Synthetic high-density lipoprotein nanoparticles for the treatment of Niemann-Pick diseases.

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第一作者： Mark L,Schultz

第一单位： Department of Pathology, University of Michigan Medical School, 3510 MSRB1, 1150 W. Medical Center Dr., Ann Arbor, MI, 48109, USA.

作者： Mark L,Schultz ^[1] ; Maria V,Fawaz ^[2] ; Ruth D,Azaria ^[1] ; Todd C,Hollon ^[3] ; Elaine A,Liu ^[1] ; Thaddeus J,Kunkel ^[4] ; Troy A,Halseth ^[5] ; Kelsey L,Krus ^[1] ; Ran,Ming ^[2] ; Emily E,Morin ^[1] ; Hayley S,McLoughlin ^[6] ; David D,Bushart ^[6] ; Henry L,Paulson ^[7] ; Vikram G,Shakkottai ^[7] ; Daniel A,Orringer ^[8] ; Anna S,Schwendeman ^[7] ; Andrew P,Lieberman ^[7]

作者单位： Department of Pathology, University of Michigan Medical School, 3510 MSRB1, 1150 W. Medical Center Dr., Ann Arbor, MI, 48109, USA. ^[1] Department of Medicinal Chemistry, College of Pharmacy, University of Michigan, Ann Arbor, MI, 48109, USA. ^[2] Department of Neurosurgery, University of Michigan Medical School, Ann Arbor, MI, 48109, USA. ^[3] Cellular and Molecular Biology Graduate Program, University of Michigan Medical School, Ann Arbor, MI, 48109, USA. ^[4] Medical Scientist Training Program, University of Michigan Medical School, Ann Arbor, MI, 48109, USA. ^[5] Department of Pharmaceutical Sciences, University of Michigan College of Pharmacy, B20-102W NCRC, 2800 Plymouth Road, Ann Arbor, MI, 48109, USA. ^[6] Department of Neurology, University of Michigan Medical School, Ann Arbor, MI, 48109, USA. ^[7] Department of Molecular & Integrative Physiology, University of Michigan Medical School, Ann Arbor, MI, 48109, USA. ^[8] Department of Pharmaceutical Sciences, University of Michigan College of Pharmacy, B20-102W NCRC, 2800 Plymouth Road, Ann Arbor, MI, 48109, USA. annaschw@umich.edu. ^[9] Biointerfaces Institute, University of Michigan, Ann Arbor, MI, 48109, USA. annaschw@umich.edu. ^[10] Department of Pathology, University of Michigan Medical School, 3510 MSRB1, 1150 W. Medical Center Dr., Ann Arbor, MI, 48109, USA. liebermn@umich.edu. ^[11]

关键词 HDL NPC1 Niemann–Pick A Niemann–Pick C SRS