Correction of Methylmalonic Aciduria In Vivo Using a Codon-Optimized Lentiviral Vector
Edward SY Wong, Chantelle McIntyre, Heidi L Peters, Enzo Ranieri, Donald S Anson, Janice M Fletcher
Human Gene Therapy | MARY ANN LIEBERT, INC | Published : 2014
This work was supported by funding from the Women's and Children's Hospital Foundation and the Australian National Health and Medical Research Council. The authors would like to thank Dr. Nicole Buck for providing assistance with the mouse model; Dr. David Johnson, Rosemarie Gerace, and Minh-Uyen Trinh for technical assistance with biochemical analyses; Dr. Peter Clements for technical support with the HPLC experiments; and Lynn Marsden for her assistance with animal sampling procedures.