Cargando…

Therapeutic Strategies for Spinocerebellar Ataxia Type 1

Spinocerebellar ataxia type 1 (SCA1) is an autosomal dominant neurodegenerative disorder that affects one or two individuals per 100,000. The disease is caused by an extended CAG repeat in exon 8 of the ATXN1 gene and is characterized mostly by a profound loss of cerebellar Purkinje cells, leading t...

Descripción completa

Detalles Bibliográficos
Autores principales:	Kerkhof, Laurie M. C., van de Warrenburg, Bart P. C., van Roon-Mom, Willeke M. C., Buijsen, Ronald A. M.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2023
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10216181/ https://www.ncbi.nlm.nih.gov/pubmed/37238658 http://dx.doi.org/10.3390/biom13050788

Ejemplares similares

Human Induced Pluripotent Stem Cell-Based Modelling of Spinocerebellar Ataxias
por: Hommersom, Marina P., et al.
Publicado: (2021)

Genetics, Mechanisms, and Therapeutic Progress in Polyglutamine Spinocerebellar Ataxias
por: Buijsen, Ronald A.M., et al.
Publicado: (2019)

Ataxin-3 Protein and RNA Toxicity in Spinocerebellar Ataxia Type 3: Current Insights and Emerging Therapeutic Strategies
por: Evers, Melvin M., et al.
Publicado: (2013)

The potential value of disease-modifying therapy in patients with spinocerebellar ataxia type 1: an early health economic modeling study
por: van Prooije, Teije, et al.
Publicado: (2023)

Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice
por: Toonen, Lodewijk J.A., et al.
Publicado: (2017)

Correction to: The potential value of disease-modifying therapy in patients with spinocerebellar ataxia type 1: an early health economic modeling study
por: van Prooije, Teije, et al.
Publicado: (2023)

Frequency of Spinocerebellar Ataxia type 1, 2, 3,6 and 7 and clinical profile of Spinocerebellar Ataxia type 3 in Malaysia
por: Mohamed Ibrahim, Norlinah, et al.
Publicado: (2020)

Transcriptional profiling and biomarker identification reveal tissue specific effects of expanded ataxin-3 in a spinocerebellar ataxia type 3 mouse model
por: Toonen, Lodewijk J. A., et al.
Publicado: (2018)

Discordance Between Patient-Reported Outcomes and Physician-Rated Motor Symptom Severity in Early-to-Middle-Stage Spinocerebellar Ataxia Type 3
por: Maas, Roderick P. P. W. M., et al.
Publicado: (2021)

Antisense Oligonucleotide-Induced Amyloid Precursor Protein Splicing Modulation as a Therapeutic Approach for Dutch-Type Cerebral Amyloid Angiopathy
por: Daoutsali, Elena, et al.
Publicado: (2021)

Cerebellar Transcranial Direct Current Stimulation in Spinocerebellar Ataxia Type 3: a Randomized, Double-Blind, Sham-Controlled Trial
por: Maas, Roderick P. P. W. M., et al.
Publicado: (2022)

Stage-dependent biomarker changes in spinocerebellar ataxia type 3
por: Faber, Jennifer, et al.
Publicado: (2023)

Cell-based therapeutic strategies for treatment of spinocerebellar ataxias: an update
por: Correia, Joana Sofia, et al.
Publicado: (2022)

Amyloid beta accumulations and enhanced neuronal differentiation in cerebral organoids of Dutch-type cerebral amyloid angiopathy patients
por: Daoutsali, Elena, et al.
Publicado: (2023)

Prediction of the age at onset in spinocerebellar ataxia type 1, 2, 3 and 6
por: Tezenas du Montcel, Sophie, et al.
Publicado: (2014)

Regional Brain and Spinal Cord Volume Loss in Spinocerebellar Ataxia Type 3
por: Faber, Jennifer, et al.
Publicado: (2021)

The cerebral metabolic topography of spinocerebellar ataxia type 3
por: Meles, Sanne K., et al.
Publicado: (2018)

The MID1 Protein: A Promising Therapeutic Target in Huntington’s Disease
por: Heinz, Annika, et al.
Publicado: (2021)

Writer's cramp in spinocerebellar ataxia Type 1
por: Khwaja, Geeta Anjum, et al.
Publicado: (2016)

Autophagy in Spinocerebellar Ataxia Type 3: From Pathogenesis to Therapeutics
por: Paulino, Rodrigo, et al.
Publicado: (2023)

Sensorimotor processing for balance in spinocerebellar ataxia type 6
por: Bunn, Lisa M., et al.
Publicado: (2015)

Cerebellar transcranial direct current stimulation in spinocerebellar ataxia type 3 (SCA3-tDCS): rationale and protocol of a randomized, double-blind, sham-controlled study
por: Maas, Roderick P. P. W. M., et al.
Publicado: (2019)

Spinocerebellar Ataxia Type 31 with Blepharospasm
por: Itaya, Sakiko, et al.
Publicado: (2018)

Therapeutic Misestimation in Patients with Degenerative Ataxia: Lessons from a Randomized Controlled Trial
por: Maas, Roderick P.P.W.M., et al.
Publicado: (2022)

Correction to: Cerebellar transcranial direct current stimulation in spinocerebellar ataxia type 3 (SCA3-tDCS): rationale and protocol of a randomized, double-blind, sham-controlled study
por: Maas, Roderick P. P. W. M., et al.
Publicado: (2021)

Antisense oligonucleotide therapy for spinocerebellar ataxia type 2
por: Scoles, Daniel R., et al.
Publicado: (2017)

The Molecular Basis of Spinocerebellar Ataxia Type 7
por: Goswami, Rituparna, et al.
Publicado: (2022)

Neurofilaments in spinocerebellar ataxia type 3: blood biomarkers at the preataxic and ataxic stage in humans and mice
por: Wilke, Carlo, et al.
Publicado: (2020)

Postural Tremor and Ataxia Progression in Spinocerebellar Ataxias
por: Gan, Shi-Rui, et al.
Publicado: (2017)

An understanding of spinocerebellar ataxia
por: Ramachandra, N.B., et al.
Publicado: (2015)

Parkinsonism in Spinocerebellar Ataxia
por: Park, Hyeyoung, et al.
Publicado: (2015)

Spinocerebellar ataxia: an update
por: Sullivan, Roisin, et al.
Publicado: (2018)

The Multiple Faces of Spinocerebellar Ataxia type 2
por: Antenora, Antonella, et al.
Publicado: (2017)

Axonal inclusions in spinocerebellar ataxia type 3
por: Seidel, Kay, et al.
Publicado: (2010)

Deletion at ITPR1 Underlies Ataxia in Mice and Spinocerebellar Ataxia 15 in Humans
por: van de Leemput, Joyce, et al.
Publicado: (2007)

Neurochemical Differences in Spinocerebellar Ataxia Type 14 and 1
por: Grosch, Anne Sophie, et al.
Publicado: (2020)

Spinocerebellar ataxia type 7: Report of an Indian family
por: Wali, Gurusidheshwar M.
Publicado: (2013)

Reply: A homozygous GDAP2 loss-of-function variant in a patient with adult-onset cerebellar ataxia; and Novel GDAP2 pathogenic variants cause autosomal recessive spinocerebellar ataxia-27 (SCAR27) in a Chinese family
por: Eidhof, Ilse, et al.
Publicado: (2020)

Spinocerebellar ataxia type 6 and episodic ataxia type 2 in a Korean family.
por: Koh, S. H., et al.
Publicado: (2001)

Spinocerebellar ataxia type 10 in Chinese Han
por: Wang, Kang, et al.
Publicado: (2015)

Cannot write session to /tmp/vufind_sessions/sess_c5it876gdadfd9n5jtppb4a3m3