Lorenzo's Oil Case Study

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“Lorenzo’s Oil” Alternate Assignment Answer the following questions in complete sentences. You may type into this file or write your answers on a separate piece of paper. Use this link to help you complete this task: www.myelin.org 1) Describe what Adrenoleukodystrophy (ALD) is and how it relates to our genetics unit. ALD is a rapidly progressive X-linked genetic disorder marked by the accumulation of saturated very long chain fatty acids (VLCFA) in all tissues and body fluids, with a preference for the brain and the adrenal glands. This buildup leads to inflammation of the brain and erosion of the white matter of the central nervous system and the adrenal glands. It affects only boys; while there is a neonatal form, it typically…show more content…
Removing VLCSFA from the diet eliminates any external contribution to the total VLCSFA blood level. Lorenzo’s Oil, made of monounsaturated fatty acids (good fatty acids), causes Competitive Inhibition to occur. The same enzyme complex that elongates fatty acid chains becomes occupied with the production of monounsaturated fatty acids. The enzyme complex cannot produce both types of fatty acids simultaneously. Therefore the two kinds of fatty acids compete for same enzyme complex; however, monounsaturated fatty acids are formed in greater concentration than VLCSFA. The reduction in the elongation of VLCSFA and a diet low in VLCSFA will slow the demyelination…show more content…
Gene therapy - One of the most promising treatments on the horizon for ALD is gene therapy - This involves temporarily removing the appropriate cells from the person with ALD, inserting the corrected genetic sequence with the appropriate blueprints into these cells and putting them back into the patient. The repaired cells will then produce the protein that had been missing or defective prior to treatment, and the disease process will halt or moderately reverse. Mesenchymal Stem Cell Therapy - Scientists at St. Jude’s Hospital, the Children’s Hospital of Philadelphia, Tulane University School of Medicine, and leading institutions in Germany are also studying the use of mesenchymal stem cells in treating ALD. These cells are multipotent precursor cells that can differentiate into many different types of cells. Cultured from adults’ bone marrow, they may have promise in treating ALD when delivered directly into the blood and brains of people with the

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