Consulta: subjectFacets:"Gene therapy"
Registros recuperados: 21
Data/hora: 03/06/2024 19:14:39

A role for adeno-associated viral vectors in gene therapy
Provedor de dados: Genetics and Molecular Biology
Autores: Coura,Renata dos Santos; Nardi,Nance Beyer.
Gene therapy constitutes a therapeutic intervention based on modification of the genetic material of living cells, by correcting genetic defects or overexpressing therapeutic proteins. The success of gene therapy protocols depends on the availability of therapeutically suitable genes, appropriate gene delivery systems and proof of safety and efficacy. Recent advances on the development of gene delivery systems, particularly on viral vectors engineering and improved gene regulatory systems, have led to marked progress in this field. Although the available vector systems can successfully transfer genes into cells, the ideal delivery vehicle has not been found. In this context, adeno-associated virus vectors (AAV) are arising as a promising tool for a wide...
Tipo: Info:eu-repo/semantics/article
Palavras-chave: Adeno-associated virus; AAV-based recombinant vectors; Gene therapy.
Ano: 2008
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S1415-47572008000100001

A snapshot of gene therapy in Latin America
Provedor de dados: Genetics and Molecular Biology
Autores: Linden,Rafael; Matte,Ursula.
Gene therapy attempts the insertion and expression of exogenous genetic material in cells for therapeutic purposes. Conceived in the 1960s, gene therapy reached its first clinical trial at the end of the 1980s and by December 2013 around 600 genuine open clinical trials of gene therapy were registered at NIH Clinical Trials Database. Here, we summarize the current efforts towards the development of gene therapy in Latin America. Our survey shows that the number of scientists involved in the development of gene therapy and DNA vaccines in Latin America is still very low. Higher levels of investment in this technology are necessary to boost the advancement of innovation and intellectual property in this field in a way that would ease both the social and...
Tipo: Info:eu-repo/semantics/article
Palavras-chave: Gene therapy; Gene transfer; Gene delivery; Viral vector; South America.
Ano: 2014
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S1415-47572014000200015

Lysosomal diseases: Overview on current diagnosis and treatment
Provedor de dados: Genetics and Molecular Biology
Autores: Poswar,Fabiano de Oliveira; Vairo,Filippo; Burin,Maira; Michelin-Tirelli,Kristiane; Brusius-Facchin,Ana Carolina; Kubaski,Francyne; Souza,Carolina Fischinger Moura de; Baldo,Guilherme; Giugliani,Roberto.
Abstract Lysosomal diseases (LDs), also known as lysosomal storage diseases (LSDs), are a heterogeneous group of conditions caused by defects in lysosomal function. LDs may result from deficiency of lysosomal hydrolases, membrane-associated transporters or other non-enzymatic proteins. Interest in the LD field is growing each year, as more conditions are, or will soon be treatable. In this article, we review the diagnosis of LDs, from clinical suspicion and screening tests to the identification of enzyme or protein deficiencies and molecular genetic diagnosis. We also cover the treatment approaches that are currently available or in development, including hematopoietic stem cell transplantation, enzyme replacement therapy, small molecules, and gene therapy.
Tipo: Info:eu-repo/semantics/article
Palavras-chave: Lysosomal storage diseases; Neonatal screening; Hematopoietic stem cell transplantation; Enzyme replacement therapy; Gene therapy.
Ano: 2019
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S1415-47572019000200165

Beyond retrovirus infection: HIV meets gene therapy
Provedor de dados: Genetics and Molecular Biology
Autores: Silva,Flávia Helena da; Dalberto,Tiago Pires; Nardi,Nance Beyer.
The human immunodeficiency virus (HIV) is classified as a retrovirus because of its RNA genome and the fact that it requires reverse transcriptase to convert it into DNA. This virus belongs to the lentivirinae subfamily and is able to infect quiescent cells but is better known for its association with acquired immunodeficiency syndrome (AIDS) and can be described as one of the most effective vectors for gene transfer. Biosafety concerns are present whenever viral vectors are employed but are particularly pertinent to the development of HIV-based vectors. Insertional mutagenesis and the production of new replication-competent viruses (RCV) have been pointed to as major problems, but experimental data have shown that safe protocols can be developed for their...
Tipo: Info:eu-repo/semantics/article
Palavras-chave: HIV; MLV; Retrovirus; Lentivirus; Gene therapy.
Ano: 2006
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S1415-47572006000200027

Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases
Provedor de dados: Genetics and Molecular Biology
Autores: Poletto,Edina; Pasqualim,Gabriela; Giugliani,Roberto; Matte,Ursula; Baldo,Guilherme.
Abstract Lysosomal storage diseases (LSDs) are inherited conditions caused by impaired lysosomal function and consequent substrate storage, leading to a range of clinical manifestations, including cardiovascular disease. This may lead to significant symptoms and even cardiac failure, which is an important cause of death among patients. Currently available treatments do not completely correct cardiac involvement in the LSDs. Gene therapy has been tested as a therapeutic alternative with promising results for the heart disease. In this review, we present the results of different approaches of gene therapy for LSDs, mainly in animal models, and its effects in the heart, focusing on protocols with cardiac functional analysis.
Tipo: Info:eu-repo/semantics/article
Palavras-chave: Lysosomal storage disease; Gene therapy; Cardiovascular disease; Animal models; Heart.
Ano: 2019
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S1415-47572019000200261

Awareness and current knowledge of breast cancer
Provedor de dados: Biological Research
Autores: Akram,Muhammad; Iqbal,Mehwish; Daniyal,Muhammad; Khan,Asmat Ullah.
Abstract Breast cancer remains a worldwide public health dilemma and is currently the most common tumour in the globe. Awareness of breast cancer, public attentiveness, and advancement in breast imaging has made a positive impact on recognition and screening of breast cancer. Breast cancer is life-threatening disease in females and the leading cause of mortality among women population. For the previous two decades, studies related to the breast cancer has guided to astonishing advancement in our understanding of the breast cancer, resulting in further proficient treatments. Amongst all the malignant diseases, breast cancer is considered as one of the leading cause of death in post menopausal women accounting for 23% of all cancer deaths. It is a global...
Tipo: Journal article
Palavras-chave: Breast cancer; Chemotherapy; Gene therapy; Stem cell therapy.
Ano: 2017
URL: http://www.scielo.cl/scielo.php?script=sci_arttext&pid=S0716-97602017000100504

Hereditary hemochromatosis: An opportunity for gene therapy
Provedor de dados: Biological Research
Autores: EZQUER,FERNANDO; NÚÑEZ,MARCO T; ROJAS,ALEJANDRO; ASENJO,JUAN; ISRAEL,YEDY.
Levels of body iron should be tightly controlled to prevent the formation of oxygen radicals, lipoperoxidation, genotoxicity, and the production of cytotoxic cytokines, which result in damage to a number of organs. Enterocytes in the intestinal villae are involved in the apical uptake of iron from the intestinal lumen; iron is further exported from the cells into the circulation. The apical divalent metal transporter-1 (DMT1) transports ferrous iron from the lumen into the cells, while the basolateral transporter ferroportin extrudes iron from the enterocytes into the circulation. Patients with hereditary hemochromatosis display an accelerated transepithelial uptake of iron, which leads to body iron accumulation that results in cirrhosis, hepatocellular...
Tipo: Journal article
Palavras-chave: Iron; Intestine; Hemochromatosis; Gene therapy; HFE; DMT1; Cirrhosis.
Ano: 2006
URL: http://www.scielo.cl/scielo.php?script=sci_arttext&pid=S0716-97602006000100014

Carbon sequestration in the rain forest: alternatives using environmentally friendly biotechnology
Provedor de dados: Biota Neotropica
Autores: Buckeridge,Marcos S.; Aidar,Marcos P.M.
As carbon dioxide increases on Earth atmosphere, the rise in average temperatures may provoke changes in the environment that could damage civilisation as we know it. As a result, the need to sequester carbon becomes urgent, and one of the options we have is to use the potential of the forests to do it by enhancing assimilation of CO2 through photosynthesis. However, if we consider the use of plants to increase carbon sequestration, a problem that looms is that species often acclimate and actually reduce CO2 assimilation through feedback mechanisms of the sugars that are the product. In the present article, we propose that some biochemical pathways, such as those in control of photosynthesis, carbohydrate metabolism and assimilation, and cellulose and...
Tipo: Info:eu-repo/semantics/other
Palavras-chave: Global change; Carbon Sequestration; Photosynthesis; Rain Forest; Sugar Sensing; Cellulose synthesis; Gene therapy.
Ano: 2002
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S1676-06032002000100002

Purification of plasmid (pVaxLacZ) by hydrophobic interaction chromatography
Provedor de dados: Brazilian Archives of Biology and Technology
Autores: Moreira,Keila Aparecida; Diogo,Margarida; Prazeres,Duarte Miguel; Lima Filho,José Luiz de; Porto,Ana Lúcia Figueiredo.
This paper describes a method for the plasmid DNA purification, which includes an ammonium sulphate precipitation, followed by hydrophobic interaction chromatography (HIC) using Phenyl Sepharose 6 Fast Flow (low sub). The use of HIC took advantage of the more hydrophobic character of single stranded nucleic acid impurities as compared with double-stranded plasmid DNA.
Tipo: Info:eu-repo/semantics/article
Palavras-chave: Gene therapy; Hydrophobic interaction chromatography; Plasmid; Purification.
Ano: 2005
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S1516-89132005000400014

Modulation of the expression of the transcription factor Max in rat retinal ganglion cells by a recombinant adeno-associated viral vector
Provedor de dados: Brazilian Journal of Medical and Biological Research
Autores: Petrs-Silva,H.; Chiodo,V.; Chiarini,L.B.; Hauswirth,W.W.; Linden,R.
Exclusion of the transcription factor Max from the nucleus of retinal ganglion cells is an early, caspase-independent event of programmed cell death following damage to the optic axons. To test whether the loss of nuclear Max leads to a reduction in neuroprotection, we developed a procedure to overexpress Max protein in rat retinal tissue in vivo. A recombinant adeno-associated viral vector (rAAV) containing the max gene was constructed, and its efficiency was confirmed by transduction of HEK-293 cells. Retinal ganglion cells were accessed in vivo through intravitreal injections of the vector in rats. Overexpression of Max in ganglion cells was detected by immunohistochemistry at 2 weeks following rAAV injection. In retinal explants, the preparation of...
Tipo: Info:eu-repo/semantics/other
Palavras-chave: Retina; Max expression; Transcription factor; Viral vector; Gene therapy; Neuroprotection.
Ano: 2005
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X2005000300008

Efficient retrovirus-mediated transfer of cell-cycle control genes to transformed cells
Provedor de dados: Brazilian Journal of Medical and Biological Research
Autores: Strauss,B.E.; Costanzi-Strauss,E.
The use of gene therapy continues to be a promising, yet elusive, alternative for the treatment of cancer. The origins of cancer must be well understood so that the therapeutic gene can be chosen with the highest chance of successful tumor regression. The gene delivery system must be tailored for optimum transfer of the therapeutic gene to the target tissue. In order to accomplish this, we study models of G1 cell-cycle control in both normal and transformed cells in order to understand the reasons for uncontrolled cellular proliferation. We then use this information to choose the gene to be delivered to the cells. We have chosen to study p16, p21, p53 and pRb gene transfer using the pCL-retrovirus. Described here are some general concepts and specific...
Tipo: Info:eu-repo/semantics/article
Palavras-chave: Gene therapy; Clinical trial; Retrovirus; Animal model; Cell cycle; Growth arrest.
Ano: 1999
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X1999000700016

Transient high-level expression of ß-galactosidase after transfection of fibroblasts from GM1 gangliosidosis patients with plasmid DNA
Provedor de dados: Brazilian Journal of Medical and Biological Research
Autores: Balestrin,R.C.; Baldo,G.; Vieira,M.B.; Sano,R.; Coelho,J.C.; Giugliani,R.; Matte,U.
GM1 gangliosidosis is an autosomal recessive disorder caused by the deficiency of lysosomal acid hydrolase ß-galactosidase (ß-Gal). It is one of the most frequent lysosomal storage disorders in Brazil, with an estimated frequency of 1:17,000. The enzyme is secreted and can be captured by deficient cells and targeted to the lysosomes. There is no effective treatment for GM1 gangliosidosis. To determine the efficiency of an expression vector for correcting the genetic defect of GM1 gangliosidosis, we tested transfer of the ß-Gal gene (Glb1) to fibroblasts in culture using liposomes. ß-Gal cDNA was cloned into the expression vectors pSCTOP and pREP9. Transfection was performed using 4 µL lipofectamine 2000 and 1.5-2.0 µg DNA. Cells (2 x 10(5)/well) were...
Tipo: Info:eu-repo/semantics/article
Palavras-chave: GM1 gangliosidosis; SS-galactosidase deficiency; Gene therapy; Lysosomal storage disorder; Lipofectamine.
Ano: 2008
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X2008000400005

p19Arf sensitizes B16 melanoma cells to interferon-β delivered via mesenchymal stem cells in vitro
Provedor de dados: Brazilian Journal of Medical and Biological Research
Autores: Da-Costa,R.C.; Vieira,I.L.; Hunger,A.; Tamura,R.E.; Strauss,B.E.
The immune stimulatory and anti-neoplastic functions of type I interferon have long been applied for the treatment of melanoma. However, the systemic application of high levels of this recombinant protein is often met with toxicity. An approach that provides localized, yet transient, production of type I interferon may overcome this limitation. We propose that the use of mesenchymal stem cells (MSCs) as delivery vehicles for the production of interferon-β (IFNβ) may be beneficial when applied together with our cancer gene therapy approach. In our previous studies, we have shown that adenovirus-mediated gene therapy with IFNβ was especially effective in combination with p19Arf gene transfer, resulting in immunogenic cell death. Here we showed that MSCs...
Tipo: Info:eu-repo/semantics/article
Palavras-chave: Gene therapy; Immunotherapy; Stem cell therapy; Adenovirus; P53; Interferon-β.
Ano: 2020
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X2020000300601

Adeno-associated virus for cystic fibrosis gene therapy
Provedor de dados: Brazilian Journal of Medical and Biological Research
Autores: Martini,S.V.; Rocco,P.R.M.; Morales,M.M.
Gene therapy is an alternative treatment for genetic lung disease, especially monogenic disorders such as cystic fibrosis. Cystic fibrosis is a severe autosomal recessive disease affecting one in 2500 live births in the white population, caused by mutation of the cystic fibrosis transmembrane conductance regulator (CFTR). The disease is classically characterized by pancreatic enzyme insufficiency, an increased concentration of chloride in sweat, and varying severity of chronic obstructive lung disease. Currently, the greatest challenge for gene therapy is finding an ideal vector to deliver the transgene (CFTR) to the affected organ (lung). Adeno-associated virus is the most promising viral vector system for the treatment of respiratory disease because it...
Tipo: Info:eu-repo/semantics/article
Palavras-chave: Gene therapy; Adeno-associated virus; Cystic fibrosis; Cystic fibrosis transmembrane conductance regulator; Vectors.
Ano: 2011
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X2011001100004

Combating oncogene activation associated with retrovirus-mediated gene therapy of X-linked severe combined immunodeficiency
Provedor de dados: Brazilian Journal of Medical and Biological Research
Autores: Strauss,B.E.; Costanzi-Strauss,E.
A successful gene therapy clinical trial that also encountered serious adverse effects has sparked extensive study and debate about the future directions for retrovirus-mediated interventions. Treatment of X-linked severe combined immunodeficiency with an oncoretrovirus harboring a normal copy of the gc gene was applied in two clinical trials, essentially curing 13 of 16 infants, restoring a normal immune system without the need for additional immune-related therapies. Approximately 3 years after their gene therapy, tragically, 3 of these children, all from the same trial, developed leukemia as a result of this experimental treatment. The current understanding of the mechanism behind this leukemogenesis involves three critical and cooperating factors,...
Tipo: Info:eu-repo/semantics/article
Palavras-chave: Retrovirus; Insertional mutagenesis; Severe combined immune deficiency; Gene therapy; Adverse event; Clinical trial.
Ano: 2007
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X2007000500002

Regulation of transgene expression in genetic immunization
Provedor de dados: Brazilian Journal of Medical and Biological Research
Autores: Harms,J.S.; Oliveira,S.C.; Splitter,G.A.
The use of mammalian gene expression vectors has become increasingly important for genetic immunization and gene therapy as well as basic research. Essential for the success of these vectors in genetic immunization is the proper choice of a promoter linked to the antigen of interest. Many genetic immunization vectors use promoter elements from pathogenic viruses including SV40 and CMV. Lymphokines produced by the immune response to proteins expressed by these vectors could inhibit further transcription initiation by viral promoters. Our objective was to determine the effect of IFN-&lt;FONT FACE="Symbol"&gt;g&lt;/FONT&gt; on transgene expression driven by viral SV40 or CMV promoter/enhancer and the mammalian promoter/enhancer for the major...
Tipo: Info:eu-repo/semantics/article
Palavras-chave: DNA vaccine; Gene therapy; Regulation; Transcription; Transgene.
Ano: 1999
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X1999000200003

Somatic gene therapy for hypertension
Provedor de dados: Brazilian Journal of Medical and Biological Research
Autores: Phillips,M.I.
Gene therapy for hypertension is needed for the next generation of antihypertensive drugs. Current drugs, although effective, have poor compliance, are expensive and short-lasting (hours or one day). Gene therapy offers a way to produce long-lasting antihypertensive effects (weeks, months or years). We are currently using two strategies: a) antisense oligodeoxynucleotides (AS-ODN) and b) antisense DNA delivered in viral vectors to inhibit genes associated with vasoconstrictive properties. It is not necessary to know all the genes involved in hypertension, since many years of experience with drugs show which genes need to be controlled. AS-ODN are short, single-stranded DNA that can be injected in naked form or in liposomes. AS-ODN, targeted to angiotensin...
Tipo: Info:eu-repo/semantics/article
Palavras-chave: Gene therapy; Hypertension; Antisense; Vectors.
Ano: 2000
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X2000000600013

The challenge of vector development in gene therapy
Provedor de dados: Brazilian Journal of Medical and Biological Research
Autores: Dani,S.U.
Gene therapy is the treatment of diseases based on the transfer of genetic information. Agents that carry or deliver DNA to target cells are called vectors (Latin vector: carrier, deliverer). Ideally, a vector should accommodate an unlimited amount of inserted DNA, lack the ability of autonomous replication of its own DNA, be easily manufactured, and be available in concentrated form. Secondly, it should have the ability to target specific cell types or to limit its gene expression to specific cell types, and to achieve sustained gene expression in the long term or in a controlled fashion. Finally, it should not be toxic or immunogenic. Such a vector does not exist and none of the DNA delivery systems so far available for in vivo gene transfer is perfect...
Tipo: Info:eu-repo/semantics/article
Palavras-chave: Gene therapy; Gene transfer; DNA delivery; Viral vectors; Diposomes; Vector development.
Ano: 1999
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X1999000200001

Death switch for gene therapy: application to erythropoietin transgene expression
Provedor de dados: Brazilian Journal of Medical and Biological Research
Autores: Souza,D.S.; Spencer,D.M.; Salles,T.S.I.; Salomão,M.A.; Payen,E.; Beuzard,Y.; Carvalho,H.F.; Costa,F.F.; Saad,S.T.Olalla.
The effectiveness of the caspase-9-based artificial "death switch" as a safety measure for gene therapy based on the erythropoietin (Epo) hormone was tested in vitro and in vivo using the chemical inducer of dimerization, AP20187. Plasmids encoding the dimeric murine Epo, the tetracycline-controlled transactivator and inducible caspase 9 (ptet-mEpoD, ptet-tTAk and pSH1/Sn-E-Fv’-Fvls-casp9-E, respectively) were used in this study. AP20187 induced apoptosis of iCasp9-modified C2C12 myoblasts. In vivo, two groups of male C57BI/6 mice, 8-12 weeks old, were injected intramuscularly with 5 µg/50 g ptet-mEpoD and 0.5 µg/50 g ptet-tTAk. There were 20 animals in group 1 and 36 animals in group 2. Animals from group 2 were also injected with the 6 µg/50 g iCasp9...
Tipo: Info:eu-repo/semantics/article
Palavras-chave: Gene therapy; Erythropoietin; Death switch; Caspase 9; AP20187; Anemia.
Ano: 2010
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X2010000700005

Myostatin: genetic variants, therapy and gene doping
Provedor de dados: Brazilian Journal of Pharmaceutical Sciences
Autores: Yamada,André Katayama; Verlengia,Rozangela; Bueno Junior,Carlos Roberto.
Since its discovery, myostatin (MSTN) has been at the forefront of muscle therapy research because intrinsic mutations or inhibition of this protein, by either pharmacological or genetic means, result in muscle hypertrophy and hyperplasia. In addition to muscle growth, MSTN inhibition potentially disturbs connective tissue, leads to strength modulation, facilitates myoblast transplantation, promotes tissue regeneration, induces adipose tissue thermogenesis and increases muscle oxidative phenotype. It is also known that current advances in gene therapy have an impact on sports because of the illicit use of such methods. However, the adverse effects of these methods, their impact on athletic performance in humans and the means of detecting gene doping are as...
Tipo: Info:eu-repo/semantics/article
Palavras-chave: Myostatin; Myostatin/genetic variants; Myostatin/pharmacological inhibitors; Gene doping; Gene therapy; Physical performance; Skeletal muscle.
Ano: 2012
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S1984-82502012000300003

P53 gene: major mutations in neoplasias and anticancer gene therapy
Provedor de dados: Ciência Rural
Autores: Lima,Caroline Rocha de Oliveira; Rabelo,Rogério Elias; Vulcani,Valcinir Aloísio Scalla; Cardoso,Lorena Damasio; Sousa,Nicaelle Luan de Moura; Moura,Veridiana Maria Brianezi Dignani de.
The p53 gene encodes a protein that has molecular weight of 53kD and is also called p53 protein, being constantly studied for its classic concept of "genome guardian". This gene plays a range of essential functions to ensure the cell cycle control, in addition to playing a central role in carcinogenesis. With respect to neoplasias, it prevents the neoplastic transformation through three intricate mechanisms. Depending on the extent of the mutation, different responses may be sent by p53 and those range since the disruption of the cell cycle, the correction of the mutation through the activation of repair proteins or still, the induction of senescence or cell death by apoptosis. This review aims to address the structural and functional aspects of the p53...
Tipo: Info:eu-repo/semantics/article
Palavras-chave: Mutations; Neoplasia; Gene therapy; P53.
Ano: 2012
URL: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0103-84782012000500014