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Literature DB >> 22875565

Production and discovery of novel recombinant adeno-associated viral vectors.

Christian Mueller¹, Dmitry Ratner, Li Zhong, Miguel Esteves-Sena, Guangping Gao.

Abstract

In this unit, we describe the detailed procedure for a three-plasmid transfection method for rAAV production, and discuss its advantages, limitations, and troubleshooting techniques. We further discuss the rAAV purification process using CsCl gradients, as well as subsequent quality control methods using SDS-PAGE and real-time PCR to assess vector purity, packaging efficiency, and viral titer. Finally, we elaborate on a PCR-based strategy that can be used to discover novel AAV capsid sequences from primate tissue, which can be used to develop newer-generation rAAVs with a greater diversity of tissue tropism for clinical gene therapy.

Entities: CellLine Chemical Disease Gene Species

Mesh：

Year: 2012 PMID： 22875565 PMCID： PMC4358842 DOI： 10.1002/9780471729259.mc14d01s26

Source DB: PubMed Journal: Curr Protoc Microbiol

37 in total

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Journal: J Virol Date: 1997-11 Impact factor: 5.103

2. Identification of factors that contribute to recombinant AAV2 particle aggregation and methods to prevent its occurrence during vector purification and formulation.

Authors: J Fraser Wright; Tannie Le; Joseph Prado; Jennifer Bahr-Davidson; Peter H Smith; Zhu Zhen; Jurg M Sommer; Glenn F Pierce; Guang Qu
Journal: Mol Ther Date: 2005-07 Impact factor: 11.454

3. The cellular transcription factor SP1 and an unknown cellular protein are required to mediate Rep protein activation of the adeno-associated virus p19 promoter.

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Journal: J Virol Date: 1997-03 Impact factor: 5.103

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Journal: Proc Natl Acad Sci U S A Date: 1967-10 Impact factor: 11.205

Review 5. Gene therapy vectors as drug delivery systems.

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Journal: Clin Pharmacokinet Date: 1995-03 Impact factor: 6.447

6. The adeno-associated virus type 2 p40 promoter requires a proximal Sp1 interaction and a p19 CArG-like element to facilitate Rep transactivation.

Authors: D J Pereira; N Muzyczka
Journal: J Virol Date: 1997-06 Impact factor: 5.103

7. The adeno-associated virus (AAV) Rep protein acts as both a repressor and an activator to regulate AAV transcription during a productive infection.

Authors: D J Pereira; D M McCarty; N Muzyczka
Journal: J Virol Date: 1997-02 Impact factor: 5.103

8. High-titer adeno-associated viral vectors from a Rep/Cap cell line and hybrid shuttle virus.

Authors: G P Gao; G Qu; L Z Faust; R K Engdahl; W Xiao; J V Hughes; P W Zoltick; J M Wilson
Journal: Hum Gene Ther Date: 1998-11-01 Impact factor: 5.695

9. In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA.

Authors: Pedro E Cruz; Christian Mueller; Travis L Cossette; Alexandra Golant; Qiushi Tang; Stuart G Beattie; Mark Brantly; Martha Campbell-Thompson; Keith S Blomenkamp; Jeffrey H Teckman; Terence R Flotte
Journal: Lab Invest Date: 2007-06-25 Impact factor: 5.662

10. Adeno-associated virus-targeted disruption of the CFTR gene in cloned ferrets.

Authors: Xingshen Sun; Ziying Yan; Yaling Yi; Ziyi Li; Diana Lei; Christopher S Rogers; Juan Chen; Yulong Zhang; Michael J Welsh; Gregory H Leno; John F Engelhardt
Journal: J Clin Invest Date: 2008-04 Impact factor: 14.808

32 in total

Review 1. Extracting structural and functional features of widely distributed biological circuits with single cell resolution via tissue clearing and delivery vectors.

Authors: Jennifer Brooke Treweek; Viviana Gradinaru
Journal: Curr Opin Biotechnol Date: 2016-07-06 Impact factor: 9.740

2. Adeno-Associated Virus Delivery of Anti-HIV Monoclonal Antibodies Can Drive Long-Term Virologic Suppression.

Authors: José M Martinez-Navio; Sebastian P Fuchs; Shara N Pantry; William A Lauer; Natasha N Duggan; Brandon F Keele; Eva G Rakasz; Guangping Gao; Jeffrey D Lifson; Ronald C Desrosiers
Journal: Immunity Date: 2019-03-05 Impact factor: 31.745

3. Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials.

Authors: Alisha M Gruntman; Lin Su; Qin Su; Guangping Gao; Christian Mueller; Terence R Flotte
Journal: Hum Gene Ther Methods Date: 2015-04 Impact factor: 2.396

4. Intralingual Administration of AAVrh10-miR^SOD1 Improves Respiratory But Not Swallowing Function in a Superoxide Dismutase-1 Mouse Model of Amyotrophic Lateral Sclerosis.

Authors: Lori A Lind; Ellyn M Andel; Angela L McCall; Justin S Dhindsa; Katherine A Johnson; Olivia E Stricklin; Christian Mueller; Mai K ElMallah; Teresa E Lever; Nicole L Nichols
Journal: Hum Gene Ther Date: 2020-07-13 Impact factor: 5.695

5. Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity.

Authors: Yuanyuan Xiao; Manish Muhuri; Shaoyong Li; Wanru Qin; Guangchao Xu; Li Luo; Jia Li; Alexander J Letizia; Sean K Wang; Ying Kai Chan; Chunmei Wang; Sebastian P Fuchs; Dan Wang; Qin Su; M Abu Nahid; George M Church; Michael Farzan; Li Yang; Yuquan Wei; Ronald C Desrosiers; Christian Mueller; Phillip Wl Tai; Guangping Gao
Journal: JCI Insight Date: 2019-05-21

6. Dopamine transporter trafficking and Rit2 GTPase: Mechanism of action and in vivo impact.

Authors: Rita R Fagan; Patrick J Kearney; Carolyn G Sweeney; Dino Luethi; Florianne E Schoot Uiterkamp; Klaus Schicker; Brian S Alejandro; Lauren C O'Connor; Harald H Sitte; Haley E Melikian
Journal: J Biol Chem Date: 2020-03-04 Impact factor: 5.157

7. AAV-delivered eCD4-Ig protects rhesus macaques from high-dose SIVmac239 challenges.

Authors: Matthew R Gardner; Christoph H Fellinger; Lisa M Kattenhorn; Meredith E Davis-Gardner; Jesse A Weber; Barnett Alfant; Amber S Zhou; Neha R Prasad; Hema R Kondur; Wendy A Newton; Kimberly L Weisgrau; Eva G Rakasz; Jeffrey D Lifson; Guangping Gao; Nancy Schultz-Darken; Michael Farzan
Journal: Sci Transl Med Date: 2019-07-24 Impact factor: 17.956