GENE THERAPY FOR GLYCOGEN STORAGE DISEASES
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United States of America Patent
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Issued Date -
Oct 1, 2015
app pub date -
Oct 17, 2013
filing date -
Oct 17, 2012
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Abandoned
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Abstract
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.
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