Abstract:
:Microwave (MW) energy consists of electric and magnetic fields and is able to penetrate deep into biological materials. We investigated the effect of MW (2450 MHz) irradiation on gene delivery in cultured mouse myoblasts and observed enhanced transgene expression. This effect is, however, highly variable and critically dependent on the power levels, duration and cycle conditions of MW exposure. MW irradiation greatly enhances delivery of 2'O methyl-phosphorothioate antisense oligonucleotide (AON) targeting mouse dystrophin exon 23 and induces specific exon skipping in cultured myoblasts. Effective delivery of AON by MW irradiation is able to correct the dystrophin reading frame disrupted by a nonsense point mutation in the H2K mdx myoblasts, resulting in the restoration of dystrophin expression. MW-mediated nucleic acid delivery does not directly link to the increase in system temperature. The high variability in gene and oligonucleotide delivery is most likely the result of considerable irregularity in the distribution of the energy and magnetic field produced by MW with the current device. Therefore, achieving effective delivery of the therapeutic molecules would require new designs of MW devices capable of providing controllable and evenly distributed energy for homogenous exposure of the target cells.
journal_name
Gene Therjournal_title
Gene therapyauthors
Doran TJ,Lu PJ,Vanier GS,Collins MJ,Wu B,Lu QLdoi
10.1038/gt.2008.144subject
Has Abstractpub_date
2009-01-01 00:00:00pages
119-26issue
1eissn
0969-7128issn
1476-5462pii
gt2008144journal_volume
16pub_type
杂志文章相关文献
GENE THERAPY文献大全abstract::We have developed and tested a transfection compound based on synthetic peptides. It consists of a 12 amino acid DNA binding peptide (P2) with an alkyl group added to the aminoterminus (P2lip) and a peptide derived from the hemagglutinin protein (HA). The components aggregate spontaneously to particles that proved to ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:
更新日期:1996-12-01 00:00:00
abstract::Virus-directed enzyme prodrug therapy utilizing the bacterial enzyme nitroreductase delivered by a replication-defective adenovirus vector to activate the prodrug CB1954 is a promising strategy currently undergoing clinical trials in patients with a range of cancers. Similarly, selectively replicating oncolytic adenov...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3302510
更新日期:2005-08-01 00:00:00
abstract::Prostate cancer is the most common cancer in men of the western world. To date, no effective treatment exists for metastatic prostate cancer and consequently, there is an urgent need to develop new and improved therapeutics. In recent years, the therapeutic potential of RNA interference (RNAi) has been extensively exp...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2015.50
更新日期:2015-10-01 00:00:00
abstract::Gene targeting by single-stranded oligodeoxyribonucleotides (ssODNs) is a promising technique for introducing site-specific sequence alterations without affecting the genomic organization of the target locus. Here, we discuss the significant progress that has been made over the last 5 years in unraveling the mechanism...
journal_title:Gene therapy
pub_type: 杂志文章,评审
doi:10.1038/gt.2010.161
更新日期:2011-03-01 00:00:00
abstract::The number of clinical trials using gene transfer technology, either active or under discussion, is increasing rapidly. However, little information is available describing the regulatory procedures or safety specifications that must be considered before initiation of such trials in Europe. We describe the procedure us...
journal_title:Gene therapy
pub_type: 杂志文章,评审
doi:
更新日期:1994-03-01 00:00:00
abstract::Lentivirus can be engineered to be a highly potent vector for gene therapy applications. However, generation of clinical grade vectors in enough quantities for therapeutic use is still troublesome and limits the preclinical and clinical experiments. As a first step to solve this unmet need we recently introduced a bac...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.162
更新日期:2011-06-01 00:00:00
abstract::Production of retroviral vectors for clinical use requires removal of cells and cellular debris. We combined a series of filters of decreasing pore size using commercially available blood banking filters approved for clinical use. The collection bag and filters can be connected to create a sterile, closed system using...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301328
更新日期:2000-12-01 00:00:00
abstract::RNA interference (RNAi) is an evolutionarily conserved process by which plants and animals protect their genomes utilizing small, double-stranded RNAs to degrade target RNAs in a sequence-specific manner. Post-transcriptional gene silencing by these moieties can lead to degradation of both cellular and viral RNAs. It ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3302339
更新日期:2004-12-01 00:00:00
abstract::The mechanism whereby cationic lipids destabilize cell membranes to facilitate the intracellular delivery of macromolecules such as plasmid DNA or antisense oligonucleotides is not well understood. Here, we show that cationic lipids can destabilize lipid bilayers by promoting the formation of nonbilayer lipid structur...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301506
更新日期:2001-08-01 00:00:00
abstract::Current generation adenovirus (Ad) vectors are deleted for the E1 region of genes and require propagation in E1 expressing 293 cells. Expression of genes delivered by Ad vectors into immunocompetent hosts is generally transient since the current vectors are not completely replication defective. Viral proteins expresse...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3300378
更新日期:1997-03-01 00:00:00
abstract::We constructed a melanoma-specific oncolytic adenoviral vector Ad.MCDIRESE1.71Hsp3, in which the cytosine deaminase and adenoviral E1A genes linked by the IRES sequence were under the control of a mouse tyrosinase enhancer/promoter transcriptional element in the E1 region of the vector. We also inserted the human heat...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3302776
更新日期:2006-08-01 00:00:00
abstract::Episomally maintained self-replicating systems present attractive alternative vehicles for gene therapy applications. Recent insights into the ability of chromosomal scaffold/matrix attachment regions (S/MARs) to mediate episomal maintenance of genetic elements allowed the development of a small circular episomal vect...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3302593
更新日期:2006-01-01 00:00:00
abstract::Ideal methods for human gene therapy will eventually include direct gene transfer to defective tissues in a patient in vivo. Toward that goal, we have used high titer, pseudotyped retroviral vectors expressing genes for the Escherichia coli beta-galactosidase (lacZ) or hepatitis B virus surface antigen (HBsAg) to infe...
journal_title:Gene therapy
pub_type: 杂志文章
doi:
更新日期:1995-03-01 00:00:00
abstract::Schwann cells (SCs) in an injured peripheral nerve form pathways for regenerating axons. Although these cells initially support regeneration, SCs lose their pro-regenerative properties following a prolonged period of denervation. Gene transfer to SC can enhance their therapeutic potential. In this article, we compared...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2015.47
更新日期:2015-10-01 00:00:00
abstract::In vivo approaches to liver gene therapy will require restriction of transgene expression to hepatocytes. Since targeting of viral vectors exclusively to the liver is not easy to achieve, use of liver-specific promoters for driving expression of therapeutic genes is an interesting alternative. We have shown previously...
journal_title:Gene therapy
pub_type: 杂志文章
doi:
更新日期:1996-11-01 00:00:00
abstract::Duchenne muscular dystrophy (DMD) is an X-linked, lethal muscular disorder caused by a defect in the DMD gene. AAV vector-mediated micro-dystrophin cDNA transfer is an attractive approach to treatment of DMD. To establish effective gene transfer into skeletal muscle, we examined the transduction efficiency of an AAV v...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301829
更新日期:2002-12-01 00:00:00
abstract::Choroidal neovascularization (CNV) leads to loss of vision in age-related macular degeneration (AMD), the leading cause of blindness in adult population over 50 years old. In this study, we developed intravenously administered, nanoparticulate, targeted nonviral retinal gene delivery systems for the management of CNV....
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2008.185
更新日期:2009-05-01 00:00:00
abstract::To confer adenovirus vectors (AdV), the feature of integration into the host cell genome hybrid vectors were characterized in vitro, which express vectors derived from the prototypic foamy virus (FV) in the backbone of a high-capacity AdV. FVs constitute a subfamily of retroviruses with a distinct replication pathway ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3302216
更新日期:2004-04-01 00:00:00
abstract::Chemically inducible gene switches that regulate expression of endogenous genes have multiple applications for basic gene expression research and gene therapy. Single-chain zinc-finger transcription factors that utilize either estrogen receptor homodimers or retinoid X receptor-alpha/ecdysone receptor heterodimers are...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2008.96
更新日期:2008-09-01 00:00:00
abstract::Chimeric antigen receptor (CAR) T-cell therapies have demonstrated durable and potentially curative therapeutic efficacy against B-cell leukemia in clinical trials. A CAR strategy can target any tumor surface antigens as long as an antigen-binding receptor can be generated. New CARs that target solid tumors and have t...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2015.29
更新日期:2015-08-01 00:00:00
abstract::Gene therapy is a potential new strategy for the treatment of cardiovascular disease. The most efficacious method of gene delivery remains a key hurdle to effective gene therapy. We present the application of a novel, nonviral gene delivery system (TerplexDNA) to augment myocardial transfection. The hearts of New Zeal...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301395
更新日期:2001-03-01 00:00:00
abstract::There is currently an urgent need to develop efficient gene-delivery systems for the lung that are free of inflammatory effects. The LID vector is a synthetic gene delivery system, comprised of lipofectin (L), an integrin-targeting peptide (I) and DNA (D) that has previously been shown to have high transfection effici...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301963
更新日期:2003-06-01 00:00:00
abstract::The efficient delivery of genetic material to the developing fetal brain represents a powerful research tool and a means to supply therapy in a number of neonatal lethal neurological disorders. In this study, we have delivered vectors based upon adenovirus serotype 5 (Ad5) and adeno-associated virus (AAV) pseudotypes ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2011.157
更新日期:2012-09-01 00:00:00
abstract::Gene transfer to airway epithelia with amphotropic pseudotyped retroviral vectors is inefficient following apical vector application. To better understand this inefficiency, we localized the expression of Pit2, the amphotropic receptor, in polarized human airway epithelia. Pit2 was expressed on both the apical and bas...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301714
更新日期:2002-07-01 00:00:00
abstract::The ability to restrict gene delivery and expression to particular cell types is of paramount importance for many types of gene therapy, especially in the lung. The alveolar epithelial type I (ATI) cell, in particular, is an attractive cell type to target, as it comprises 95% of the internal surface area of the lung. ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2016.52
更新日期:2016-10-01 00:00:00
abstract::Rescue of dystrophic skeletal muscle in mdx and utrophin/dystrophin-deficient (dko) mouse models by reintroduction of dystrophin has validated gene therapy as a potential therapeutic approach for Duchenne muscular dystrophy. However, the size of the dystrophin gene exceeds the capacity of adeno-associated viral (AAV) ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3302686
更新日期:2006-05-01 00:00:00
abstract::Human adipose tissue mesenchymal stromal cells (AMSCs) share common traits, including similar differentiation potential and cell surface markers, with their bone marrow counterparts. Owing to their general availability, higher abundance and ease of isolation AMSCs may be convenient autologous delivery vehicles for loc...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2008.176
更新日期:2009-04-01 00:00:00
abstract::The CMV promoter drives high transgene expression and is one of the most commonly used promoters for gene transfer. Tissue-specific mammalian promoters provide an alternative, and it would be useful to have a system to directly compare them to viral promoters free from potential confounding vector-related effects. In ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3302700
更新日期:2006-05-01 00:00:00
abstract::The role of two receptors, previously proposed to mediate the entry of adenoviruses into human cells, the coxsackie and adenovirus receptor (CAR) and the major histocompatibility complex (MHC) class I heavy chain has been investigated. The expression of MHC class I in many tumours is reduced or absent, therefore if th...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301006
更新日期:1999-09-01 00:00:00
abstract::Rhesus macaque tripartite motif (TRIM)5alpha potently inhibits early stages of human immunodeficiency virus (HIV)-1 replication, while the human orthologue has little effect on this virus. We used PCR-based random mutagenesis to construct a large library of human TRIM5alpha variants containing mutations in the PRYSPRY...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.40
更新日期:2010-07-01 00:00:00