Yale University, Ospedale San Raffaele Srl and Fondazione Telethon | Date: 2013-06-18
The invention is based upon the discovery that T regulatory type 1 (Tr1) cells express particular cell surface markers that allow for their selection, enrichment, isolation, purification and administration. The ability to use the particular markers described herein to select, enrich, isolate, purity and administer Tr1 cells allows for improved methods of Tr1 therapies for treating a wide variety of diseases and disorders.
Fondazione Telethon | Date: 2015-07-29
The invention refers to TFEB related molecules, as variants, mutants, truncated proteins, chimeras etc. that are constitutively localized in the nucleus of a eukaryote cell. Such molecules have a therapeutic applicability in all of disorders that need of an induction of the cell authophagic/lysosomal system, as lysosomal storage disorders, neurodegenerative diseases, hepatic diseases, muscle diseases and metabolic diseases.
Fondazione Telethon | Date: 2014-04-18
The present invention relates to constructs, vectors, relative host cells and pharmaceutical compositions which allow an effective gene therapy, in particular of genes larger than 5 Kb.
Fondazione Telethon | Date: 2013-10-17
The present invention relates to a transcription factor EB (TFEB) protein, ortholog, recombinant or synthetic or bio technological functional derivative thereof, allelic variant thereof and fragments thereof; a chimeric molecule comprising the TFEB protein, ortholog, recombinant or synthetic or biotechnological functional derivative thereof, allelic variant thereof and fragments thereof; a polynucleotide coding for said protein or ortholog, recombinant or synthetic or biotechnological functional derivative thereof, allelic variant thereof and fragments thereof; a vector comprising said polynucleotide; a host cell genetically engineered expressing said polypeptide or a pharmaceutical composition for use in the treatment or/and prevention of a glycogen storage disease. Preferably of Pompe or Danon disease.
Fondazione Telethon | Date: 2015-11-12
The invention provides for nucleotide sequences encoding for a chimeric sulfatase, viral vectors expressing such sequences for gene therapy and pharmaceutical uses of the chimeric expressed protein. The invention is particularly applied in the therapy of mucopolysaccharidosis, preferably type IIIA.