Molecular design for recombinant adeno-associated virus (rAAV) vector production.

Abstract:

:Recombinant adeno-associated virus (rAAV) vectors are increasingly popular tools for gene therapy applications. Their non-pathogenic status, low inflammatory potential, availability of viral serotypes with different tissue tropisms, and prospective long-lasting gene expression are important attributes that make rAAVs safe and efficient therapeutic options. Over the last three decades, several groups have engineered recombinant AAV-producing platforms, yielding high titers of transducing vector particles. Current specific productivity yields from different platforms range from 103 to 105 vector genomes (vg) per cell, and there is an ongoing effort to improve vector yields in order to satisfy high product demands required for clinical trials and future commercialization.Crucial aspects of vector production include the molecular design of the rAAV-producing host cell line along with the design of AAV genes, promoters, and regulatory elements. Appropriately, configuring and balancing the expression of these elements not only contributes toward high productivity, it also improves process robustness and product quality. In this mini-review, the rational design of rAAV-producing expression systems is discussed, with special attention to molecular strategies that contribute to high-yielding, biomanufacturing-amenable rAAV production processes. Details on molecular optimization from four rAAV expression systems are covered: adenovirus, herpesvirus, and baculovirus complementation systems, as well as a recently explored yeast expression system.

authors

Aponte-Ubillus JJ,Barajas D,Peltier J,Bardliving C,Shamlou P,Gold D

doi

10.1007/s00253-017-8670-1

subject

Has Abstract

pub_date

2018-02-01 00:00:00

pages

1045-1054

issue

3

eissn

0175-7598

issn

1432-0614

pii

10.1007/s00253-017-8670-1

journal_volume

102

pub_type

杂志文章,评审
  • Spatial and temporal distribution of nitrite-dependent anaerobic methane-oxidizing bacteria in an intertidal zone of the East China Sea.

    abstract::Nitrite-dependent anaerobic methane oxidation (N-DAMO), which couples anaerobic methane oxidation and nitrite reduction, is a recently discovered bioprocess coupling microbial nitrogen and carbon cycles. The discovery of this microbial process challenges the traditional knowledge of global methane sinks and nitrogen l...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-017-8521-0

    authors: Wang J,Shen L,He Z,Hu J,Cai Z,Zheng P,Hu B

    更新日期:2017-11-01 00:00:00

  • Phosphoproteomic investigation of a solvent producing bacterium Clostridium acetobutylicum.

    abstract::In this study, we employed TiO₂ enrichment and high accuracy liquid chromatography-mass spectrometry-mass spectrometry to identify the phosphoproteome of Clostridium acetobutyicum ATCC824 in acidogenesis and solventogenesis. As many as 82 phosphopeptides in 61 proteins, with 107 phosphorylated sites on serine, threoni...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-012-4156-3

    authors: Bai X,Ji Z

    更新日期:2012-07-01 00:00:00

  • Cellular fatty acid profile and H(+)-ATPase activity to assess acid tolerance of Bacillus sp. for potential probiotic functional attributes.

    abstract::The present study has been focused widely on comparative account of probiotic qualities of Bacillus spp. for safer usage. Initially, 170 heat resistant flora were isolated and selected for non-pathogenic cultures devoid of cytK, hblD, and nhe1 virulence genes. Subsequently, through biochemical tests along with 16S rRN...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-014-5981-3

    authors: Shobharani P,Halami PM

    更新日期:2014-11-01 00:00:00

  • Batch cultures of recombinant Lactococcus lactis subsp. lactis in a stirred fermentor. II. Plasmid transfer in mixed cultures.

    abstract::The transfer of plasmids was studied in a stirred fermentor in the course of mixed batch cultures combining recombinant strains of Lactococcus lactis subsp. lactis (donor strains) with L. lactis subsp. lactis CNRZ 268M3 (recipient strain). Donor strains contained one or two of the following plasmids (coding for erythr...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/BF00210993

    authors: el Alami N,Boquien CY,Corrieu G

    更新日期:1992-06-01 00:00:00

  • Silica fertilization and nano-MnO₂ amendment on bacterial community composition in high arsenic paddy soils.

    abstract::Silica fertilization and nano-MnO2 amendment are reported as useful approaches in lowering the accumulation of arsenic in rice grains, but the effects of silica fertilization or nano-MnO2 amendment on microbial community in the paddy soils containing high concentration of arsenic are still unknown. In order to elucida...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-015-7131-y

    authors: Shao J,He Y,Zhang H,Chen A,Lei M,Chen J,Peng L,Gu JD

    更新日期:2016-03-01 00:00:00

  • Plant exudates promote PCB degradation by a rhodococcal rhizobacteria.

    abstract::Rhodococcus erythropolis U23A is a polychlorinated biphenyl (PCB)-degrading bacterium isolated from the rhizosphere of plants grown on a PCB-contaminated soil. Strain U23A bphA exhibited 99% identity with bphA1 of Rhodococcus globerulus P6. We grew Arabidopsis thaliana in a hydroponic axenic system, collected, and con...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-011-3824-z

    authors: Toussaint JP,Pham TT,Barriault D,Sylvestre M

    更新日期:2012-09-01 00:00:00

  • Biodegradation of aromatic compounds: current status and opportunities for biomolecular approaches.

    abstract::Biodegradation can achieve complete and cost-effective elimination of aromatic pollutants through harnessing diverse microbial metabolic processes. Aromatics biodegradation plays an important role in environmental cleanup and has been extensively studied since the inception of biodegradation. These studies, however, a...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章,评审

    doi:10.1007/s00253-009-2192-4

    authors: Cao B,Nagarajan K,Loh KC

    更新日期:2009-11-01 00:00:00

  • Chain transfer reaction catalyzed by various polyhydroxyalkanoate synthases with poly(ethylene glycol) as an exogenous chain transfer agent.

    abstract::Polyhydroxyalkanoate (PHA) synthases catalyze chain transfer (CT) reaction after polymerization reaction of PHA by transferring PHA chain from PHA synthase to a CT agent, resulting in covalent bonding of CT agent to PHA chain at the carboxyl end. Previous studies have shown that poly(ethylene glycol) (PEG) is an effec...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-010-2601-8

    authors: Tomizawa S,Saito Y,Hyakutake M,Nakamura Y,Abe H,Tsuge T

    更新日期:2010-07-01 00:00:00

  • Immunoaffinity layering of enzymes. Stabilization and use in flow injection analysis of glucose and hydrogen peroxide.

    abstract::A general procedure for the high yield immobilization of enzymes with the help of specific anti-enzyme antibodies is described. Polyclonal antibodies were raised against Aspergillus niger glucose oxidase and horseradish peroxidase in rabbits and the gamma globulin (IgG) fraction from the immune sera isolated by ammoni...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s002530051534

    authors: Farooqi M,Sosnitza P,Saleemuddin M,Ulber R,Scheper T

    更新日期:1999-09-01 00:00:00

  • Effect of glucose feeding on the glycosylation quality of antibody produced by a human cell line, F2N78, in fed-batch culture.

    abstract::The human cell line rF2N78 produces an antibody with a high galactosylation ratio which resembles human IgG. However, it has been observed that the aglycosylated antibody starts to appear when glucose is depleted. To determine whether glucose depletion is a main cause for aglycosylation of the antibody, fed-batch cult...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-013-5462-0

    authors: Seo JS,Min BS,Kim YJ,Cho JM,Baek E,Cho MS,Lee GM

    更新日期:2014-04-01 00:00:00

  • Induction of gene expression in bacteria at optimal growth temperatures.

    abstract::Traditional temperature-sensitive systems use either heat shock (40-42 °C) or cold shock (15-23 °C) to induce gene expression at temperatures that are not the optimal temperature for host cell growth (37 °C). This impacts the overall productivity and yield by disturbing cell growth and cellular metabolism. Here, we ha...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-012-4633-8

    authors: Jiang X,Zhang H,Yang J,Liu M,Feng H,Liu X,Cao Y,Feng D,Xian M

    更新日期:2013-06-01 00:00:00

  • Production of trehalose synthase from a basidiomycete, Grifola frondosa, in Escherichia coli.

    abstract::The genomic DNA and cDNA for a gene encoding a novel trehalose synthase (TSase) catalyzing trehalose synthesis from alpha-D-glucose 1-phosphate and D-glucose were cloned from a basidiomycete, Grifola frondosa. Nucleotide sequencing showed that the 732-amino-acid TSase-encoding region was separated by eight introns. Co...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s002530051276

    authors: Saito K,Yamazaki H,Ohnishi Y,Fujimoto S,Takahashi E,Horinouchi S

    更新日期:1998-08-01 00:00:00

  • Cardoon-based rennets for cheese production.

    abstract::The use of crude aqueous extracts of Cynara cardunculus flowers as coagulants in the production of high-quality sheep and goat cheeses-as are the cases of several Portuguese and Spanish cheese varieties with Protected Designation of Origin status-has been maintained since ancient times. The unique rheological attribut...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章,评审

    doi:10.1007/s00253-018-9032-3

    authors: Almeida CM,Simões I

    更新日期:2018-06-01 00:00:00

  • Lytic enzyme discovery through multigenomic sequence analysis in Clostridium perfringens.

    abstract::With their ability to lyse Gram-positive bacteria, phage lytic enzymes (or lysins) have received a great deal of attention as novel anti-infective agents. The number of known genes encoding these peptidoglycan hydrolases has increased markedly in recent years, due in large part to advances in DNA sequencing technology...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-010-2982-8

    authors: Schmitz JE,Ossiprandi MC,Rumah KR,Fischetti VA

    更新日期:2011-03-01 00:00:00

  • Expression and characterization of a thermostable sarcosine oxidase (SOX) from Bacillus sp. in Escherichia coli.

    abstract::A heat-stable sarcosine oxidase produced by Bacillus sp. BSD-8 (SOX) had been studied and its complete gene sequence, which contained 1,164 bp nucleotides and encoded a protein of 387 amino acids, was obtained by DNA Walking method. The sox gene was cloned and functionally overexpressed in E. coli and the recombinant ...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-006-0502-7

    authors: Guo K,Ma X,Sun G,Zhao Y,Li X,Zhao W,Kai L

    更新日期:2006-12-01 00:00:00

  • Biosynthesis of mono-acylated mannosylerythritol lipid in an acyltransferase gene-disrupted mutant of Pseudozyma tsukubaensis.

    abstract::The basidiomycetous yeast genus Pseudozyma produce large amounts of mannosylerythritol lipids (MELs), which are biosurfactants. A few Pseudozyma strains produce mono-acylated MEL as a minor compound using excess glucose as the sole carbon source. Mono-acylated MEL shows higher hydrophilicity than di-acylated MEL and h...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-017-8698-2

    authors: Saika A,Utashima Y,Koike H,Yamamoto S,Kishimoto T,Fukuoka T,Morita T

    更新日期:2018-02-01 00:00:00

  • Biochemical characterization of Magnaporthe oryzae β-glucosidases for efficient β-glucan hydrolysis.

    abstract::β-Glucosidases designated MoCel3A and MoCel3B were successfully overexpressed in Magnaporthe oryzae. MoCel3A and MoCel3B showed optimal activity at 50 °C and pH 5.0-5.5. MoCel3A exhibited higher activity on higher degree of polymerization (DP) oligosaccharides and on β-1,3-linked oligosaccharides than on β-1,4-linked ...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-011-3340-1

    authors: Takahashi M,Konishi T,Takeda T

    更新日期:2011-08-01 00:00:00

  • On-line infrared spectroscopy for bioprocess monitoring.

    abstract::One of the major aims of bioprocess engineering is the real-time monitoring of important process variables. This is the basis of precise process control and is essential for high productivity as well as the exact documentation of the overall production process. Infrared spectroscopy is a powerful analytical technique ...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章,评审

    doi:10.1007/s00253-010-2743-8

    authors: Landgrebe D,Haake C,Höpfner T,Beutel S,Hitzmann B,Scheper T,Rhiel M,Reardon KF

    更新日期:2010-09-01 00:00:00

  • The cellulase system of the anaerobic rumen fungus Neocallimastix frontalis: studies on the properties of fractions rich in endo-(1-->4)-beta-D-glucanase activity.

    abstract::Seven fractions rich in endoglucanase activity were separated from the extracellular cellulase system of the anaerobic rumen fungus Neocallimastix frontalis. The fractions (ES1, ES3, ES2U1, ES2U2, ES2U4, ES2U3C1 and ES2U3C2) were separated from each other and from a fraction that could solubilize crystalline cellulose...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/BF00164499

    authors: Wood TM,Wilson CA,McCrae SI

    更新日期:1995-12-01 00:00:00

  • Microbial transformation of benzene to cis-3,5-cyclohexadien-1,2-diols by recombinant bacteria harboring toluene dioxygenase gene tod.

    abstract::Toluene dioxygenase (TDO) catalyzes asymmetric cis-dihydroxylation of aromatic compounds. To achieve high efficient biotransformation of benzene to benzene cis-diols, Pseudomonas putida KT2442, Pseudomonas stutzeri 1317, and Aeromonas hydrophila 4AK4 were used as hosts to express TDO gene tod. Plasmid pSPM01, a deriva...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-006-0637-6

    authors: Ouyang SP,Sun SY,Liu Q,Chen J,Chen GQ

    更新日期:2007-02-01 00:00:00

  • Biology and biotechnology of Trichoderma.

    abstract::Fungi of the genus Trichoderma are soilborne, green-spored ascomycetes that can be found all over the world. They have been studied with respect to various characteristics and applications and are known as successful colonizers of their habitats, efficiently fighting their competitors. Once established, they launch th...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章,评审

    doi:10.1007/s00253-010-2632-1

    authors: Schuster A,Schmoll M

    更新日期:2010-07-01 00:00:00

  • Bioresolution of (R)-glycidyl azide by Aspergillus niger ZJUTZQ208: a new and concise synthon for chiral vicinal amino alcohols.

    abstract::A newly isolated Aspergillus niger strain containing epoxide hydrolase was used to resolve racemic glycidyl azide and four derivatives to the (R)-enantiomers. After optimization of the biotransformation conditions, (R)-glycidyl azide was produced with good enantioselectivity (e.e.s > 95 %, E > 20). The substrate struc...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-012-4382-8

    authors: Chen L,Shen H,Wei C,Zhu Q

    更新日期:2013-03-01 00:00:00

  • Astaxanthin production by Phaffia rhodozyma and Haematococcus pluvialis: a comparative study.

    abstract::Phaffia rhodozyma (now Xanthophyllomyces dendrorhous) and Haematococcus pluvialis are known as the major prominent microorganisms able to synthesize astaxanthin natural pigment. Important research efforts have been made to determine optimal conditions for astaxanthin synthesis. When the focus is on astaxanthin product...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-007-0889-9

    authors: Domínguez-Bocanegra AR,Ponce-Noyola T,Torres-Muñoz JA

    更新日期:2007-06-01 00:00:00

  • Opportunities for broadening the application of cell wall lytic enzymes.

    abstract::In light of emerging antibiotic resistance, bacterial cell wall lytic enzymes are promising antimicrobial agents that degrade bacterial peptidoglycan while specifically recognizing the target bacterium. The efficacy of lytic enzymes against several multi-drug-resistant pathogens infecting humans has led to many effort...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章,评审

    doi:10.1007/s00253-020-10862-y

    authors: Bhagwat A,Mixon M,Collins CH,Dordick JS

    更新日期:2020-11-01 00:00:00

  • Characterization of two distinct feruloyl esterases, AoFaeB and AoFaeC, from Aspergillus oryzae.

    abstract::Two hypothetical proteins XP_001818628 and XP_001819091 (designated AoFaeB and AoFaeC, respectively), showing sequence identity with known type-C feruloyl esterases, have been found in the genomic sequence of Aspergillus oryzae. We cloned the putative A. oryzae feruloyl esterase-encoding genes and expressed them in Pi...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-009-1913-z

    authors: Koseki T,Hori A,Seki S,Murayama T,Shiono Y

    更新日期:2009-06-01 00:00:00

  • Molecular cloning and characterization of a GH11 endoxylanase from Chaetomium globosum, and its use in enzymatic pretreatment of biomass.

    abstract::An endo-1,4-β-xylanase gene, xylcg, was cloned from Chaetomium globosum and successfully expressed in Escherichia coli. The complete gene of 675 bp was amplified, cloned into the pET 28(a) vector, and expressed. The optimal conditions for the highest activity of the purified recombinant XylCg were observed at a temper...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-012-4577-z

    authors: Singh RK,Tiwari MK,Kim D,Kang YC,Ramachandran P,Lee JK

    更新日期:2013-08-01 00:00:00

  • Acid phosphatase production by recombinant Arxula adeninivorans.

    abstract::Acid phosphatase production by recombinant Arxula adeninivorans was carried out in submerged fermentation. Using the Plackett-Burman design, three fermentation variables (pH, sucrose concentration, and peptone concentration) were identified to significantly affect acid phosphatase and biomass production, and these wer...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-007-1021-x

    authors: Minocha N,Kaur P,Satyanarayana T,Kunze G

    更新日期:2007-08-01 00:00:00

  • Peptidoglycan turnover and recycling in Gram-positive bacteria.

    abstract::Bacterial cells are protected by an exoskeleton, the stabilizing and shape-maintaining cell wall, consisting of the complex macromolecule peptidoglycan. In view of its function, it could be assumed that the cell wall is a static structure. In truth, however, it is steadily broken down by peptidoglycan-cleaving enzymes...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章,评审

    doi:10.1007/s00253-011-3486-x

    authors: Reith J,Mayer C

    更新日期:2011-10-01 00:00:00

  • A novel E. coli biosensor for detecting aromatic aldehydes based on a responsive inducible archaeal promoter fused to the green fluorescent protein.

    abstract::A whole-cell bacterial biosensor for measuring aqueous concentrations of aromatic aldehydes was developed. It is based on the E. coli BL21DE3(RIL) expressing the green fluorescent protein under the control of an alcohol dehydrogenase inducible promoter belonging to the archaeon Sulfolobus solfataricus (Sso2536adh prom...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/s00253-008-1771-0

    authors: Fiorentino G,Ronca R,Bartolucci S

    更新日期:2009-02-01 00:00:00

  • Expression of Streptomyces genes encoding extracellular enzymes in Brevibacterium lactofermentum: secretion proceeds by removal of the same leader peptide as in Streptomyces lividans.

    abstract::The alpha-amylase gene (amy) from Streptomyces griseus IMRU 3570 and the beta-galactosidase gene (lac) from S. lividans were subcloned into Brevibacterium lactofermentum or B. lactofermentum/Escherichia coli shuttle vectors. The amy gene was not expressed in B. lactofermentum from its own promoter but was efficiently ...

    journal_title:Applied microbiology and biotechnology

    pub_type: 杂志文章

    doi:10.1007/BF00170087

    authors: Cadenas RF,Gil JA,Martín JF

    更新日期:1992-12-01 00:00:00