KJ -a child affected by a rare inborn error of metabolism- became the first patient ever to receive a  gene editing drug specifically designed for the genetic variants he has. The drug was used for somatic gene editing, aiming at modifying only KJ's liver cells.

Carbamoyl-phosphate synthase 1 deficiency (CPS1) is caused by defects in the CPS1 gene. Which type of protein is encoded by the CPS1 gene and where in the body the gene is mostly expressed?

Choose an adjective that, in your opinion, better describes Dr He Jankui professional behavior

After reading the two stories (CRISPR'd babies: human germline genome editing in the 'He Jiankui affair' and "The Future of Personalized Medicine is Here: KJ’s Story") list 2 facts from each article and 1 question you have about each of these two gene-editing procedures or their implications.

You may organize your answer like this:

CRISPR'd babies

• Fact1:
• Fact2:
• Question:

KJ story

• Fact1:
• Fact2:
• Question:

CRISPR-Cas9 genome editing could be used to repair mutations in inherited genetic diseases such as cystic fibrosis or sickle cell anemia.

What makes the CRISPR/Cas technology revolutionary compared to other genome editing techniques?

The type of DNA repair process that uses template DNA to guide the rebuilding process and either repairs a defective gene or inserts a new sequence after the DNA is cut is called

The type of double-strand break DNA repair process that is prone to errors and can lead to extra or missing bases after the DNA is cut is called

In the engineered CRISPR/Cas9 system, what is the name of the specific nucleotide sequence that directs Cas9 to the target gene to be modified?