Fetal therapies and trials for lysosomal storage diseases: a survey of attitudes of parents and patients.

导出 OA

翻译打印收藏纠错

作者： Marisa E,Schwab ^[1] ; Julia E H,Brown ^[2] ; Billie,Lianoglou ^[3] ; Chengshi,Jin ^[4] ; Patricia C,Conroy ^[5] ; Renata C,Gallagher ^[1] ; Paul,Harmatz ^[2] ; Tippi C,MacKenzie ^[6]

作者单位： Fetal Treatment Center, University of California, San Francisco, CA, USA. ^[1] Center for Maternal-Fetal Precision Medicine, University of California, San Francisco, CA, USA. ^[2] Department of Surgery, Division of Pediatric Surgery, University of California, 505 Parnassus Avenue, San Francisco, CA, 94143, USA. ^[3] Program in Bioethics, University of California, San Francisco, CA, USA. ^[4] Institute for Health & Aging, University of California, San Francisco, CA, USA. ^[5] Biostatistics Core, Department of Surgery, University of California, San Francisco, CA, USA. ^[6] Department of Pediatrics, University of California, San Francisco, CA, USA. ^[7] Fetal Treatment Center, University of California, San Francisco, CA, USA. tippi.mackenzie@ucsf.edu. ^[8] Center for Maternal-Fetal Precision Medicine, University of California, San Francisco, CA, USA. tippi.mackenzie@ucsf.edu. ^[9] Department of Surgery, Division of Pediatric Surgery, University of California, 505 Parnassus Avenue, San Francisco, CA, 94143, USA. tippi.mackenzie@ucsf.edu. ^[10]

关键词 Clinical trial ELSI (ethical, legal and social implications)Enzyme replacement therapy Fetal therapy Gene therapy Lysosomal storage disease Mucopolysaccharidosis Patient attitudes