Organoid transduction using recombinant adeno-associated viral vectors: Challenges and opportunities

被引：1
|
作者：
Belova, Lyubava [1 ]
Lavrov, Alexander [1 ]
Smirnikhina, Svetlana [1 ]
机构：
[1] Res Ctr Med Genet, Moscow 115478, Russia
来源：
BIOESSAYS | 2022年 / 44卷 / 09期
关键词：
adeno-associated viral vectors; cellular heterogeneity; gene therapy; organoids; retinal organoids; PLURIPOTENT STEM-CELLS; GENE-THERAPY; LONG-TERM; CEREBRAL ORGANOIDS; VIRUS SEROTYPES; IMMUNE-RESPONSES; PROGENITOR CELLS; AAV VECTORS; HUMAN LIVER; FACTOR-IX;
D O I：
10.1002/bies.202200055
中图分类号：
Q5 [生物化学]; Q7 [分子生物学];
学科分类号：
071010 ; 081704 ;
摘要：
Cellular 3D structures, for example, organoids, are an excellent model for studying and developing treatments for various diseases, including hereditary ones. Therefore, they are increasingly being used in biomedical research. From the point of view of safety and efficacy, recombinant adeno-associated viral (rAAV) vectors are currently most in demand for the delivery of various transgenes for gene replacement therapy or other applications. The delivery of transgenes using rAAV vectors to various types of organoids is an urgent task, however, it is associated with a number of problems that are discussed in this review. Cellular heterogeneity and specifics of cultivation of 3D structures determine the complexity of rAAV delivery and are sometimes associated with low transduction efficiency. This review surveys the main ways to solve emerging problems and increase the efficiency of transgene delivery using rAAVs to organoids. A clear understanding of the stage of development of the organoid, its cellular composition and the presence of surface receptors will allow obtaining high levels of organoid transduction with existing rAAV vectors.
引用
收藏
页数：13
相关论文
共 50 条
  • [1] The role of furin in recombinant adeno-associated viral vectors infection and transduction
    Suarez, L.
    Paneda, A.
    Zabaleta, V.
    Olaguee, C.
    Vales, A.
    Buning, H.
    Aldabe, R.
    Prieto, J.
    Gonzalez-Aseguinolaza, G.
    [J]. HUMAN GENE THERAPY, 2013, 24 (12) : A72 - A72
  • [2] Transduction of human neural progenitor cells using recombinant adeno-associated viral vectors
    P Wu
    Y Ye
    CN Svendsen
    [J]. Gene Therapy, 2002, 9 : 245 - 255
  • [3] Transduction of human neural progenitor cells using recombinant adeno-associated viral vectors
    Wu, P
    Ye, Y
    Svendsen, CN
    [J]. GENE THERAPY, 2002, 9 (04) : 245 - 255
  • [4] Purification of Recombinant Adeno-Associated Viral Vectors
    Pan, Xing
    [J]. MOLECULAR THERAPY, 2020, 28 (04) : 538 - 538
  • [5] The use of recombinant adeno-associated viral vectors for the transduction of epithelial tumor cells
    Hoerer, M
    Bogedain, C
    Scheer, U
    Heberger, C
    Steyrer, S
    Burger, A
    Maass, G
    [J]. INTERNATIONAL JOURNAL OF IMMUNOPHARMACOLOGY, 1997, 19 (9-10): : 473 - 479
  • [6] Enhancing transduction of the liver by adeno-associated viral vectors
    A C Nathwani
    M Cochrane
    J McIntosh
    C Y C Ng
    J Zhou
    J T Gray
    A M Davidoff
    [J]. Gene Therapy, 2009, 16 : 60 - 69
  • [7] Enhancing transduction of the liver by adeno-associated viral vectors
    Nathwani, A. C.
    Cochrane, M.
    McIntosh, J.
    Ng, C. Y. C.
    Zhou, J.
    Gray, J. T.
    Davidoff, A. M.
    [J]. GENE THERAPY, 2009, 16 (01) : 60 - 69
  • [8] Cellular transduction mechanisms of adeno-associated viral vectors
    Berry, Garrett Edward
    Asokan, Aravind
    [J]. CURRENT OPINION IN VIROLOGY, 2016, 21 : 54 - 60
  • [9] Recombinant adeno-associated viral vectors in the nervous system
    Burger, C
    Nash, K
    Mandel, RJ
    [J]. HUMAN GENE THERAPY, 2005, 16 (07) : 781 - 791
  • [10] Production and Titering of Recombinant Adeno-associated Viral Vectors
    McClure, Christina
    Cole, Katy L. H.
    Wulff, Peer
    Klugmann, Matthias
    Murray, Andrew J.
    [J]. JOVE-JOURNAL OF VISUALIZED EXPERIMENTS, 2011, (57):
12345