TFEB GENE THERAPY OF ALPHA-1-ANTITRYPSIN DEFICIENCY
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United States of America Patent
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Issued Date -
Mar 19, 2015
app pub date -
Apr 19, 2013
filing date -
Apr 19, 2012
priority date (Note) -
Abandoned
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Abstract
The present invention refers to a vector for gene therapy comprising a TFEB coding sequence under the control of a promoter able to efficiently express said TFEB coding sequence, to host cell comprising said vector and to their use in the gene therapy of a pathological condition characterized by a deficiency of alpha-1-antitrypsin (AAT). The present invention also refers to a pharmaceutical composition comprising the vector or the host cell of the invention for gene therapy and to a method for gene therapy of a pathological condition characterized by a deficiency of alpha-1-antitrypsin (AAT).
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