CORDIS Project
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This project focuses on developing gene therapy for the most common type of severe combined immunodeficiency (SCID) caused by recombination activating gene-1 (RAG-1) deficiency. It aims to conduct clinical trials using autologous stem cell-based therapies to effectively treat over 70% of SCID patients in Europe.
Gene therapy for rare inherited immune disorders has become a clinical reality.
Especially for SCID, two major types of SCID (ADA-SCID, X-SCID) have been successfully treated by autologous stem cell based gene therapy.
However, for the most common group of SCID, the SCID underlying recombination defects, this has not yet occurred due to the higher complexities of the affected genes involved.
The aim of the current proposal is to fill the unmet medical need for the most common major category of…
Leiden University Medical Center
ACADEMISCH ZIEKENHUIS LEIDEN
Partner organizations (coordinator is shown above), with normalized type and CORDIS activity type. Guests see up to 4 partners.
United Kingdom, London
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
United Kingdom, London
Type: Public body
Activity type: Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
SME: No
Italy, Milano
Type: Company (for-profit)
Activity type: Private for-profit entities (excluding Higher or Secondary Education Establishments)
SME: No
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