scholarly journals Immunological Ignorance Allows Long-Term Gene Expression After Perinatal Recombinant Adeno-Associated Virus-Mediated Gene Transfer to Murine Airways

2014 ◽  
Vol 25 (6) ◽  
pp. 517-528 ◽  
Author(s):  
Marianne S. Carlon ◽  
Dragana Vidović ◽  
James Dooley ◽  
Marina Mori da Cunha ◽  
Michael Maris ◽  
...  
Keyword(s):  
Gene Expression ◽  
Gene Transfer ◽  
Adeno Associated Virus

Adeno-Associated Viral Vectors for Gene Transfer and Gene Therapy

1999 ◽  
Vol 380 (6) ◽  
Author(s):  
H. Büeler
Keyword(s):  
Gene Expression ◽  
Gene Therapy ◽  
Gene Transfer ◽  
Gene Delivery ◽  
Viral Vectors ◽  
Delivery Systems ◽  
Adeno Associated Virus ◽  
Viral Genes ◽  
Virus Recombinant

AbstractAdeno-associated virus (AAV) is a defective, non-pathogenic human parvovirus that depends for growth on coinfection with a helper adenovirus or herpes virus. Recombinant adeno-associated viruses (rAAVs) have attracted considerable interest as vectors for gene therapy. In contrast to other gene delivery systems, rAAVs lack all viral genes and show long-term gene expression

scholarly journals Inducible long-term gene expression in brain with adeno-associated virus gene transfer

Gene Therapy ◽  
1998 ◽  
Vol 5 (12) ◽  
pp. 1604-1611 ◽  
Author(s):  
RP Haberman ◽  
TJ McCown ◽  
RJ Samulski
Keyword(s):  
Gene Expression ◽  
Gene Transfer ◽  
Adeno Associated Virus ◽  
Virus Gene

Long-Term Inducible Gene Expression in the Eye via Adeno-Associated Virus Gene Transfer in Nonhuman Primates

2005 ◽  
Vol 16 (2) ◽  
pp. 178-186 ◽  
Author(s):  
Corinna Lebherz ◽  
Alberto Auricchio ◽  
Albert M. Maguire ◽  
Victor M. Rivera ◽  
Waixing Tang ◽  
...  
Keyword(s):  
Gene Expression ◽  
Gene Transfer ◽  
Nonhuman Primates ◽  
Inducible Gene Expression ◽  
Adeno Associated Virus ◽  
Virus Gene ◽  
Inducible Gene

Long-Term Gene Transfer in Porcine Myocardium After Coronary Infusion of an Adeno-Associated Virus Vector

1996 ◽  
Vol 62 (6) ◽  
pp. 1669-1676 ◽  
Author(s):  
Michael G Kaplitt ◽  
Xiao Xiao ◽  
Richard J Samulski ◽  
Juan Li ◽  
Kaie Ojamaa ◽  
...  
Keyword(s):  
Gene Transfer ◽  
Virus Vector ◽  
Adeno Associated Virus

scholarly journals Breaching the Delivery Barrier: Chemical and Physical Airway Epithelium Disruption Strategies for Enhancing Lentiviral-Mediated Gene Therapy

2021 ◽  
Vol 12 ◽  
Author(s):  
Alexandra McCarron ◽  
Nigel Farrow ◽  
Patricia Cmielewski ◽  
Emma Knight ◽  
Martin Donnelley ◽  
...  
Keyword(s):  
Gene Expression ◽  
Gene Transfer ◽  
Barrier Properties ◽  
Airway Epithelial Cells ◽  
Viral Gene ◽  
Airway Epithelial ◽  
Expression Levels ◽  
Epithelial Disruption ◽  
Gene Expression Levels

The lungs have evolved complex physical, biological and immunological defences to prevent foreign material from entering the airway epithelial cells. These mechanisms can also affect both viral and non-viral gene transfer agents, and significantly diminish the effectiveness of airway gene-addition therapies. One strategy to overcome the physical barrier properties of the airway is to transiently disturb the integrity of the epithelium prior to delivery of the gene transfer vector. In this study, chemical (lysophosphatidylcholine, LPC) and physical epithelium disruption using wire abrasion were compared for their ability to improve airway-based lentiviral (LV) vector mediated transduction and reporter gene expression in rats. When luciferase expression was assessed at 1-week post LV delivery, LPC airway conditioning significantly enhanced gene expression levels in rat lungs, while a long-term assessment in a separate cohort of rats at 12 months revealed that LPC conditioning did not improve gene expression longevity. In rats receiving physical perturbation to the trachea prior to gene delivery, significantly higher LacZ gene expression levels were found when compared to LPC-conditioned or LV-only control rats when evaluated 1-week post gene transfer. This proof-of-principle study has shown that airway epithelial disruption strategies based on physical perturbation substantially enhanced LV-mediated airway gene transfer in the trachea.

Efficient and sustained gene expression in primary T lymphocytes and primary and cultured tumor cells mediated by adeno-associated virus plasmid DNA complexed to cationic liposomes

1994 ◽  
Vol 14 (4) ◽  
pp. 2411-2418
Author(s):  
R Philip ◽  
E Brunette ◽  
L Kilinski ◽  
D Murugesh ◽  
M A McNally ◽  
...  
Keyword(s):  
Gene Expression ◽  
Tumor Cells ◽  
Plasmid Dna ◽  
Interleukin 2 ◽  
Cationic Liposomes ◽  
Lymphoid Cells ◽  
Chromosomal Dna ◽  
Adeno Associated Virus ◽  
Cultured Tumor Cells

We have used cationic liposomes to facilitate adeno-associated virus (AAV) plasmid transfections of primary and cultured cell types. AAV plasmid DNA complexed with liposomes showed levels of expression several fold higher than those of complexes with standard plasmids. In addition, long-term expression (> 30 days) of the gene, unlike the transient expression demonstrated by typical liposome-mediated transfection with standard plasmids, was observed. Southern analysis of chromosomal DNA further substantiated the hypothesis that the long-term expression was due to the presence of the transgene in the AAV plasmid-transfected group and not in the standard plasmid-transfected group. AAV plasmid-liposome complexes induced levels of transgene expression comparable to those obtained by recombinant AAV transduction. Primary breast, ovarian, and lung tumor cells were transfectable with the AAV plasmid DNA-liposome complexes. Transfected primary and cultured tumor cells were able to express transgene product even after lethal irradiation. High-level gene expression was also observed in freshly isolated CD3+, CD4+, and CD8+ T cells from normal human peripheral blood. Transfection efficiency ranged from 10 to 50% as assessed by intracellular interleukin-2 levels in interleukin-2-transfected cells. The ability to express transgenes in primary tumor and lymphoid cells may be applied toward tumor vaccine studies and protocols which may eventually permit highly specific modulation of the cellular immune response in cancer and AIDS.

scholarly journals Long-Term Enzymatic and Phenotypic Correction in the Phenylketonuria Mouse Model by Adeno-Associated Virus Vector-Mediated Gene Transfer

2004 ◽  
Vol 56 (2) ◽  
pp. 278-284 ◽  
Author(s):  
Hyun-Jeong Oh ◽  
Eun-Sook Park ◽  
Seongman Kang ◽  
Inho Jo ◽  
Sung-Chul Jung
Keyword(s):  
Gene Transfer ◽  
Mouse Model ◽  
Virus Vector ◽  
Adeno Associated Virus

scholarly journals Efficient and long-term intracardiac gene transfer in δ-sarcoglycan-deficiency hamster by adeno-associated virus-2 vectors

Gene Therapy ◽  
2003 ◽  
Vol 10 (21) ◽  
pp. 1807-1813 ◽  
Author(s):  
J Li ◽  
D Wang ◽  
S Qian ◽  
Z Chen ◽  
T Zhu ◽  
...  
Keyword(s):  
Gene Transfer ◽  
Adeno Associated Virus
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