CORDIS Project
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This project aims to enhance gene therapy for hematopoietic stem cells by improving gene transfer methods. It focuses on using microRNA regulation and targeted integration techniques to ensure safe and effective gene expression, with potential applications in regenerative medicine.
Hematopoietic stem cell gene therapy has a tremendous potential to treat human disease.
Yet, in conjunction with the first successful results in the clinic, severe adverse events linked to the gene transfer protocol were reported.
Recently, we provided proof-of-principle of two new powerful strategies to improve the efficacy and safety of gene transfer: 1) regulating transgene expression by exploiting cellular microRNAs; 2) targeting integration at predetermined sites of the genome by forcing ho…
UNIVERSITA VITA-SALUTE SAN RAFFAELE
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Italy, Milano
Type: Company (for-profit)
Activity type: Private for-profit entities (excluding Higher or Secondary Education Establishments)
SME: No
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