We provide detailed revision materials for A-Level Biology students and teachers. Gene therapy is . Once bound to these receptors, the therapeutic DNA constructs are engulfed by the cell membrane and passed into the interior of the target cell. It is carried out by introducing DNA containing the functional gene into a patient, to correct a disease-causing mutation. Dr Garry Scarlett, Dr Colin Sharpe Year 3 'Gene Therapy' notes for the module Genomics in... View more. Although in principal gene therapy seems simple, there are many technical, biological and ethical challenges. By chemically linking the DNA to a molecule, therapeutic DNA also can get inside target cells. It was first conceptualized in 1972, with the authors urg­ing caution before commencing gene therapy studies in humans. Gene Therapy Pros and Cons: Gene therapy is involved in the replacement of a defective gene within a cell’s nucleus with the genetically healthy and functional gene. For general information about gene therapy: MedlinePlus offers a list of links to information about genes and gene therapy. If you want to see what we offer before purchasing, we have a free membership with sample revision materials. Summary. Gene therapy can be used to treat this disease. GENE THERAPY Presented by:Nurul Miza Shasheiha binti Abdul Mutalib -5089- Wan Fathiah Nasuha binti Wan Nudri -5117- Nurul Husna binti Muryadi -5088- Nur Amalin Shafirah binti Nur Farzana Sahirah binti Ariffuddin TOS 7. The simple process of gene therapy is shown in the figure below: In the figure, the cell with the defective gene is injected with a normal gene which helps in the … This type of therapy only works if the lack of the functional protein hasn’t caused lasting damage to the body, or wasn’t required during development. The simplest method is the direct introduction of therapeutic DNA into target cells. Gene therapy is likely to be a promising treatment option for certain genetic diseases in which there is one faulty gene that affects a particular cell type that can be repaired. 19. Disclaimer 9. Although genes get a lot of attention, it’s the proteins that perform most life functions and even make up the majority of cellular structures. Gene augmentation therapy is used to treat diseases where a mutation in the DNA causes a loss of the function of a gene, stopping it producing a working protein. If you're ready to pass your A-Level Biology exams, become a member now to get complete access to our entire library of revision materials. R. Michael Blaese, W. French Anderson and Kenneth Culver at a press conference announcing the start of the first gene therapy trial for treating children with severe combined immunodeficiency, 13 September 1990. It can be used to … Viruses have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner. The factors that are holding gene therapy from becoming an effective treatment for genetic disease are: To use gene therapy as a permanent cure for any condition, the therapeutic DNA introduced into target cells must remain functional and the cells containing the therapeutic DNA must be long-lived and stable. Viruses are small infectious agents that have evolved mechanisms to be highly efficient at infecting our cells. Gene therapy is a technique for correcting defective genes responsible for disease development. Gene therapy has the potential to eliminate and prevent hereditary diseases such as cystic fibrosis, ADA- SCID etc. Gene inhibition therapy is used in the treatment of diseases caused by spurious gene activity, either by the gene being over-active (perhaps the gene is on when it should be off) or by production of a new protein that interferes with the function of another gene. Some of the different types of viruses used as gene therapy vectors are: A class of viruses that can create double-stranded DNA copies of their RNA genomes. (ii) An abnormal gene could be swapped for a normal gene through homologous recombination.

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