Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors

被引：0
|
作者：
Kim, Ah Young [1 ]
Duerr, Felix Michael [1 ]
Phillips, Jennifer [1 ]
Samulski, Richard Jude [2 ,3 ]
Grieger, Josh C. [3 ]
Goodrich, Laurie R. [1 ]
机构：
[1] Colorado State Univ, Coll Vet Med & Biomed Sci, Dept Clin Sci, 300 Drake Rd, Ft Collins, CO 80523 USA
[2] Gene Therapy Ctr, 7011 Thurston Bowles Bldg,104 Manning Dr, Chapel Hill, NC 27599 USA
[3] Asklepios Biopharmaceut Inc, 20 TW Alexander Dr 110, Res Triangle Pk, NC 27709 USA
来源：
GENE THERAPY | 2023年 / 30卷 / 3-4期
关键词：
MEDIATED GENE-THERAPY; NEUTRALIZING ANTIBODIES; AAV VECTORS; OSTEOARTHRITIS; EXPRESSION; DELIVERY; RECEPTOR; INJECTION;
D O I：
10.1038/s41434-022-00366-x
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：
A formal screening of self-complementary adeno-associated virus (scAAV) vector serotypes in canine joint tissues has not been performed to date. Selecting appropriate serotypes is crucial for successful treatment due to their varying levels of tissue tropism. The objective of this study is to identify the most optimal scAAV vector serotype that maximizes transduction efficiencies in canine cell monolayer cultures (chondrocytes, synoviocytes, and mesenchymal stem cells) and tissue explant cultures (cartilage and synovium). Transduction efficiencies of scAAV serotypes 1, 2, 2.5, 3, 4, 5, 6, 8, and 9 were evaluated in each culture type in three different vector concentrations by encoding a green fluorescent protein. It was found that scAAV2 and 2.5 showed the overall highest transduction efficiency among serotypes with dose-response. Since possible immune response against conventional AAV2 was previously reported in dogs, the chimeric scAAV2.5 may be more suitable to use. Evaluation of the safety and efficacy of the scAAV2.5 vector with an appropriate therapeutic gene in vivo is indicated.
引用
收藏
页码：398 / 404
页数：7
相关论文
共 50 条
  • [11] Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges
    Raj, Deepak
    Davidoff, Andrew M.
    Nathwani, Amit C.
    EXPERT REVIEW OF HEMATOLOGY, 2011, 4 (05) : 539 - 549
  • [12] Self-Complementary Adeno-Associated Virus Vectors: Packaging Capacity and Purity
    Wu, Jianqing
    Zhao, Weihong
    Zhong, Li
    Warrington, Kenneth H., Jr.
    Srivastava, Arun
    MOLECULAR THERAPY, 2006, 13 : S286 - S286
  • [13] Limitations of Encapsidation of Recombinant Self-Complementary Adeno-Associated Viral Genomes in Different Serotype Capsids and Their Quantitation
    Wang, Yuan
    Ling, Chen
    Song, Liujiang
    Wang, Lina
    Aslanidi, George V.
    Tan, Mengqun
    Ling, Changquan
    Srivastava, Arun
    HUMAN GENE THERAPY METHODS, 2012, 23 (04) : 225 - 233
  • [14] High Efficiency Gene Targeting to Mammalian Joint Tissue Using Self-Complementary Adeno-Associated Viral Vector Serotypes
    Goodrich, Laurie R.
    Choi, Vivian W.
    DudaCarbone, Beth C.
    McIlwraith, C. Wayne
    Samulski, Richard J.
    MOLECULAR THERAPY, 2006, 13 : S191 - S191
  • [15] Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis
    DM McCarty
    PE Monahan
    RJ Samulski
    Gene Therapy, 2001, 8 : 1248 - 1254
  • [16] Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis
    McCarty, DM
    Monahan, PE
    Samulski, RJ
    GENE THERAPY, 2001, 8 (16) : 1248 - 1254
  • [17] Self-complementary recombinant adeno-associated viral vectors: Packaging capacity and the role of rep proteins in vector purity
    Wu, Jianqing
    Zhao, Weihong
    Zhong, Li
    Han, Zongchao
    Li, Baozheng
    Ma, Wenqin
    Weigel-Kelley, Kirsten A.
    Warrington, Kenneth H., Jr.
    Srivastava, Arun
    HUMAN GENE THERAPY, 2007, 18 (02) : 171 - 182
  • [18] Differential internalization and nuclear uncoating of self-complementary adeno-associated virus pseudotype vectors as determinants of cardiac cell transduction
    Sipo, I.
    Fechner, H.
    Pinkert, S.
    Suckau, L.
    Wang, X.
    Weger, S.
    Poller, W.
    GENE THERAPY, 2007, 14 (18) : 1319 - 1329
  • [19] Differential internalization and nuclear uncoating of self-complementary adeno-associated virus pseudotype vectors as determinants of cardiac cell transduction
    I Sipo
    H Fechner
    S Pinkert
    L Suckau
    X Wang
    S Weger
    W Poller
    Gene Therapy, 2007, 14 : 1319 - 1329
  • [20] Serotype-dependent transduction efficiencies of recombinant adeno-associated viral vectors in monkey neocortex
    Gerits, Annelies
    Vancraeyenest, Pascaline
    Vreysen, Samme
    Laramee, Marie-Eve
    Michiels, Annelies
    Gijsbers, Rik
    Van den Haute, Chris
    Moons, Lieve
    Debyser, Zeger
    Baekelandt, Veerle
    Arckens, Lutgarde
    Vanduffel, Wim
    NEUROPHOTONICS, 2015, 2 (03)
12345