نتایج جستجو برای: aav base vector

تعداد نتایج: 451713  

CRISPR/Cas9 system is a powerful gene editing tool in vivo and in vitro. Currently, CRISPR/Cas9 delivery cells or tissue with different vehicles are available, and Adeno- associated virus (AAV) in one of them. Due to AAV packaging size limitation, AAV base vectors that carry CRISPR/Cas9 system do not have florescent tag like GFP for simple detection and navigation of cells, containing AAV. The ...

Journal: :The Journal of biological chemistry 1993
T R Flotte S A Afione R Solow M L Drumm D Markakis W B Guggino P L Zeitlin B J Carter

Adeno-associated virus type 2 (AAV) vectors have been used for gene expression in respiratory epithelial cells and may be useful in gene therapy for diseases like cystic fibrosis (CF) which affect the airways. The AAV p5 promoter together with the AAV inverted terminal repeat (ITR) forms a 263-base pair cassette which mediated efficient expression in a CF bronchial epithelial cell line. We repo...

Journal: :Journal of virology 1988
S K McLaughlin P Collis P L Hermonat N Muzyczka

We used two kinds of adeno-associated virus (AAV) vectors to transduce the neomycin resistance gene into human cells. The first of these (dl52-91) retains the AAV rep genes; the second (dl3-94) retains only the AAV terminal repeats and the AAV polyadenylation signal (428 base pairs). Both vectors could be packaged into AAV virions and produced proviral structures that were essentially the same....

2017
Qizhao Wang Biao Dong Katie A. Pokiniewski Jenni Firrman Zhongren Wu Mario P.S. Chin Xiongwen Chen LinShu Liu Ruian Xu Yong Diao Weidong Xiao

Adeno-associated virus (AAV) vectors have emerged as a safe and efficient gene therapy platform. One complication is that a significant amount of empty particles have always been generated as impurities during AAV vector production. However, the effects of such particles on AAV vector performance remain unclear. Here we systemically evaluated the biological properties of three types of "empty" ...

Journal: :Journal of virology 1999
D Duan P Sharma L Dudus Y Zhang S Sanlioglu Z Yan Y Yue Y Ye R Lester J Yang K J Fisher J F Engelhardt

A central feature of the adeno-associated virus (AAV) latent life cycle is persistence in the form of both integrated and episomal genomes. However, the molecular processes associated with episomal long-term persistence of AAV genomes are only poorly understood. To investigate these mechanisms, we have utilized a recombinant AAV (rAAV) shuttle vector to identify circular AAV intermediates from ...

Journal: :Journal of virology 2002
Chunping Qiao Juan Li Anna Skold Xudong Zhang Xiao Xiao

The adeno-associated virus (AAV) vector system is based on nonpathogenic and helper-virus-dependent parvoviruses. The vector system offers safe, efficient, and long-term in vivo gene transfer in numerous tissues. Clinical trials using AAV vectors have demonstrated vector safety as well as efficiency. The increasing interest in the use of AAV for clinical studies demands large quantities of vect...

2017
Jaerin Sohn Megumu Takahashi Shinichiro Okamoto Yoko Ishida Takahiro Furuta Hiroyuki Hioki

Visualization of neurons is indispensable for the investigation of neuronal circuits in the central nervous system. Virus vectors have been widely used for labeling particular subsets of neurons, and the adeno-associated virus (AAV) vector has gained popularity as a tool for gene transfer. Here, we developed a single AAV vector Tet-Off platform, AAV-SynTetOff, to improve the gene-transduction e...

Journal: :Journal of virology 1997
J M Allen D J Debelak T C Reynolds A D Miller

Adeno-associated virus (AAV) vector preparations are often contaminated with variable amounts of replication-competent AAV (rcAAV), which may influence the behavior of these vectors both in cultured cells and in animals. A packaging plasmid/vector plasmid system containing no significant homology and lacking the wild-type AAV p5 promoter was constructed to eliminate the production of wild-type ...

2001
LINGFEI XU THOMAS DALY CUIHUA GAO TERENCE R. FLOTTE SIHONG SONG BARRY J. BYRNE MARK S. SANDS

Although AAV vectors show promise for hepatic gene therapy, the optimal transcriptional regulatory elements have not yet been identified. In this study, we show that an AAV vector with the CMV enhancer/chicken b -actin promoter results in 9.5-fold higher expression after portal vein injection than an AAV vector with the EF1a promoter, and 137-fold higher expression than an AAV vector with the C...

Journal: :Molecular therapy : the journal of the American Society of Gene Therapy 2012
Aravind Asokan David V Schaffer R Jude Samulski

The discovery of naturally occurring adeno-associated virus (AAV) isolates in different animal species and the generation of engineered AAV strains using molecular genetics tools have yielded a versatile AAV vector toolkit. Promising results in preclinical animal models of human disease spurred the much awaited transition toward clinical application, and early successes in phase I/II clinical t...

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