Projektbeschreibung
Innovative Therapien zur Verhinderung von Blindheit durch Hornhauterkrankungen
Weltweit leben schätzungsweise 2,2 Milliarden Menschen mit einer Sehbeeinträchtigung, davon über 30 Millionen in Europa. Viele dieser Menschen sind aufgrund einer Hornhauterkrankung, der zweithäufigsten Ursache für Blindheit, erblindet. Diese Menschen erhalten nicht die benötigte Behandlung, da es an Zugang zu Spendergewebe mangelt, Behandlungen nicht standardisiert sind und es bei der Umsetzung innovativer regenerativer Therapien in die klinische Anwendung zu Verzögerungen kommt. Im EU-finanzierten Projekt ARREST BLINDNESS wird dieses Problem angegangen, indem biotechnisch erzeugte Gerüste, therapeutische Stamm- und Endothelzellen sowie regenerative Faktoren eingesetzt werden, um das neuronale Wachstum und die neuronale Funktion zu fördern. Darüber hinaus werden fortschrittliche Methoden zur Bildgebung und Überwachung entwickelt.
Ziel
Over 30 million Europeans are blind or visually impaired, leading to reduced quality of life and a tremendous loss of productivity in society. Corneal blindness is the second largest cause of blindness globally and while treatable, millions remain unnecessarily blind due to issues of access to transplantable tissue, lack of standardized treatments, and the lag in translating new regenerative medicine therapies to the clinic. The objective of ARREST BLINDNESS is therefore to develop and validate new regenerative-based therapies addressing a spectrum of blinding disorders of the cornea. These conditions either have no effective current treatments, depend on a scarce supply of donor tissue, or non-standardized methods are hindering validation of promising regenerative treatments. To achieve our objective, we will implant GMP-fabricated collagen-based bioengineered scaffolds to replace or regenerate the corneal stroma in cases of stromal thinning, scarring, dystrophy or trauma; deliver therapeutic epithelial stem and endothelial cells to the cornea to restore its transparency; deliver regenerative factors to promote neural growth and function; and actively maintain corneal immune privilege in high-risk situations by targeted therapeutic approaches to regress blood and lymphatic vessels. We will additionally develop advanced methods to image and monitor therapy throughout the cycle from GMP-compliant cell and scaffold preparation through the pre- and intra-operative stages, to postoperative follow-up and evaluation. After proof-of-concept and preclinical validation of key enabling components, these technologies will be used by one or several partners in preclinical models and in phase I/II human clinical studies. ARREST BLINDNESS directly addresses the translation of regenerative medicine, bio-artificial organs, tissue engineered scaffolds, and advanced cell and gene therapies into clinical use and will help to alleviate the worldwide problem of corneal blindness.
Wissenschaftliches Gebiet
- medical and health sciencesmedical biotechnologygenetic engineeringgene therapy
- medical and health sciencesbasic medicinepharmacology and pharmacypharmaceutical drugs
- medical and health sciencesclinical medicineophthalmology
- medical and health sciencesmedical biotechnologycells technologiesstem cells
- medical and health sciencesclinical medicinetransplantation
Programm/Programme
Aufforderung zur Vorschlagseinreichung
Andere Projekte für diesen Aufruf anzeigenUnterauftrag
H2020-PHC-2015-two-stage
Finanzierungsplan
RIA - Research and Innovation actionKoordinator
581 83 Linkoping
Schweden