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Developing an ON/OFF switch for gene therapy

Written by Dr Hannah K Shorrock Edited by Dr Larissa Nitschke Splice modifying compounds can regulate whether or not gene therapy is active Gene therapy is an incredibly versatile therapeutic strategy that can be used to treat monogenic disorders. Gene therapy can inactivate or silence mutant gene transcripts, edit the 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…

Spotlight: The Cvetanovic Lab

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…

Regulating ataxin-1 expression as a therapeutic avenue for SCA1

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…

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…

Spotlight: The Neuro-D lab Leiden

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…

Spotlight: The Watt Lab

Principal Investigator: Dr. Alanna Watt Location: McGill University, Montreal, Canada Year Founded: 2011 What disease areas do you research? SCA6 ARSACS Aging Basic questions about how the cerebellum functions. What models and techniques do you use? Animal models of ataxia and aging Electrophysiology Imaging RNA sequencing Behavioural assays Research Focus Read More…

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