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Drug Discovery Research

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…

El BDNF puede revertir la ataxia en ratones SCA1

Escrito por Anna Cook Editado por Dr. David Bushart. Publicado inicialmente en el 19 de Marzo de 2021. Traducción al español fueron hechas por FEDAES y Carlos Barba. El factor neurotrófico derivado del cerebro -BDNF- puede prevenir la ataxia en ratones SCA1. Una nueva investigación muestra que el tratamiento funciona Read More…

BDNF can reverse ataxia in SCA1 mice, even after symptom onset

Written by Anna Cook Edited by Dr. David Bushart Brain-derived neurotrophic factor can prevent ataxia in SCA1 mice. New research shows that the treatment works even if it’s started after mice develop signs of ataxia. SCA1 is a neurodegenerative disease caused by a mutation in the Ataxin1 gene. People with Read More…

Clearing aggregated ataxin-2 protein as a therapeutic avenue for SCA2

Written by Dr. Vitaliy Bondar Edited by Dr. Hayley McLoughlin New research suggests that mutant ataxin-2 protein overwhelms cells in SCA2, leading to decreased autophagy and clearance of damaged proteins. Many comparisons can be made between cells and human beings. Just like humans, cells can accumulate junk and waste at Read More…

2 minuti di Scienza: Cosa sono I nucleotidi antisenso?

I nucleotidi anti-senso (anche noti come ASOs o AON, dall’inglese Antisense oligonucleotides) sono piccole molecole che possono essere usate per prevenire o alterare la produzione di proteine. Le proteine sono la forza lavoro della cellula, e dirigono la maggior parte dei processi cellulari. Le proteine sono prodotte in due fasi: Read More…

A new molecule identified that controls cerebellar communication

Written by Dr. Ambika Tewari Edited by Dr. Sriram Jayabal Targeting phosphatases in the cerebellum can correct miscommunication in multiple models of ataxia. The cerebellum is essential for motor coordination and consists of the coordinated activity of different types of cells. Purkinje cells are one of the most fascinating cell 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…

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