Bill Nye is back in his lab with NAF and Biogen—breaking down the science of Friedreich Ataxia in a new video series. WATCH NOW
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…
Written by Dr. Ambika Tewari Edited by Dr. Maria do Carmo Costa Mutations in Ataxin-3 protein prevent the normal functioning of a DNA repair enzyme leading to an accumulation of errors Cells are bombarded by thousands of DNA damaging events each day from internal and external sources. Internal sources include Read More…
Written By Dr. David Bushart Edited by Celeste Suart The RISCA study will help researchers design smarter, more efficient clinical trials by teaching us about the very early stages of SCA Ataxia research has grown significantly in recent years. Although much work still remains, we are gaining a better understanding Read More…
Written by Dr. Ambika Tewari Edited by Dr. Gulin Oz Neurofilament light chain could provide a reliable readout of how far an SCA3 patient’s disease has progressed How often have you heard that the most effective way to treat a disorder is early intervention? In reality, “early” is not possible Read More…
Principal Investigator: Dr. Willeke van Roon-Mom Location: Leiden University Medical Centre, Leiden, The Netherlands Year Founded: 1995 What disease areas do you research? SCA1 SCA3 Huntington’s Disease Dutch – Cerebral Amyloid Angiopathy Alzheimer Disease What models and techniques do you use? Mouse Models hiPSC-derived stem cells models Post-mortem patient tissue Read More…
Written by Ambika Tewari Edited by Brenda Toscano Márquez Trehalose, a natural autophagy inducer shows promise as a therapeutic candidate for MJD/SCA3 Every cell has an elaborate set of surveillance mechanisms to ensure optimal functioning. As proteins are synthesized, errors can occur leading to misfolded proteins. These abnormal proteins can Read More…
Ataxias can occur due to a multitude of reasons. One way a patient might acquire ataxia is from an accident or an injury – not as a result of genetics. On the other hand, a patient could also inherit a specific mutation (a genetic defect, in other words) from one Read More…
Written by Dr. Maria do Carmo Costa, Edited by Dr. Hayley McLoughlin Collaboration between researchers in Portugal and the United Kingdom discover that a canola oil by-product shows promise, corrects MJD/SCA3 and Parkinson’s Disease symptoms in animal models. Isolated compounds or extracts (containing a mixture of compounds) from certain plants are Read More…
