HPP
Hum Gene Ther. 2012 Apr;23(4):399-406. doi: 10.1089/hum.2011.148. Epub 2012 Jan 26. Successful gene therapy in utero for lethal murine hypophosphatasia. Sugano H1, Matsumoto T, Miyake K, Watanabe A, Iijima O, Migita M, Narisawa S, Millán J…
J Bone Miner Res. 2011 Jan;26(1):135-42. doi: 10.1002/jbmr.201. Prolonged survival and phenotypic correction of Akp2(-/-) hypophosphatasia mice by lentiviral gene therapy. Yamamoto S1, Orimo H, Matsumoto T, Iijima O, Narisawa S, Maeda T, M…