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Development of gene therapy and genome editing strategies to treat adenosine deaminase 2 deficiency

Project description

Gene therapy for adenosine deaminase deficiency

Deficiency of adenosine deaminase type 2 (DADA2) is a rare, inherited disorder characterised by abnormal inflammation of various tissues and immunodeficiency. It is caused by mutations in the ADA2 gene, rendering it an ideal candidate for gene therapy. The EU-funded DADA2GT project is exploring a gene therapy strategy for correcting ADA2 mutations in homologous haematopoietic stem cells using gene-editing technologies or gene addition by lentiviral vectors. In a clinical setting, these gene-corrected cells will be infused back to the patient. DADA2GT is a proof-of-concept study with a key goal to evaluate the efficacy of gene correction at the preclinical level using patient-derived cells.

Coordinator

OSPEDALE SAN RAFFAELE SRL
Net EU contribution
€ 183 473,28
Address
Via Olgettina 60
20132 Milano
Italy

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Region
Nord-Ovest Lombardia Milano
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Other funding
€ 0,00