IDUA gene based therapy using phiC31 system to treat MPSI mice

ConclusionThe phiC31 mediated gene therapy resulted an important improvement in IDUA‐/‐ mice, including locomotion, but we also highlighted obstacles to be overcome to reach a long‐term gene therapy for MPSI. This article is protected by copyright. All rights reserved.

Source: The Journal of Gene Medicine - November 1, 2014 Category: Genetics & Stem Cells Authors: Roberta Sessa Stilhano, Priscila Keiko Matsumoto Martin, Suely Maymone Melo, Vivian Yochiko Samoto, Giovani Bravin Peres, Yara Maria Correa da Silva Michelacci, Flavia Helena Silva, Vanessa Gonçalves Pereira, Vania D'Almeida, Adriana Taveira Cruz, Miriam Tags: Research Article Source Type: research