You are a researcher working on a treatment for Hutchinson-Gilford progeria syndrome, an extremely rare genetic disorder that causes accelerated aging in children. Children with progeria generally appear healthy at birth but soon start growing more slowly than other children and lose their hair. Additional symptoms include stiffness of joints, heart problems, and stroke. These children typically die of heart disease at an average age of 13 years.

Progeria is caused by a mutation in a single gene, called lamin A. Scientists have identified over 1,400 mutations in the lamin A gene that result in changes in transcription, RNA splicing, and/or protein production. Lamin A codes for a protein required for the structural support of the nuclear envelope in cells. Without a functional protein, the nuclear envelope becomes unstable, eventually damaging the nucleus and causing cells to die.

Based on what you learned in this Click & Learn, propose a genetic medicine strategy you could develop to treat patients with progeria. Describe which step in gene expression you might target and why you would target that step, the intervention tool you would use, and explain how this strategy would treat the disease

If predominantly, we can't say of any treatment for progeria, aside from the routine observation of heart and blood vessel (cardiovascular) disease which is important in taking care of the health situation in the affected kids. Treatment relies majorly on the situation and clinical manifestations of the kid. At first, in 1999, the findings of the initial clinical drug trial for kids with an unusual rapid-aging disease, often referred to as Progeria, revealed positive results with a farnesyltransferase inhibitor (FTI), a drug initially designed to treat cancer.

The PATHWAY in gene expression to be TARGETED is progenin formulation---

The Progeria-forming mutation results in the formation of the protein progerin that disrupts the cell activities. Some areas of progerin's toxic impact on the body is due to the formation of a farnesyl group, Â that adheres to the progerin protein aiding it's disruption of the body's cells. FTIs means of action is through inhibition of the adhesion of the farnesyl group to progerin, decreasing the hazardous substances that leads to progerin.

The genetic medicine technique that acts as.an intervention tool gene therapy to regulate the gene that causes the stimulation and formation of the protein progenin.

Escalated cardiovascular disease results in death in the case of Progeria.

It's worthwhile to note that if the basic blood vessels can with time get better as a result of treatment, the treatment shows positive results. Slowing down stroke and aging manifestations will boost the chances of treatment.

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