Question

1. You are a genetic counselor and the year is 2035. All of the CRISPR technologies discussed in BIOL A252 have become safe and reliable for therapeutic use in humans. One of your patients presents a wasting disease where they fail to produce enough collagen in their bones. You find no mutations in the coding region of the collagen gene and the protein is at healthy levels in all other tissues. Fully explain the steps that you would suggest to treat the disease, including what molecules are necessary for each step?

2. Briefly describe two ethical concerns and/or potential societal impacts of genetic engineering.

Answer 1

Muscle wasting occurs with aging and ina wide range of catabolic diseases, suchas cancer, diabetes, chronic renal disease,and heart failure, which can lead to adramatically reduced quality of life and increased disease mortality. Several treat-ments are currently in development as result of our understanding of the cellular mechanisms that cause muscle wasting.Myostatin, encoded by the MSTN gene,is a well-known cytokine that is activated in most disease conditions associatedwith wasting muscle.Myostatin is primarily secreted by skeletal and muscle cells mainly through anaparacrine mode by activatingthe SMAD2/SMAD3 signaling pathway transcription programs leading to muscle atrophy.1, 2Inhibition of myostatin has been identified as a promising strategy to alleviatemuscle wasting.

Social impact of genetic engineering .

World wide epidemic spread.

Development of more bio weapons.