Disease Models

Alzheimer’s disease (AD) is a neurodegenerative condition that is strongly linked with age and accounts for 60-70% of all cases of dementia.

Axol iPSCs are derived from patients with Amyotrophic lateral sclerosis (ALS), also known as motor neurone disease (MND) or Lou Gehrig’s disease.

Charcot–Marie–Tooth disease (CMT) is a hereditary motor and sensory neuropathy of the peripheral nervous system characterized by progressive loss of muscle tissue and touch sensation across various parts of the body.

Axol has over 60 disease lines from patients and genetically engineered models available

Dentatorubral-Pallidoluysian Atrophy (DRPLA) is an autosomal dominant spinocerebellar degeneration caused by an expansion of a CAG repeat encoding a polyglutamine tract in the atrophin-1 protein.

Friedreich’s ataxia (FRDA or FA) is an autosomal-recessive genetic disease caused by mutations in the FXN gene on chromosome 9, which makes a protein called frataxin.

Frontotemporal dementia (FTD) refers to a spectrum of clinical diseases that may have several different underlying pathologies.

Frontotemporal dementia (FTD) refers to a spectrum of clinical diseases that may have several different underlying pathologies.

Mucolipidosis IV is a rare inherited disorder characterized by intellectual disability and caused by mutations in the gene MCOLN1.

Associated with mutations in the TREM2 and TYROBP genes, Nasu-Hakola disease (NHD), also referred to as polycystic lipomembranous osteodysplasia with sclerosing leukoencephalopathy (PLOSL), is a rare inherited leukodystrophy.

 Parkinson’s Disease is a long-term degenerative disorder of the central nervous system that mainly affects the motor system

Axol has six lines derived from Huntington’s Disease patients. They are excellent models for investigating the disease and developing assays to screen potential therapies

Parkinsonism/Machado-Joseph Disease

Spinal and bulbar muscular atrophy is a debilitating neurodegenerative disorder caused by degeneration of motor neurons in the brainstem and spinal cord, and muscle wasting.

Spinocerebellar Ataxia is a rare inherited autosomal dominant disorder that causes progressive cerebellar ataxia.