Bill Nye the Science Guy is speaking at the 2024 Annual Ataxia Conference! Register now. LEARN MORE!
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…
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…
Escrito por el Dr. Vitaliy Bondar Editado por el Dr. Hayley McLoughlin. Publicado inicialmente en el 5 de febrero de 2021. Traducción al español fueron hechas por FEDAES y Carlos Barba. Una nueva investigación sugiere que la proteína ataxina-2 mutante abruma a las células en SCA2, lo que lleva a una Read More…
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…
Escrito por el Dr. Ronald Buijsen Editado por la Dra. Larissa Nitschke. Publicado inicialmente en el 28 de Enero de 2021. Traducción al español fueron hechas por FEDAES y Carlos Barba. O’Callaghan y sus colegas muestran que los enfoques terapéuticos novedosos para reducir la proteína que causa la enfermedad en Read More…
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…
Written by Dr. Ronald Buijsen Edited by Dr. Larissa Nitschke O’Callaghan and colleagues show that novel therapeutic approaches to reduce the disease-causing protein in SCA1 do not increase the risk of developing cancer or Alzheimer’s disease in SCA1 mice. People affected with Spinocerebellar Ataxia type 1 or SCA1 carry an Read More…
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…
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…
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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