CORDIS Project
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UshTher focuses on developing a novel gene therapy for Usher syndrome type 1B, which causes deafness and blindness. The project tests a dual AAV vector approach to deliver the MYO7A gene, aiming for a potential cure.
Usher syndrome (USH) is the most common combination of deafness and blindness due to retinitis pigmentosa. USHIB caused by mutations in the large MYO7A gene, is among the most severe and frequent forms of USH.
While deafness can be improved with cochlear implants, blindness remains untreatable.
Retinal gene therapy with AAV vectors is both safe and effective in humans, however it is limited by AAV cargo capacity which would not allow transfer of the large MYO7A expression cassette.
UshTher ob…
Partner organizations (coordinator is shown above), with normalized type and CORDIS activity type. Guests see up to 4 partners.
France, EVRY
Type: SME
Activity type: Private for-profit entities (excluding Higher or Secondary Education Establishments)
SME: Yes
AZIENDA OSPEDALIERA UNIVERSITARIA UNIVERSITA DEGLI STUDI DELLA CAMPANIA LUIGI VANVITELLI
Italy, Napoli
Type: Public body
Activity type: Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
SME: No
UNIVERSITA DEGLI STUDI DELLA CAMPANIA LUIGI VANVITELLI
Italy, Caserta
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
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