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The pole test is a common and straightforward test to assess motor coordination in mice. While ataxia might be easy to see in patients, it is not always as apparent in ataxia mouse models. Therefore, this fast and simple test is important for researchers to measure disease severity. It is Read More…
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…
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…
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…
Principal Investigator: Dr. Marija Cvetanovic Location: University of Minnesota, Minneapolis, USA Year Founded: 2012 What disease areas do you research? SCA1 What models and techniques do you use? Mouse models Human stem cells Microscopy RNA Sequencing Research Focus What is your research about? The human brain is made up of many different Read More…
Written by Dr. Ambika Tewari Edited by Dr. Hayley McLoughlin Lentiviral expression of an shRNA against ataxin-3 was well-tolerated and produced no measurable adverse effects in wild-type mice. Evaluating the safety profile is a necessary and crucial step in qualifying a therapy for use in patients. Gene therapy is an Read More…
Location: University of Minnesota, MN, USA Year Research Group Founded: 2008 What disease areas do you research? Ataxia (SCA1, SCA2, SCA3, SCA6, Friedreich Ataxia) Multiple System Atrophy – Cerebellar Ataxia Huntington’s Disease Amyotrophic Lateral Sclerosis Multiple Sclerosis Alzheimer’s disease Parkinson’s disease Traumatic Brain Injury Diabetes What models and techniques do you Read More…
Principal Investigator: Dr. Sheng-Han Kuo Location: Columbia University, New York, NY, United States Year Founded: 2012 What disease areas do you research? SCA1 SCA2 SCA3 SCA6 Tremor Essential Tremor What models and techniques do you use? Mouse models Post-mortem patient tissue Optogenetics Human physiology EEG (electroencephalogram) Neuromodulation Research Focus What is your Read More…
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…
Written by Dr. Hannah Shorrock Edited by Dr. Hayley McLoughlin Nitschke and colleagues identify a microRNA that regulates ataxin-1 levels and rescues motor deficits in a mouse model of SCA1 What if you could use systems already in place in the cell to regulate levels of toxic proteins in disease? Read More…
