SCA1

Chr 4AD

caspase 3

Also known as: CPP32, CPP32B, SCA-1

The protein encoded by this gene is a cysteine-aspartic acid protease that plays a central role in the execution-phase of cell apoptosis. The encoded protein cleaves and inactivates poly(ADP-ribose) polymerase while it cleaves and activates sterol regulatory element binding proteins as well as caspases 6, 7, and 9. This protein itself is processed by caspases 8, 9, and 10. It is the predominant caspase involved in the cleavage of amyloid-beta 4A precursor protein, which is associated with neuronal death in Alzheimer's disease. [provided by RefSeq, Aug 2017]

Primary Disease Associations & Inheritance

Spinocerebellar ataxia 1MIM #164400
AD
0
ClinVar variants
0
Pathogenic / LP
pLI score
6
Active trials
Clinical SummarySCA1
💊
Clinical Trials
6 active or recruiting trials — potential therapeutic options may be available
Some data sources returned errors (2)

ensembl: Error: Ensembl fetch failed: 400 for https://rest.ensembl.org/lookup/symbol/homo_sapiens/SCA1?content-type=application/json&expand=1

gnomad: Error: Gene not found

Population Genetics & Constraint

Constraint data not available from gnomAD.

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

SCA1 · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

OMIM — Genotype-Phenotype Relationships

1 OMIM entry

Spinocerebellar ataxia 1

MIM #164400

Molecular basis of disorder known

Autosomal dominant
Clinical Literature
Landmark / reviewRecent case evidence
Key Publications
Landmark & review papers · by relevance
PubMed
The hereditary ataxias.
Koeppen AH·J Neuropathol Exp Neurol
1998Review
Spinocerebellar degenerations.
Perlman SL·Handb Clin Neurol
2011Review
Hereditary Ataxias in Argentina.
Rossi M et al.·Cerebellum
2025Review
B cells in inflammatory bowel disease.
Castillo FA et al.·Immunol Lett
2026Review
Pericytes in Bone Marrow.
Kunisaki Y·Adv Exp Med Biol
2019Review
The electrophysiology of spinocerebellar ataxias.
Liang L et al.·Neurophysiol Clin
2016Review
Toward cell specificity in SCA1.
Humbert S et al.·Neuron
2002Review
Top 10 resultsSearch PubMed ↗

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Lung DiseasesCarcinoma, Non-Small-Cell LungResectable Lung Non-Small Cell Carcinoma

Study of Treatment With Sacituzumab and Zimberelimab for Patients With Lung Cancer Confined to the Chest and Previously Operated on Who Were Not Disease-free.

RECRUITING
NCT06431633Phase PHASE3Fundación GECPStarted 2025-02-04
ZimberelimabSacituzumab govitecanCisplatin
Time Restricted EatingObesityWeight Loss

Time-restricted Eating Versus Daily Continuous Calorie Restriction on Body Weight and Colorectal Cancer Risk Markers

RECRUITING
NCT05114798Phase NAUniversity of Illinois at ChicagoStarted 2022-05-17
Time restricted eatingCalorie Restriction
Colorectal CarcinomaUlcerative Colitis

Testing Atorvastatin to Lower Colon Cancer Risk in Longstanding Ulcerative Colitis

ACTIVE NOT RECRUITING
NCT04767984Phase PHASE2Northwestern UniversityStarted 2021-09-24
Atorvastatin CalciumBiopsy of ColonBiospecimen Collection
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

RECRUITING
NCT01793168Sanford HealthStarted 2010-07
Advanced Malignant Solid NeoplasmAnatomic Stage III Breast Cancer AJCC v8Anatomic Stage IIIA Breast Cancer AJCC v8

ALRN-6924 and Paclitaxel in Treating Patients With Advanced, Metastatic, or Unresectable Solid Tumors

ACTIVE NOT RECRUITING
NCT03725436Phase PHASE1M.D. Anderson Cancer CenterStarted 2019-01-24
MDM2/MDMX Inhibitor ALRN-6924Paclitaxel
High-Risk CancerLocally Advanced Breast Cancer

Three Fraction Radiation to Induce Immuno-Oncologic Response

ACTIVE NOT RECRUITING
NCT03978663Phase NALondon Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph'sStarted 2020-09-02
Neoadjuvant radiotherapy