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Gene therapy

Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs.

Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs.

... to gene therapy was almost double that expected for colony dogs (11–12 ...following gene ther- apy [13,32] and with the prophylactic replacement therapy that is the standard care of humans ...

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A biophysical approach to mitochondrial gene therapy: gemini surfactant-based colloidal vectors

A biophysical approach to mitochondrial gene therapy: gemini surfactant-based colloidal vectors

... Mitochondrial gene therapy techniques and protocols are becoming ever more popular due to their potential ability to treat otherwise incurable diseases and syndromes, such as the ones referred in chapter ...

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Gene therapy: Current status and future perspectives

Gene therapy: Current status and future perspectives

... a gene are called mutations, which often are harmless but sometimes can lead to serious ...disease. Gene therapy is a technique in which a functional gene replaces the defective gene so ...

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Adeno-associated virus for cystic fibrosis gene therapy

Adeno-associated virus for cystic fibrosis gene therapy

... the gene transfer of AAV vectors for CF gene therapy, the activities of different promoters, cytomegalovirus (CMV), Rous sarcoma virus and cytomega- lovirus promoter conjugated/β - actin (CB), were ...

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Minicircular DNA as a potential gene therapy vector in cervical cancer

Minicircular DNA as a potential gene therapy vector in cervical cancer

... horizontal gene transfer, placing biological safety at ...in gene therapy because they are immunostimulating by promoting activation of Toll-Liker receptors, affecting the expression of therapeutic ...

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Beyond retrovirus infection: HIV meets gene therapy

Beyond retrovirus infection: HIV meets gene therapy

... specific gene and to evaluate its interaction with other genes (Sui et ...stable gene transfer systems for K562 cancer cell lines and for CD34+ cells from chronic myeloid leuke- mia patients and have shown ...

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Transfection efficiency of a vector for mitochondrial gene therapy

Transfection efficiency of a vector for mitochondrial gene therapy

... Non-viral gene therapy methods have been extensively studied lately due to the advantages they present. They are safer, cheaper, less toxic, and thus have a higher biocompatibility, and can carry larger ...

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Tumor Restrictive Suicide Gene Therapy for Glioma Controlled by the FOS Promoter.

Tumor Restrictive Suicide Gene Therapy for Glioma Controlled by the FOS Promoter.

... a gene therapy strategy to target hepatocellular carcinoma based inhibition of protein phosphatase 2A using the alpha-fetoprotein promoter enhancer and pgk promoter: an in vitro and in vivo ...

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The challenge of vector development in gene therapy

The challenge of vector development in gene therapy

... a gene of ...a gene of interest, it is necessary to use a packaging cell line harboring the gag, pol and env genes which have been previ- ously incorporated into the genome of such ...vivo gene ...

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Cationic polyene phospholipids as DNA carriers for ocular gene therapy

Cationic polyene phospholipids as DNA carriers for ocular gene therapy

... Recent success in the treatment of congenital blindness demonstrates the potential of ocular gene therapy as a therapeutic approach. The eye is a good target due to its small size, minimal diffusion of ...

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Cancer gene therapy

Cancer gene therapy

... in gene therapy is a challenge because of its inability in the extracellular matrix and its inability to cross the eukaryotic cell membrane barrier and reach the nucleus in its native solution ...

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Development of the system for gene therapy of AIDS on the basis of antisense RNAs

Development of the system for gene therapy of AIDS on the basis of antisense RNAs

... Taking into account the presence of viral protein ð24 in transfected cells and some others, as well as a tra- ce level of the formation of mature virions, one might expect that during this controlled infectious process ...

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Gene therapy based on interleukin-12 loaded chitosan nanoparticles in a mouse model of fibrosarcoma

Gene therapy based on interleukin-12 loaded chitosan nanoparticles in a mouse model of fibrosarcoma

... IL-12 gene therapy was mostly dependent on NK cells and partly on T ...Also, gene therapy with pUMVC3-mIL-12 loaded chitosan nanoparticles could decrease Ki-67 expression in the fibrosarcoma ...

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Multifactorial approach to non-viral gene therapy: development of an efficient system for the retina

Multifactorial approach to non-viral gene therapy: development of an efficient system for the retina

... for gene therapy because it is an accessible and immune-privileged organ (Bloquel et ...for gene therapy (Bloquel et ...therapeutic gene that can be incorporated into the viral vector ...

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Combating oncogene activation associated with retrovirus-mediated gene therapy of X-linked severe combined immunodeficiency

Combating oncogene activation associated with retrovirus-mediated gene therapy of X-linked severe combined immunodeficiency

... essential gene, thus killing the ...the gene therapy strategy since this would be a very rare event in a large population of independently transduced ...tial gene or tumor suppressor less ...

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Gene therapy: development of a new nanocarrier system for mitochondrial gene therapy

Gene therapy: development of a new nanocarrier system for mitochondrial gene therapy

... Mitochondrial gene therapy appears to be quite promising as a valuable approach to reestablish normal metabolic ...therapeutic gene into this organelle with the ultimate goal of functional protein ...

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Somatic gene therapy for hypertension

Somatic gene therapy for hypertension

... gene therapy. Adenovirus vectors have been used with kallikrein gene inser- tion ...human therapy so ...hypertension therapy in ...large gene-carrying capacity and are easily ...

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Targeted gene therapy of xeroderma pigmentosum cells using meganuclease and TALEN™.

Targeted gene therapy of xeroderma pigmentosum cells using meganuclease and TALEN™.

... functional gene benefits from the natural chromosomal context and regulatory regions (endogenous pro- moter, terminator, and enhancer), known to play key roles in the fine tuning of gene ...that gene ...

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Gammaretroviral and Lentiviral Vectors for Gene Therapy

Gammaretroviral and Lentiviral Vectors for Gene Therapy

... Dissertation presented to obtain a Ph.D degree in Engineering and Technology Sciences, Gene Therapy at the Instituto de Tecnologia Quimica e Biológica,. Universidade Nova de Lisboa[r] ...

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Apoptosis as a Target for Gene Therapy in Rheumatoid Arthritis

Apoptosis as a Target for Gene Therapy in Rheumatoid Arthritis

... for gene therapy. Gene transfer strategies include inhibition of pro-inflammatory cytokines, blockade of cartilage-de- grading metalloproteinases, inhibition of synovial cell activation and ...

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