Virus-Based Nanoreactors with GALT Activity for Classic Galactosemia Therapy

被引:13
|
作者
Gama, Pedro [1 ]
Cadena-Nava, Ruben D. [1 ]
Juarez-Moreno, Karla [1 ]
Perez-Robles, Javier [1 ]
Vazquez-Duhalt, Rafael [1 ]
机构
[1] Univ Nacl Autonoma Mexico, Ctr Nanociencias & Nanotecnol, Dept Bionanotechnol, Ensenada, Baja California, Mexico
关键词
bio-nanoreactor; classic galactosemia; galactose-1-phosphate uridylyl-transferase; GALT; virus-like nanoparticles;
D O I
10.1002/cmdc.202000999
中图分类号
R914 [药物化学];
学科分类号
100701 ;
摘要
Enzymatic nanoreactors were obtained by galactose-1-phosphate uridylyl-transferase (GALT) encapsulation into plant virus capsids by a molecular self-assembly strategy. The aim of this work was to produce virus-like nanoparticles containing GALT for an enzyme-replacement therapy for classic galactosemia. The encapsulation efficiency and the catalytic constants of bio-nanoreactors were determined by using different GALT and virus coat protein ratios. The substrate affinity of nanoreactors was slightly lower than that of the free enzyme; the activity rate was 16 % of the GALT free enzyme. The enzymatic nanoreactors without functionalization were internalized into different cell lines including fibroblast and kidney cells, but especially into hepatocytes. The enzymatic nanoreactors are an innovative enzyme preparation with potential use for the treatment of classic galactosemia.
引用
收藏
页码:1438 / 1445
页数:8
相关论文
共 50 条
  • [21] Gene therapy using herpes simplex virus-based vectors
    Latchman, DS
    HISTOLOGY AND HISTOPATHOLOGY, 2000, 15 (04) : 1253 - 1259
  • [22] Adeno-associated virus-based vectors in gene therapy
    Tal, J
    JOURNAL OF BIOMEDICAL SCIENCE, 2000, 7 (04) : 279 - 291
  • [23] Evaluation of Virus-based Vectors for Cystic Fibrosis Gene Therapy
    Rodriguez-Villafane, Andrea
    Kuntz, Leah
    Limberis, Maria
    FASEB JOURNAL, 2020, 34
  • [24] Adeno-associated virus-based gene therapy for cystinuria
    Liao, Dazhou
    Yi, Xianyanling
    Bai, Yunjin
    Yang, Yang
    Ai, Jianzhong
    BJU INTERNATIONAL, 2024, 134 (05) : 742 - 744
  • [25] Targeting autophagy to enhance oncolytic virus-based cancer therapy
    Meng, Songshu
    Xu, Jiansheng
    Wu, Yantao
    Ding, Chan
    EXPERT OPINION ON BIOLOGICAL THERAPY, 2013, 13 (06) : 863 - 873
  • [26] Progress in clinical oncolytic virus-based therapy for hepatocellular carcinoma
    Jebar, Adel H.
    Errington-Mais, Fiona
    Vile, Richard G.
    Selby, Peter J.
    Melcher, Alan A.
    Griffin, Stephen
    JOURNAL OF GENERAL VIROLOGY, 2015, 96 : 1533 - 1550
  • [27] iPS CELL-BASED MODELING OF CLASSIC GALACTOSEMIA
    Demirbas, Didem
    Huang, Xiaoping
    Li, Xueli
    Cianci, Amy
    Fitzgerald, Kelly
    DeVine, Alexander
    Niederst, Emily
    Sahin, Mustafa
    Schlaeger, Thorsten
    He, Miao
    Daley, George Q.
    Berry, Gerard T.
    MOLECULAR GENETICS AND METABOLISM, 2016, 117 (03) : 235 - 235
  • [28] Epstein-Barr virus-based prognostic model in nodular sclerosis classic Hodgkin lymphoma
    Jiang, Chen
    Huang, Li-Yun
    Zhou, Ji-Hao
    Li, Zhi-Min
    Wang, Yu
    Li, Shuo
    Fu, Jian-Chang
    Huang, Qi-Tao
    Yan, Qin
    Huang, Yu-Yuan
    Zuo, Min
    Hu, Shimin
    Gale, Robert Peter
    Laing, Yang
    Yun, Jing-Ping
    Huang, Yu-Hua
    ISCIENCE, 2024, 27 (01)
  • [29] Classic Galactosemia: Clinical and Computational Characterization of a Novel GALT Missense Variant (p.A303D) and a Literature Review
    Forte, Giovanna
    Buonadonna, Antonia Lucia
    Pantaleo, Antonino
    Fasano, Candida
    Capodiferro, Donatella
    Grossi, Valentina
    Sanese, Paola
    Cariola, Filomena
    De Marco, Katia
    Lepore Signorile, Martina
    Manghisi, Andrea
    Guglielmi, Anna Filomena
    Simonetti, Simonetta
    Laforgia, Nicola
    Disciglio, Vittoria
    Simone, Cristiano
    Sergi, Consolato M.
    INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2023, 24 (24)
  • [30] Neonatal GALT gene replacement offers metabolic and phenotypic correction through early adulthood in a rat model of classic galactosemia
    Daenzer, Jennifer M. I.
    Rasmussen, Shauna A.
    Patel, Sneh
    McKenna, James, III
    Fridovich-Keil, Judith L.
    JOURNAL OF INHERITED METABOLIC DISEASE, 2022, 45 (02) : 203 - 214