Cystic FibrosisAPPLY WHAT YOU HAVE LEARNEDYou are a researcher working on a treatment for Hutchinson Gilford progeria syndrome, an extrernely raregenetic disorder that causes accelerated aging in children. Children with progeria generally appear healthyat birth but soon start growing more slowly than other children and lose their hair. Additional symptomsinclude stiffness of joints, heart problems, and stroke. These children typically die of heart disease at anaverage age of 13 years.5.Progeria is caused by a mutation in a single gene, called lamin A. Scientists have identified over 1,400mutations in the lamin A gene that result in changes in transcription, RNA splicing, and/or proteinproduction. Lamin A codes fora protein required for the structural support of the nuclear envelope in cells.Without a functional protein, the nuclear envelope becomes unstable, eventually damaging the nucleusand causing cells to die,Based on what you learned in this Click & Learn, propose a genetic medicine strategy you could develop totreat patients with progeria. Describe which step in gene expression you might target and why you wouldtarget that step, the intervention tool you would use, and explain how this strategy would treat the disease.637 wordsOS English (Philippines)acer

Name: Cystic FibrosisAPPLY WHAT YOU HAVE LEARNEDYou are a researcher workin
Uploaded: 2024-03-20T06:42:41.000Z
Channel: Bartleby
Description: Cystic FibrosisAPPLY WHAT YOU HAVE LEARNEDYou are a researcher working on a treatment for Hutchinson Gilford progeria syndrome, an extrernely raregenetic disorder that causes accelerated aging in children. Children with progeria generally appear hea