CORDIS Project
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This project seeks to enhance gene therapy techniques for treating inherited diseases and cancer using engineered nucleases. It aims to develop safer, more effective methods for genetic modification of hematopoietic cells.
Substantial evidence supports the therapeutic potential of ex vivo gene therapy based on Hematopoietic Stem Cell (HSC) or T lymphocytes to treat inherited diseases or cancer.
Yet, the intrinsic limitations of current gene replacement approaches based on semi-randomly integrating vectors, and the occurrence in some clinical trials of severe adverse events related to gene transfer, prevent safe deployment and broad application of gene therapy.
This project aims to overcome these limits by exploiti…
UNIVERSITA VITA-SALUTE SAN RAFFAELE
Partner organizations (coordinator is shown above), with normalized type and CORDIS activity type. Guests see up to 4 partners.
Germany, Heidelberg
Type: Research institute
Activity type: Research Organisations
SME: No
LONDON
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
Italy, BRESSO MI
Type: Company (for-profit)
Activity type: Private for-profit entities (excluding Higher or Secondary Education Establishments)
SME: No
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