α‐ l‐iduronidase gene‐based therapy using the phiC31 system to treat mucopolysaccharidose type I mice

ConclusionsphiC31‐mediated gene therapy resulted in an important improvement in IDUA –/– mice, including locomotion, although the obstacles that need to be overcome to enable long‐term gene therapy for MPSI are also noted. Copyright © 2015 John Wiley & Sons, Ltd.

Source: The Journal of Gene Medicine - March 27, 2015 Category: Genetics & Stem Cells Authors: Roberta Sessa Stilhano, Priscila Keiko Matsumoto Martin, Suely Maymone Melo, Vivian Yochiko Samoto, Giovani Bravin Peres, Yara Maria Correa Silva Michelacci, Flavia Helena Silva, Vanessa Gonçalves Pereira, Vania D'Almeida, Adriana Taveira Cruz, Miriam Ga Tags: Research Article Source Type: research