Therapies

Question 1

CRISPR

Answer 1

clustered regularly interspaced palindromic short repeats

Question 2

Steps of CRISPR

Answer 2

1. CRISPR/cas9 identifies the target DNA using guide RNA
2. CRISPR/cas9 cuts the target DNA introducing a double strand DNA cut
3. Scientists can now edit the gene through DNA deletion, modification and addition

Question 3

How can be regulate gene expression?

Answer 3

cas9 can be fused to an effector domain

Question 4

1st CRISPR-based gene editing therapy

Answer 4

Casgevy for sickle cell anemia