subject
Biology, 26.02.2021 09:00 lychee1406

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, 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.

ansver
Answers: 2

Another question on Biology

question
Biology, 22.06.2019 03:00
During the day, plants produce by splitting water molecules in the light-dependent reactions of photosynthesis. at the same time, plants use cellular respiration to produce some of the needed by the light-independent reactions to make sugars. during the night, plants produce because takes place.
Answers: 3
question
Biology, 22.06.2019 05:00
Asea turtle has washed up on a remote section of a beach. this known as a stranding occurs when a dead,sick or injured sea turtle washed up on the shoreline. which statment best explain why stranding should be reported immediately to local authorities
Answers: 1
question
Biology, 22.06.2019 05:20
Categorize each term as something that is typical of a scientific theory, a scientific hypothesis, or both. makes predictions
Answers: 2
question
Biology, 22.06.2019 11:30
Which of the following statements is true about the relationship between genetic variation and natural selection? a. genetic variation must be present in a population before natural selection can act on it. b. genetic variation arises in a population as a result of natural selection. c. natural selection can act on a population whether there is genetic variation within the population or not. d. after natural selection acts on a population, the amount of genetic variation in the population always increases.
Answers: 1
You know the right answer?
You are a researcher working on a treatment for Hutchinson-Gilford progeria syndrome, an extremely r...
Questions
question
Mathematics, 10.03.2020 07:09
question
Mathematics, 10.03.2020 07:10
question
Mathematics, 10.03.2020 07:10
Questions on the website: 13722362