El uso de oligonucleótidos antisentido dirigidos hacia la ataxina-3 mutante puede mejorar la disfunción de las neuronas de Purkinje

July 25, 2025

Escrito por Asmer Aliyeva Editado por Dr. Hannah K Shorrock Traducido por BS Ismael Araujo-Aliaga El tratamiento con oligonucleótidos antisentido (ASO) mejora la función de las neuronas de Purkinje regulada por canales de potasio y resalta la conectividad cerebral alterada en el trastorno del movimiento en SCA3. Comprender qué tipos de células Read More…

“Where did you come from, where did you go?”: Measuring disease progression in SCA1, 2, 3, and 6

December 13, 2024

Written by Alexandra Putka Edited by Dr Hannah K Shorrock Scientists characterize SCA1, 2, 3, & 6 disease progression, finding that symptoms change most rapidly in the first 10 years of the disease. Designing clinical trials for rare diseases such as spinocerebellar ataxias (SCAs) requires us to know the timeline Read More…

Unveiling Peripheral Nerve Abnormalities in Spinocerebellar Ataxia Type 3

October 18, 2024

Written by Juan MatoEdited by Hayley McLoughlin, PhD German research team uses specialized imaging technology to measure neuropathy in SCA3 patients. The Unexplored Depths of Peripheral Neuropathy in SCA3 Spinocerebellar Ataxia Type 3 (SCA3) is an inherited neurodegenerative disease that affects balance, coordination, speech, and overall mobility. However, did you Read More…

Preparação para testes em SCA1 and SCA3 com exames de imagem

August 9, 2024

Escrito por Dr. Gülin ÖzEditado por Dr. Celeste SuartTraduzido por Ana Carolina Martins A ressonância magnética (RM) detecta alterações cerebrais antes dos sintomas de ataxia. A lista de empresas farmacêuticas que direcionaram a sua atenção para as ataxias tem aumentado ao longo da última década. Isto se deve ao promissor Read More…

Uncovering the Impact of Toxic RAN Proteins Produced by CAG Repeat Expansions

July 26, 2024

Written by Tala Ortiz Edited by Dr. Larissa Nitschke Repeat-associated non-AUG (RAN) translation in CAG repeat expansion diseases is toxic to cells and causes them to die. The Jain group found that RAN proteins and expanded RNA can be found in the same location in cells and interfere with essential Read More…

Targeting Mutant Ataxin-3 with Antisense Oligonucleotides can Improve Purkinje Neuron Dysfunction

June 28, 2024

Written by Asmer Aliyeva Edited by Dr. Hannah K Shorrock Antisense Oligonucleotide (ASO) treatment improves Purkinje neuron function regulated by potassium channels and highlights altered brain connectivity in movement disruption in SCA3. Understanding which cell types are important for disease onset is vital for therapy development and treatment options for Read More…

Walking toward a better understanding of SCA3 progression

May 17, 2024

Written by Alexandra Putka Edited by Dr. Hayley S. McLoughlin Walking measurements detect changes in SCA3 severity across a 1-year study and represent an important biomarker of disease progression Medical doctors use a number of tools to assess a patient’s health: a thermometer and blood pressure monitor, for example. These Read More…

Desarrollo de biomarcadores terapéuticamente relevantes para SCA3

February 9, 2024

Escrito por el Dr. Hannah K. Shorrock Editado por el Dr. Vitaliy V. Bondar La medición de los niveles de las proteínas de poliQ ATXN3 en el líquido cefalorraquídeo y plasma distingue a pacientes SCA3 de los individuos no afectados Para que los medicamentos o terapias modificadores de una enfermedad Read More…

Snapshot: What are oligodendrocytes?

November 3, 2023

Oligodendrocytes are a type of brain cell that play a crucial role in insulating neurons to facilitate efficient transmission of electrical signals. Similar to the rubber coating on cords, oligodendrocytes wrap around axons with a cholesterol-rich myelin structure, which speeds up the transmission of electrical impulses. Each oligodendrocyte can myelinate Read More…

Getting ready for trials in SCA1 and SCA3 with imaging

September 22, 2023

Written by Dr. Gülin ÖzEdited by Dr. Celeste Suart Advanced Magnetic Resonance Imaging (MRI) detects brain changes before ataxia symptoms. The list of pharmaceutical companies that turned their attention to ataxias has been steadily increasing over the last decade. This is in large part thanks to exciting developments in gene Read More…

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