Fetal Gene Therapy for Ornithine Transcarbamylase Deficiency by Intrahepatic Plasmid DNA-Micro-bubble Injection Combined with Hepatic Ultrasound Insonation

Abstract We evaluated the therapeutic efficacy of hepatic transfection of plasmid DNA using micro-bubbles and ultrasound insonation for fetal correction of ornithine transcarbamylase (OTC) deficiency in mice. Twenty-three sparse-fur heterozygous pregnant mice (day 16 of gestation) were divided into...

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Veröffentlicht in:	Ultrasound in medicine & biology 2016-06, Vol.42 (6), p.1357-1361
Hauptverfasser:	Oishi, Yoshie, Kakimoto, Takashi, Yuan, Wenji, Kuno, Shuichi, Yamashita, Hiromasa, Chiba, Toshio
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Animals, Newborn Disease Models, Animal Female Genetic Therapy - methods Liver Male Mice Mice, Inbred BALB C Micro-bubble Microbubbles Ornithine Carbamoyltransferase Deficiency Disease - embryology Ornithine Carbamoyltransferase Deficiency Disease - therapy OTC deficient mouse Plasmid DNA Plasmids - therapeutic use Prenatal gene therapy Radiology Sonotransfection Transfection - methods Ultrasonic Therapy - methods Ultrasound insonation
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