New gene therapy constructs
AssigneesAlma Mater Studiorum - Università di Bologna, Oregon Health & Science University
InventorsElisabetta CIANI, Hiroyuki NAKAI, Stefania TRAZZI, Claudia FUCHS
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Abstract
The present invention provides a new vector for gene therapy said vector being therapeutically very efficient, viral particles comprising said vector, compositions comprising said viral particle, uses thereof, methods for the preparation of the vector), and therapies using said vector.
Claims42
A gene therapy vector comprising a first nucleic acid coding for a secretory leader sequence, operatively linked to a second nucleic acid coding for a protein transduction domain (PTD), operatively linked to a third nucleic acid coding for a therapeutic protein.
The gene therapy vector of 1 wherein said vector is an AAV vector, said secretory leader sequence is mouse IgK and said PTD is TATk.
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A viral particle consisting of a viral capsid and a gene therapy vector comprising a first nucleic acid coding for a secretory leader sequence, operatively linked to a second nucleic acid coding for a protein transduction domain (PTD), operatively linked to a third nucleic acid coding for a therapeutic protein.
(canceled)
A pharmaceutical composition comprising the viral particle according to claim and a pharmaceutically acceptable carrier.
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Dependencies are inferred from the claim text.