CORDIS Project
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This project focuses on developing gene therapy as a treatment for adenosine deaminase type 2 deficiency, a severe immunodeficiency disorder. It aims to correct genetic mutations in patient-derived stem cells to provide targeted therapeutic options.
Deficiency of adenosine deaminase type 2 (DADA2) caused by loss-of-function mutations in the ADDeficiency of adenosine deaminase type 2 (DADA2) caused by loss-of-function mutations in the ADA2 gene is a Primary immunodeficiency (PID) diseases characterized by vasculopathy, stroke, intracranial haemorrhages, systemic inflammation, immunodeficiency, and cytopenia.
Without proper treatment, DADA2 patients are at high risk of severe disability or death.
Targeted pharmacological therapies are not ava…
OSPEDALE SAN RAFFAELE SRL
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