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Folleto Juan Felipe Giraldo Aristizábal
 

Folleto Juan Felipe Giraldo Aristizábal

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    Folleto Juan Felipe Giraldo Aristizábal Folleto Juan Felipe Giraldo Aristizábal Presentation Transcript

    • Juan Felipe Giraldo Aristizábal Medicine Student
    • FOLDING
    • FOLDING
    • N • Gene therapy is the use of DNA as a pharmacological agent in order to alter certain parts of the genome of a life being that are mutated or dysfunctional, to repair the protein non efficient synthesis.
    • Gene Therapy Using Lentivirus to Treat Wiskott-Aldrich Syndrome Promising
    • Gene Therapy Using Lentivirus to Treat Wiskott- Aldrich Syndrome Promising • The Wiskott-Aldrich Syndrome is caused by a mutation in a gene that encodes the protein WASP. • The most often used therapy is a bone marrow or stem cell transplant from a matching donor, and can be curative mostly for those whose donor is strongly matched.
    • Gene Therapy Using Lentivirus to Treat Wiskott- Aldrich Syndrome Promising An alternative is to use a form of retrovirus using gene therapy in the laboratory and apply it to the blood cells previously obtained from the patient so the retrovirus can correct the defect, and then give the corrected cells back to the patient.
    • Gene Therapy Using Lentivirus to Treat Wiskott- Aldrich Syndrome Promising This approach has been successful in a number of diseases, including who had Wiskott-Aldrich Syndrome. However, over the long term, some patients with immune deficiencies, including those with Wiskott-Aldrich Syndrome, developed blood cancers. In this new research, the team used a partially inactivated lentivirus to take the normal gene into the cell, while reducing the risk of the gene inserting next to a cancer- promoting gene.
    • This type of treatment instead of previous treatments using gene therapy with retroviruses is an amazing option for this type of patients, considering the lower risk of developing a cancer or other collateral diseases. This gene therapy also allows the patients to have a more normal and comfortable life increasing their life quality.
    • Gene Therapy Cures a Severe Pediatric Neurodegenerative Disease in Animal Models
    • Gene Therapy Cures a Severe Pediatric Neurodegenerative Disease in Animal Models • Sanfilippo Syndrome type A, is a neurodegenerative disease caused by mutations in the gene that encodes the enzyme sulfamidase. • The accumulation of glycosaminoglicans in the cells causes neuroinflammation and organ dysfunction.
    • Gene Therapy Cures a Severe Pediatric Neurodegenerative Disease in Animal Models A team of researchers of the UAB's Centre for Animal Biotechnology and Gene Therapy (CBATEG), has developed a gene therapy treatment that cures this disease in animal models, with pre- clinical studies in mice and dogs. The treatment consists of a single surgical intervention in which an adenoassociated viral vector, which is completely harmless, is injected into the cerebrospinal fluid, the liquid that surrounds the brain and the spinal cord.
    • Gene Therapy Cures a Severe Pediatric Neurodegenerative Disease in Animal Models Once the enzyme's activity is restored, glycosaminoglican levels return to normal for life, their accumulation in cells disappears, along with the neuroinflammation and dysfunctions of the brain and other affected organs, and the animal's behaviour and its life expectancy return to normal.
    • It’s overwhelming how the gene therapy has gone so far to the point of cure once and for all a neurodegenerative disease like Sanfilippo Syndrome type A. Although the treatment has not being studied in humans, the fact that it has been successful in animal models leaves us with great expectations of what’s coming next.
    • • Baylor College of Medicine (2013, July 11). Gene therapy using lentivirus to treat Wiskott-Aldrich Syndrome promising. ScienceDaily. Retrieved July 22, 2013, from http://www.sciencedaily.com-/releases/201 3/07/130711142403.htm • Universitat Autònoma de Barcelona (2013, July 2). Gene therapy cures a severe pediatric neurodegenerative disease in animal models.ScienceDaily. Retrieved July 22, 2013, from http://www.sciencedaily.com-/releases/201 3/07/130702100344.htm
    • THANKS