Examples of gene therapy in the following topics:
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- The discovery of potential therapies will be made easier using genome targets.
- With modern biotechnology, these genes can be used as targets for the development of effective new therapies, which could significantly shorten the drug discovery process.
- Gene therapy is a genetic engineering technique used to cure disease.
- More advanced forms of gene therapy try to correct the mutation at the original site in the genome, such as is the case with treatment of severe combined immunodeficiency (SCID).
- Gene therapy using an adenovirus vector can be used to cure certain genetic diseases in which a person has a defective gene.
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- Genes may be removed, or "knocked out" , using a nuclease.
- Gene targeting is a different technique that uses homologous recombination to change an endogenous gene, and can be used to delete a gene, remove exons, add a gene, or introduce point mutations.
- The evolving field of gene therapy involves manipulating human genes to treat or cure genetic diseases and disorders.
- Since the 1990s, gene therapy has been used in clinical trials to treat diseases and conditions such as AIDS, cystic fibrosis, cancer, and high cholesterol.
- Drawbacks of gene therapy are that sometimes the person's immune system destroys the cells that have been genetically altered, and also that it is hard to get the genetic material into enough cells to have the desired effect.
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- This idea, that therapy and medicines can be tailored to an individual, has given rise to the field of personalized medicine.
- With an increased understanding of gene regulation and gene function, medicines can be designed to specifically target diseased cells without harming healthy cells.
- Some new medicines, called targeted therapies, have exploited the overexpression of a specific protein or the mutation of a gene to develop a new medication to treat disease.
- Undoubtedly, more targeted therapies will be developed as scientists learn more about how gene expression changes can cause cancer .
- Target therapies exploit the overexpression of a specific protein or gene mutation to develop new medications against the specific cancer.
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- Recombinant DNA technology engineers microbial cells for producing foreign proteins, and its success solely depends on the precise reading of equivalent genes made with the help of bacterial cell machinery.
- The last two decades of cloned-DNA sequence studies have revealed detailed knowledge about gene structure as well as its organization.
- Hereditary diseases carrier diagnosis: tests now available to determine if a person is carrying the gene for cystic fibrosis, the Tay-Sachs diseases, Huntington's disease or Duchenne muscular dystrophy.
- Gene transfer from one organism to other: the advanced gene therapy can benefit people with cystic fibrosis, vascular disease, rheumatoid arthritis and specific types of cancers.
- In this example, the gene indicated by the white color is inactivated upon insertion of the foreign DNA fragment.
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- CF is caused by a mutation in the gene for the protein cystic fibrosis transmembrane conductance regulator (CFTR).
- CF develops when neither gene works normally (as a result of mutation) and therefore has autosomal recessive inheritance.
- Gene therapy has been explored as a potential cure for cystic fibrosis.
- Ideally, gene therapy attempts to place a normal copy of the CFTR gene into affected cells.
- Finally, a number of small molecules that aim at compensating various mutations of the CFTR gene are under development.
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- Silencing genes through epigenetic mechanisms is very common in cancer cells and include modifications to histone proteins and DNA that are associated with silenced genes.
- When these modifications occur, the gene present in that chromosomal region is silenced.
- Because these changes are temporary and can be reversed (for example, by preventing the action of the histone deacetylase protein that removes acetyl groups, or by DNA methyl transferase enzymes that add methyl groups to cytosines in DNA) it is possible to design new drugs and new therapies to take advantage of the reversible nature of these processes.
- In cancer cells, silencing genes through epigenetic mechanisms is a common occurrence.
- Describe the role played by epigenetic alterations to gene expression in the development of cancer
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- In the medical field, DNA is used in diagnostics, new vaccine development, and cancer therapy.
- It is now also possible to determine predispositions to some diseases by looking at genes.
- CRISPR (Clustered, Regularly-Interspaced Short Palindromic Repeats) allows scientists to edit genomes, far better than older techniques for gene splicing and editing.
- Genetic modification involves the mutation, insertion, or deletion of genes.
- Inserted genes usually come from a different species in a form of horizontal gene-transfer.
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- These pathways are controlled by signaling proteins, which are, in turn, expressed by genes.
- Mutations in these genes can result in malfunctioning signaling proteins.
- The genes that regulate the signaling proteins are one type of oncogene: a gene that has the potential to cause cancer.
- The gene encoding RAS is an oncogene that was originally discovered when mutations in the RAS protein were linked to cancer.
- Herceptin therapy helps to control signaling through HER2.
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- Examples of such biological control included bacteriotherapy, bacteriophage therapy, malaria therapy, probiotics, and the use of living maggots .
- Modern studies suggest that the use of biological control in the treatment of human infections should be re-evaluated in the light of the increasing world-wide occurrence of antibiotic-resistant bacteria, and the opportunities provided by recent developments in gene technology.
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- Antisense agents can be specifically targeted to genes that control expression of antibiotic resistance mechanisms, thereby potentially restoring an antibiotic-sensitive phenotype to the cell.
- The advantage of antisense therapy is that they can be manufactured fairly fast, they produce a lasting clinical effect, and they are highly specific to the target.
- Antisense agents also exhibit efficacy in broader clinical applications such as cancer therapy.
- Discuss the mechanism of antisense agents and the advantages and disadvantages of antisense therapy