W8(2): CRISPR Cas9

Question 1

How are breaks/cleavages in the DNA repaired

Answer 1

NHEJ (non-homologus end joining)

Question 2

How has CRISPR cured blood disorders (eg. sickle cell anaemia & thalassaemia)

Answer 2

CRISPR was used to effectively “turn on” Fetal haemoglobin
* High HbF expression is associated with low morbidities
* HbF replaced HbA (adult) –> patients no longer needed blood transfusions