4 projets européens trouvés
Recherche sur 125080 projets européens
TERMINÉ
DNA binding proteins for treatment of gain of function mutations (ALLELECHOKER)
Date du début: 1 févr. 2013, Date de fin: 31 janv. 2018,
Zinc finger (ZF) and transcription activator-like effector (TALE) based technologies are been allowing the tailored design of “artificial” DNA-binding proteins targeted to specific and unique DNA genomic sequences. Coupling DNA binding proteins to effectors domains enables the constitution of DNA binding factors for genomic directed transcriptional modulation or targeted genomic editing. We have d ...
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1
Participant partenaire
TERMINÉ
DULBECCO TELETHON INSTITUTE-INTERNATIONAL MOBILITY FOR POSTDOCTORAL RESEARCH TRAINING (DTI-IMPORT)
Date du début: 1 déc. 2012, Date de fin: 30 nov. 2017,
With this proposal we are seeking an economical support from the European Union to implement an International Programme of Postdoctoral Research Training to enhance the international dimension of the fellows operating in the Dulbecco Telethon Institute (DTI) and to provide an excellent training to experienced researchers in the field of Research in the Biomedical Sciences that will likely contribu ...
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1
Participant partenaire
TERMINÉ
Clinical trial of gene therapy for MPS VI - a severe lysosomal storage disorder (MEUSIX)
Date du début: 1 déc. 2012, Date de fin: 30 nov. 2017,
"Mucopolysaccharidosis VI (MPS VI, or Maroteaux-Lamy syndrome; OMIM #253200) is a rare lysosomal storage disease caused by deficient activity of arylsulfatase B (ARSB). MPS VI is characterized by growth retardation, corneal clouding, cardiac valve disease, organomegaly, skeletal dysplasia, without central nervous system involvement. Thus, systemic therapies targeting peripheral organs have the pot ...
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9
Participants partenaires
TERMINÉ
Overcoming the challenge of large gene transfer for the therapy of inherited retinal diseases (RETGENTX)
Date du début: 1 janv. 2012, Date de fin: 31 déc. 2016,
Inherited retinal diseases (IRDs) cause blindness in over 200,000 individuals in Europe. The majority are due to mutations in genes expressed in photoreceptors (PR) in the retina. We have recently demonstrated the safety and efficacy of gene therapy for IRDs in patients with Leber congenital amaurosis (LCA). One of the major limitations to extend this clinical success to other blinding conditions ...
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