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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- 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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- 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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- One form of the disease is usually caused by mutations in one of three known genes.
- One particular gene, apolipoprotein E (APOE) has a variant (E4) that increases a carrier's probability of developing the disease.
- Many other genes have been identified that may be involved in the pathology.
- Other clinical interventions focus on behavioral therapies such as psychotherapy, sensory therapy, and cognitive exercises.
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- Variants of several genes correlate with the presence of ASD, but for any given patient, many different mutations in different genes may be required for the disease to develop.
- Treatment for autism usually combines behavioral therapies and interventions, along with medications to treat other disorders common to people with autism (depression, anxiety, obsessive compulsive disorder).
- There are several candidate genes that may contribute to the disorder, but no definitive links have been discovered.
- Treatment for ADHD often involves behavioral therapies and the prescription of stimulant medications, which, paradoxically, cause a calming effect in these patients.
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- The B gene controls black (B_) vs. brown (bb) color, while the E gene controls yellow (ee) color.
- Genes may also oppose each other with one gene modifying the expression of another.
- Often the biochemical basis of epistasis is a gene pathway in which the expression of one gene is dependent on the function of a gene that precedes or follows it in the pathway.
- In this case, the C gene is epistatic to the A gene.
- Thus, the C gene is epistatic to the A gene.
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- Highly plastic adult stem cells are routinely used in medical therapies, for example in bone marrow transplantation.
- Embryonic cell lines and autologous embryonic stem cells generated through therapeutic cloning have also been proposed as promising candidates for future therapies.
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- It is important that the targeted proteins be encoded by viral genes and that these molecules are not present in a healthy host cell.
- The breakthrough in the treatment of HIV was the development of HAART, highly-active anti-retroviral therapy, which involves a mixture of different drugs, sometimes called a drug "cocktail."
- Still, even with the use of combination HAART therapy, there is concern that, over time, the virus will develop resistance to this therapy.