GENE THERAPY FOR THE TREATMENT OF HT1

Abstract: the present disclosure provides compositions and methods for gene therapy. further, the present disclosure provides compositions and methods for treatment of ht1 through novel gene therapy mechanisms.

Inventor(s): Qiang Xiong, Chih-Wei Ko

CPC Classification: A61K48/005 (Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy)

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