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 Brenda Toscano Marquez Edited by Marija Cvetanovic Insoluble clumps of mutated ataxin-1 capture essential proteins and trigger the creation of toxic reactive oxygen species. All proteins produced by our cells consist of long chains of amino acids that are coiled and bent into a particular 3D structure. Changes 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 Dr. Marija Cvetanovic Edited by Dr. Ronald Buijsen Researchers from the University of California show they can “edit” the Frataxin gene in human cells from Friedreich’s Ataxia and transplant them into mice. This lays the groundwork for this method to be tested for safety. Friedreich’s ataxia is a Read More…
Escrito por Dr. Marija Cvetanovic, Editado por Dr. Sriram Jayabal, Traduzido para Português por Guilherme Santos, Publicado inicialmente em: 20 de março de 2020. Grupo de pesquisa em Michigan relata a criação do primeiro modelo de célula humana aprovado pelo NIH que reflete as características da doença SCA3 – defeitos Read More…
Written by Dr. Marija Cvetanovic Edited by Dr. Sriram Jayabal Research group in Michigan report the creation of the first NIH-approved human cell model that mirrors SCA3 disease features – cellular defects that, after gene therapy, show improvement Spinocerebellar ataxia type 3 (SCA3) is a dominantly-inherited, late onset genetic disease Read More…
Embryonic and adult stem cells Stem cells are cells that provide new cells during growth, and replace cells that are damaged or lost during life. They have the following two important properties that enable them to do this: The ability to develop (differentiate) into many other, different cell types, for Read More…
Written by Dr. Vitaliy V Bondar Edited by Dr. Chandrakanth Edamakanti Researchers for the first time identified that spinocerebellar ataxia type 1 (SCA1) may have roots in early cerebellar circuit malfunction. Since the discovery of the cause of SCA1, researchers have wondered: why does it take three to four decades Read More…
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