acetylcysteine used for? Cysteine is an amino acid that is used as a building block in our bodies to make proteins. We consume cysteine in our diets through protein-rich foods, like beef or lentils. N-acetylcysteine is a chemical derivative of cysteine. This means which means that N-acetylcysteine contains Read More…

Identificación de moléculas aprobadas por la FDA para tratar SCA6

Escrito por la Dra. Hannah Shorrock Editado por la Dra. Larissa Nitschke. Publicado inicialmente en el 7 de mayo de 2021. Traducción al español fueron hechas por FEDAES y Carlos Barba. Pastor y sus colegas identifican pequeñas moléculas aprobadas por la FDA que reducen selectivamente la proteína tóxica expandida con poliglutamina Read More…

Terapia génica lentiviral en ratones SCA3: Seguridad a largo plazo

Escrito por la Dra. Ambika Tewari Editado por la Dra. Hayley McLoughlin. Publicado inicialmente en el 6 de agosto de 2021. Traducción al español fueron hechas por FEDAES. La expresión lentiviral de un ARNhc contra ataxina-3 fue bien tolerada y no produjo efectos adversos medibles en ratones de tipo salvaje. La Read More…

“Expanding” the therapeutic promise for SCA1

Written by Dr. Judit M Perez Ortiz Edited by Dr. Maria do Carmo Costa A druggable target in Spinocerebellar Ataxia type 1 (SCA1) shows promise in treating cerebellar and non-cerebellar aspects of disease. Spinocerebellar Ataxia type 1 (SCA1) is a neurodegenerative disease that typically starts with coordination difficulties (ataxia) in Read More…

Spotlight: The Zoghbi Lab

Principal Investigator: Dr. Huda Zoghbi Location: Baylor College of Medicine and Texas Children’s Hospital, Houston, TX, USA Year Founded:  1988 What disease areas do you research? SCA1 Rett Syndrome MeCP2 duplication syndrome Alzheimer’s disease Parkinson’s disease Brain development What models and techniques do you use? Mouse models Human cell biology Molecular biology Read More…

Identifying FDA-approved molecules to treat SCA6

Written by Dr Hannah Shorrock Edited by Dr. Larissa Nitschke Pastor and colleagues identify FDA-approved small molecules that selectively reduce the toxic polyglutamine-expanded protein in SCA6. Selectively targeting disease-causing genes without disrupting cellular functions is essential for successful therapy development. In spinocerebellar ataxia type 6 (SCA6), achieving this selectivity is Read More…

El BDNF puede revertir la ataxia en ratones SCA1

Escrito por Anna Cook Editado por Dr. David Bushart. Publicado inicialmente en el 19 de Marzo de 2021. Traducción al español fueron hechas por FEDAES y Carlos Barba. El factor neurotrófico derivado del cerebro -BDNF- puede prevenir la ataxia en ratones SCA1. Una nueva investigación muestra que el tratamiento funciona Read More…

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