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Welcome to the Pubget Medical Subject Headings (MESH) browser. Click on a topic or subtopic below to explore related papers. In the gold box is the most recent paper about the current MESH term, and below are 20 related papers.
Targeted Gene Repair (0):
Recent article
Targeted Gene Repair
Molecular Therapy (18)6 2010
Abstract
We created a mouse model of a generic recessive genetic disease to establish a preclinical system to develop the use of ZFN-mediated gene correction for gene therapy. We knocked a mutated GFP gene into the ROSA26 locus in murine embryonic stem (ES) cells and used these cells to create a transgenic m...
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PMID: 20389291
PDF is available here.
PDF is available here.
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Abstract
We show long-term splice correction of the DMD gene in mdx mice following intramuscular PNA delivery and effective splice correction in aged mdx mice. Further, we report detailed optimization of systemic PNA delivery dose regimens and PNA AO lengths to yield splice correction, with 25-mer PNA AOs pr...
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PMID: 20068555
PDF is available here.
PDF is available here.
Abstract
Correcting a mutated gene directly at its endogenous locus represents an alternative to gene therapy protocols based on viral vectors with their risk of insertional mutagenesis. When solely a single-stranded oligodeoxynucleotide (ssODN) is used as a repair matrix, the efficiency of the targeted gene...
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PMID: 20068556
PDF is available here.
PDF is available here.
Current Gene Therapy (9)4 2009
Abstract
Primary immunodeficiencies (PID) are caused by mutations in genes that impair the development or activity of the immune system. Although bone marrow transplants achieve long time restoration in up to 90% of treated patients, morbidity and mortality are still high for some PID and ade...
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PMID: 19534652
PDF is available here.
PDF is available here.
Abstract
NF-kappaB decoy ODNs may exert anti-inflammatory effects in ectopic endometrial stromal cells via the suppression of NF-kappaB activation, RANTES expression, and monocyte chemotactic activity....
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PMID: 19172384
PDF is available here.
PDF is available here.
Gene Therapy (16)6 2009
Abstract
We describe experiments designed to meet stringent criteria and completely eliminate artefactual results. In particular, by targeting cells expressing mutated enhanced green fluorescence protein (EGFP), which allow editing measurements at the protein level, and analyzing corrected clones by Southern...
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PMID: 19340016
PDF is available here.
PDF is available here.
DNA Repair (8)3 2009
Abstract
We demonstrate that after ssODN mediated gene correction, phosphorylation of the damage sensor protein H2AX could be observed in 5.8% and 29% of the corrected cells, using a single copy and a multi copy reporter, respectively. When using the ZFN strategy in a single copy reporter only 1.5% of the co...
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PMID: 19071233
PDF is available here.
PDF is available here.
Abstract
We discuss how the impact of the cell on TGA has evolved through the years and illustrate ways that inherent cellular pathways could be used to enhance TGA activity. We also describe the cost to cell metabolism and survival when certain processes are altered to achieve a higher frequency of repair....
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PMID: 19204988
PDF is available here.
PDF is available here.
BMC Molecular Biology (10)1 2009
Abstract
Single-stranded DNA oligonucleotides that were designed to repair this splice site mutation corrected the mutation in the gene and restored expression of wild-type dystrophin. This repair was validated at the DNA, RNA and protein level. We also report that the frequency of genetic repair of the mdx...
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PMID: 19236710
PDF is available here.
PDF is available here.
Abstract
Cystic Fibrosis (CF) is an autosomal recessive disorder due to mutations in the CF transmembrane conductance regulator (CFTR) gene that lead to defective ion transport in the conducting pulmonary airways and exocrine glands. Through a process that is not fully understood, CFTR defects predispose aff...
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PMID: 18600482
PDF is available here.
PDF is available here.
BioTechniques (45)6 2008
Vivo-Morpholinos: a non-peptide transporter delivers Morpholinos into a wide array of mouse tissues.
Abstract
We have developed a new transporter structure that provides effective delivery of Morpholino antisense oligomers into a wide variety of tissues in living mice. This transporter comprises a dendritic structure assembled around a triazine core which serves to position eight guanidinium head groups in...
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PMID: 19238792
PDF is available here.
PDF is available here.
Abstract
This review is to highlight the most current mutation-targeted therapeutic approaches and provide insights into new developments for treating primary immunodeficiencies.
Significant progress in mutation-targeted treatment was achieved in the past year with the identif...
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PMID: 18978469
PDF is available here.
PDF is available here.
Abstract
Biochemistry (Washington) (47)33 2008
Abstract
A 606-base single-stranded (ss) DNA fragment, prepared by restriction enzyme digestion of ss phagemid DNA, corrects a hygromycin resistance and enhanced green fluorescent protein (Hyg-EGFP) fusion gene more efficiently than a PCR fragment, which is the conventional type of DNA fragment used in gene...
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PMID: 18642931
PDF is available here.
PDF is available here.
Progrès en Urologie (18)7 2008
Abstract
Cancer is a complex disease characterized by a multitude of molecular and genetic abnormalities affecting cell proliferation and differentiation, apoptosis, and mobility (invasion). Each of these alterations represents a potential target for the development of targeted therapy. These new therapies i...
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PMID: 18602599
PDF is available here.
PDF is available here.
Gene Therapy (15)14 2008
Abstract
These results suggest that L/M cones, the predominant class of cone photoreceptors in the retinas of dogs and most mammalian species can be successfully targeted using the human red cone opsin promoter....
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PMID: 18337838
PDF is available here.
PDF is available here.
Gene Therapy (15)14 2008
Abstract
We established MSCs cell lines with stably integrated mutant neomycin resistance and enhanced green fluorescent protein reporter genes. The established cultures showed morphologically homogenous population with phenotypic and functional features of mesenchymal progenitors. Transfection with gene-spe...
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PMID: 18337839
PDF is available here.
PDF is available here.
Abstract
I review how novel therapies can exploit endogenous replication lesions in cancer cells and convert them to toxic lesions. The aim of these therapies is to produce similar lesions to those produced by DNA damaging anti-cancer drugs. The difference is that the lesions will be cancer-specific and prod...
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PMID: 18374562
PDF is available here.
PDF is available here.
Laryngoscope (118)4 2008
Abstract
The enhanced targeting achieved with Ad5-Flag-LDS highlights a potential strategy for clinically applicable targeted gene therapy against HNSCC or any tumor type expressing Hsp47....
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PMID: 18176343
PDF is available here.
PDF is available here.
Abstract
We focus on methods that have been developed for gene correction and for the modification of gene products. This mutation-focused approach offers the opportunity for 'personalized' gene therapy for muscular dystrophy and might also be a logical strategy for the treatment of other genetic disorders....
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PMID: 18268530
PDF is available here.
PDF is available here.
Abstract
Currently, the laboratory diagnosis of toxocariasis, caused by Toxocara canis or T. cati, mainly relies on serological tests. Unfortunately, however, the specificities of most of the commercial tests that are available for the serodiagnosis of this disease are not very high and this may cause proble...
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PMID: 18318937
PDF is available here.
PDF is available here.
Abstract
The heterogeneity of acute myeloid leukemia (AML) has been established by many new insights from molecular biological studies. In AML with favorable cytogenetic changes, KIT gene mutation has been known as a worse prognostic marker. Even in AML with normal cytogenetics, numerous molecular genetic al...
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PMID: 18079616
PDF is available here.
PDF is available here.
Abstract
Hematopoietic stem cells comprise a prominent target for gene therapy aimed at treating various genetic and acquired disorders. A number of limitations associated with hematopoietic cell transplantation can be circumvented by the use of cells stably modified by retroviral gene transfer. Oncoretrovir...
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PMID: 18022569
PDF is available here.
PDF is available here.
Gene Therapy (14)24 2007
Abstract
We highlight the important advances over the last two to three years, some of which have moved the technology closer to the clinic while some others have introduced new reasons for caution....
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PMID: 17972921
PDF is available here.
PDF is available here.
médecine/sciences (23)10 2007
Abstract
The ability to achieve site-specific correction or modification of the genome has widespread implications for basic and applied research. Individual zinc finger (ZF) domain recognizes DNA triplets with high specificity and affinity. They are used to create zinc finger protein (ZFP), like the ZF-nucl...
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PMID: 17937891
PDF is available here.
PDF is available here.
Gene Therapy (14)19 2007
Abstract
We prepared galactosylated chitosan-graft-polyethylenimine (GC-g-PEI) copolymer by an imine reaction between periodate-oxidized GC and low-molecular-weight PEI. The molecular weight and composition were characterized using gel permeation chromatography column with multi-angle laser scattering and (1...
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PMID: 17637795
PDF is available here.
PDF is available here.
Pharmaceutical Research (24)5 2007
Abstract
Idiopathic Pulmonary Fibrosis (IPF) is a chronic, progressive, and often fatal form of interstitial lung disease. It is characterized by injury with loss of lung epithelial cells and abnormal tissue repair, resulting in replacement of normal functional tissue, abnormal accumulation of fibroblasts an...
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PMID: 17333393
PDF is available here.
PDF is available here.
Seminars in Oncology (34)2 Suppl 1 2007
Abstract
The availability of a reference human genome sequence, an increasingly dense catalog, knowledge of common genetic variation, and new developments in technology present an unprecedented opportunity to systematically explore the genetic basis of complex human diseases such as cancer. An understanding...
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PMID: 17449348
PDF is available here.
PDF is available here.
Abstract
We previously reported that replication plays a pivotal role during this process. Replication direction dictates strand bias, such that single stranded oligonucleotide (SSO) with sequence corresponding to the nascent lagging strand directs higher levels of recombinant formation compared to its compl...
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PMID: 17174937
PDF is available here.
PDF is available here.
Biochimica et Biophysica Acta (1772)2 2007
Abstract
We must acknowledge significant limitations of present vector systems that must be overcome prior to successful treatment of humans with such approaches. This review will present a variety of viral-mediated therapeutic strategies aimed at counteracting the muscle-wasting symptoms associated with mus...
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PMID: 17064882
PDF is available here.
PDF is available here.
Molecular Vision (13) 2007
Abstract
Short ODNs can be delivered with repeatable efficiency to mouse photoreceptor cells in vivo using a combination of intravitreal injection and iontophoresis. Delivery of therapeutic ODNs to rd1 mouse eyes resulted in genomic DNA conversion from mutant to wild type sequence, low but observable beta-PD...
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PMID: 17563719
PDF is available here.
PDF is available here.
Abstract
In this report, we find that addition of selenomethionine reduces gene repair frequency in a dose-dependent fashion. A correlation between gene repair and altered cell cycle progression is observed. We also find that selenium induces expression of Ref-1 which, in turn, modifies the activity of p53 d...
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PMID: 17257432
PDF is available here.
PDF is available here.
Laryngo-Rhino-Otologie (86)1 2007
Abstract
In search of new targeted therapies for squamous cell carcinoma of the head neck (HNSCC), a better understanding of the carcinogenesis is of outmost importance. Recent studies show that not only genetic but also epigenetic alterations initiate the multistep process of tumordevelopment. Epigenetic ch...
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PMID: 17226729
PDF is available here.
PDF is available here.
Oligonucleotides (17)2 2007
Abstract
We hypothesized that enhancement of DNA binding affinity of SSOs by intercalating agents may increase the number of corrected cells. Several biochemical modifications of SSOs were tested for their capability to correct a chromosomally integrated and mutated GFP reporter gene in human 293 cells. SSOs...
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PMID: 17638529
PDF is available here.
PDF is available here.
Abstract
Embryonal tumours constitute the largest group of malignant pediatric brain tumours. Their origin and histological classification is debated. Diagnosis is controversial and patients' response to therapy on the basis of morphologic appearance alone, is difficult to predict. Therefore accurate clinica...
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PMID: 16794961
PDF is available here.
PDF is available here.
Kidney International (61)1 Suppl 2002
Abstract
The gene correction frequency varied, ranging from less than 1% to more than 40%. This represented several magnitudes higher conversion rate compared with homologous recombination frequency, which is in the range of 10(-5) to 10(-6). The resulting phenotype changes lasted longer than one year in som...
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PMID: 11841612
PDF is available here.
PDF is available here.