Literature DB >> 21571082

Single intra-articular injection of adeno-associated virus results in stable and controllable in vivo transgene expression in normal rat knees.

K A Payne1, H H Lee, A M Haleem, C Martins, Z Yuan, C Qiao, X Xiao, C R Chu.   

Abstract

OBJECTIVE: To test the hypothesis that in vivo transgene expression mediated by single intra-articular injection of adeno-associated virus serotype 2 (AAV2) persists within intra-articular tissues 1 year post-injection and can be externally controlled using an AAV2-based tetracycline-inducible gene regulation system containing the tetracycline response element (TRE) promoter.
METHODS: Sprague Dawley rats received intra-articular injections of AAV2-cytomegalovirus (CMV)-enhanced green fluorescent protein (GFP) and AAV2-CMV-luciferase (Luc) into their right and left knees, respectively. Luciferase expression was evaluated over 1 year using bioluminescence imaging. After sacrifice, tissues were analyzed for GFP+ cells by fluorescent microscopy. To study external control of intra-articular AAV-transgene expression, another set of rats was co-injected with AAV2-TRE-Luc and AAV2-CMV-reverse-tetracycline-controlled transactivator (rtTA) into the right knees, and AAV2-CMV-Luc and AAV2-CMV-rtTA into the left knees. Rats received oral doxycycline (Dox), an analog of tetracycline, for 7 days. Luciferase expression was assessed by bioluminescence imaging.
RESULTS: Luciferase expression was localized to the injected joint and persisted throughout the 1-year study period. Abundant GFP+ cells were observed within intra-articular soft tissues. Transgene expression in AAV2-TRE-Luc injected joints was upregulated by oral administration of Dox, and downregulated following its removal, at 14 days and 13 months post-AAV injection.
CONCLUSIONS: This longitudinal in vivo study shows that sustained and stable AAV-mediated intra-articular transgene expression can be achieved through a single intra-articular injection and can be controlled using a tetracycline-controlled inducible AAV system in a normal rat knee model. Highly regulatable long-term intra-articular transgene expression is of potential clinical utility for development of treatment strategies for chronic intra-articular disease processes such as inflammatory and degenerative arthritis.
Copyright © 2011 Osteoarthritis Research Society International. Published by Elsevier Ltd. All rights reserved.

Entities:  

Mesh:

Substances:

Year:  2011        PMID: 21571082      PMCID: PMC3139006          DOI: 10.1016/j.joca.2011.04.009

Source DB:  PubMed          Journal:  Osteoarthritis Cartilage        ISSN: 1063-4584            Impact factor:   6.576


  42 in total

1.  Tight control of gene expression in mammalian cells by tetracycline-responsive promoters.

Authors:  M Gossen; H Bujard
Journal:  Proc Natl Acad Sci U S A       Date:  1992-06-15       Impact factor: 11.205

2.  Transgene persistence and cell turnover in the diarthrodial joint: implications for gene therapy of chronic joint diseases.

Authors:  Elvire Gouze; Jean-Noel Gouze; Glyn D Palmer; Carmencita Pilapil; Christopher H Evans; Steven C Ghivizzani
Journal:  Mol Ther       Date:  2007-04-17       Impact factor: 11.454

3.  Comparison of the efficacy of gentamicin for 5 days plus doxycycline for 8 weeks versus streptomycin for 2 weeks plus doxycycline for 45 days in the treatment of human brucellosis: a randomized clinical trial.

Authors:  M R Hasanjani Roushan; M J Soleimani Amiri; N Janmohammadi; M Sadeghi Hadad; M Javanian; M Baiani; A Bijani
Journal:  J Antimicrob Chemother       Date:  2010-03-09       Impact factor: 5.790

4.  Gene therapy platform for bone regeneration using an exogenously regulated, AAV-2-based gene expression system.

Authors:  Yossi Gafni; Gadi Pelled; Yoram Zilberman; Gadi Turgeman; Florence Apparailly; Hagit Yotvat; Eithan Galun; Zulma Gazit; Christian Jorgensen; Dan Gazit
Journal:  Mol Ther       Date:  2004-04       Impact factor: 11.454

5.  IGF-1 gene therapy to protect articular cartilage in a rat model of joint damage.

Authors:  Iñigo Izal; Carlos Alberto Acosta; Purificación Ripalda; Mikel Zaratiegui; Juan Ruiz; Francisco Forriol
Journal:  Arch Orthop Trauma Surg       Date:  2007-07-28       Impact factor: 3.067

6.  Ex vivo serotype-specific transduction of equine joint tissue by self-complementary adeno-associated viral vectors.

Authors:  L R Goodrich; V W Choi; B A Duda Carbone; C W McIlwraith; R J Samulski
Journal:  Hum Gene Ther       Date:  2009-12       Impact factor: 5.695

7.  Suppression of intra-articular responses to interleukin-1 by transfer of the interleukin-1 receptor antagonist gene to synovium.

Authors:  G L Hung; J Galea-Lauri; G M Mueller; H I Georgescu; L A Larkin; M K Suchanek; M H Tindal; P D Robbins; C H Evans
Journal:  Gene Ther       Date:  1994-01       Impact factor: 5.250

8.  Safety, tolerability, and clinical outcomes after intraarticular injection of a recombinant adeno-associated vector containing a tumor necrosis factor antagonist gene: results of a phase 1/2 Study.

Authors:  Philip J Mease; Nathan Wei; Edward J Fudman; Alan J Kivitz; Joy Schechtman; Robert G Trapp; Kathryn F Hobbs; Maria Greenwald; Antony Hou; Stephen A Bookbinder; Galen E Graham; Craig W Wiesenhutter; Larry Willis; Eric M Ruderman; Joseph Z Forstot; Michael J Maricic; Kathryn H Dao; Charles H Pritchard; Darrell N Fiske; Francis X Burch; H Malin Prupas; Pervin Anklesaria; Alison E Heald
Journal:  J Rheumatol       Date:  2009-12-23       Impact factor: 4.666

9.  Local delivery of beta interferon using an adeno-associated virus type 5 effectively inhibits adjuvant arthritis in rats.

Authors:  J Adriaansen; F J Fallaux; C J de Cortie; M J Vervoordeldonk; P P Tak
Journal:  J Gen Virol       Date:  2007-06       Impact factor: 3.891

Review 10.  The state of the art of adeno-associated virus-based vectors in gene therapy.

Authors:  Renata dos Santos Coura; Nance Beyer Nardi
Journal:  Virol J       Date:  2007-10-16       Impact factor: 4.099

View more
  19 in total

1.  FSH modulated cartilage ECM metabolism by targeting the PKA/CREB/SOX9 pathway.

Authors:  Lei Kong; Jin Xu; Mengqi Zhang; Yan Wang; Zhikun Huan; Yaping Liu; Wenwen Zhang; Dehuan Kong
Journal:  J Bone Miner Metab       Date:  2021-05-14       Impact factor: 2.626

2.  Multiple recombinant adeno-associated viral vector serotypes display persistent in vivo gene expression in vector-transduced rat stifle joints.

Authors:  Jeffrey B Mason; Brittney L Gurda; Julie B Engiles; Kurt D Hankenson; James M Wilson; Dean W Richardson
Journal:  Hum Gene Ther Methods       Date:  2013-06       Impact factor: 2.396

3.  Release of bioactive adeno-associated virus from fibrin scaffolds: effects of fibrin glue concentrations.

Authors:  Hannah H Lee; Amgad M Haleem; Veronica Yao; Juan Li; Xiao Xiao; Constance R Chu
Journal:  Tissue Eng Part A       Date:  2011-05-11       Impact factor: 3.845

Review 4.  Recent tissue engineering-based advances for effective rAAV-mediated gene transfer in the musculoskeletal system.

Authors:  Ana Rey-Rico; Magali Cucchiarini
Journal:  Bioengineered       Date:  2016-04       Impact factor: 3.269

5.  Humoral and cell-mediated immune response, and growth factor synthesis after direct intraarticular injection of rAAV2-IGF-I and rAAV5-IGF-I in the equine middle carpal joint.

Authors:  Kyla Ortved; Bettina Wagner; Roberto Calcedo; James Wilson; Deanna Schaefer; Alan Nixon
Journal:  Hum Gene Ther       Date:  2015-03       Impact factor: 5.695

6.  Persistence, localization, and external control of transgene expression after single injection of adeno-associated virus into injured joints.

Authors:  Hannah H Lee; Michael J O'Malley; Nicole A Friel; Karin A Payne; Chunping Qiao; Xiao Xiao; Constance R Chu
Journal:  Hum Gene Ther       Date:  2013-04       Impact factor: 5.695

7.  The Challenge and the Promise of Bone Marrow Cells for Human Cartilage Repair.

Authors:  Constance R Chu
Journal:  Cartilage       Date:  2015-03-24       Impact factor: 4.634

8.  Osteoarthritis: From Palliation to Prevention: AOA Critical Issues.

Authors:  Constance R Chu; Michael B Millis; Steven A Olson
Journal:  J Bone Joint Surg Am       Date:  2014-08-06       Impact factor: 5.284

Review 9.  Arthritis gene therapy and its tortuous path into the clinic.

Authors:  Christopher H Evans; Steven C Ghivizzani; Paul D Robbins
Journal:  Transl Res       Date:  2013-01-29       Impact factor: 7.012

Review 10.  Nondestructive Techniques to Evaluate the Characteristics and Development of Engineered Cartilage.

Authors:  Joseph M Mansour; Zhenghong Lee; Jean F Welter
Journal:  Ann Biomed Eng       Date:  2016-01-27       Impact factor: 3.934

View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.