TFEB GENE THERAPY OF ALPHA-1-ANTITRYPSIN DEFICIENCY

摘要

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).