CORDIS Project
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The project focuses on developing a gene therapy for Acute Intermittent Porphyria (AIP), a rare genetic disorder. It aims to deliver a modified virus to enhance the production of a critical enzyme, potentially preventing life-threatening symptoms associated with the disease.
Acute intermittent porphyria (AIP) is a rare genetic disease in which mutations in the porphobilinogen deaminase (PBGD) gene produce insufficient activity of a protein necessary for heme synthesis.
This leads to an accumulation of toxic intermediates resulting in a wide variety of problems including acute, severe abdominal pains, psychiatric and neurological disorders, and muscular weakness.
Acute porphyric attacks can be life-threatening and the long-term consequences include irreversible nerve…
FUNDACION PARA LA INVESTIGACION MEDICA APLICADA FIMA
Partner organizations (coordinator is shown above), with normalized type and CORDIS activity type. Guests see up to 4 partners.
Netherlands, AMSTERDAM
Type: Company (for-profit)
Activity type: Private for-profit entities (excluding Higher or Secondary Education Establishments)
Spain, Pamplona
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
Sweden, Stockholm
Type: Public body
Activity type: Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
SME: No
Germany, Heidelberg
Type: Research institute
Activity type: Research Organisations
SME: No
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