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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…

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