ATON - Ataxia Telangiectasia Oligonucleotide Therapy

Ataxia Telangiectasia (A-T) is a rare genetic disease caused by mutations in the ATM gene, leading to neurological impairments, immunodeficiency, premature aging, and increased cancer and diabetes risk. No therapies are currently available for A-T,

IFOM researchers have identified a new target, alternative to ATM gene, that is upregulated in A-T cells, causing metabolic dysfunction and toxicity. Our approach uses antisense oligonucleotides (ASOs) to downregulate the specific target, restoring normal metabolism and enhancing cell viability in A-T cells, through a synthetic viability strategy, and blocking neurodegeneration.