Diagnosis to Treatment
A fever measured by a doctor is a symptom or a sign?
Sign
What treatment trains the immune system before disease strikes?
Vaccine
Which type of cholesterol is described as the “bad” cholesterol?
LDL
How long did baby KJ's gene editing therapy took to get approved?
6 months
What do we call a change felt and reported by the patient, such as nausea or fatigue?
Symptom
What type of treatment uses lab-made proteins or antibodies?
Biologic
If PCSK9 activity decreases, what happens to the number of LDL receptors?
More LDL receptors survive.
Which enzyme was deficient in baby KJ?
CPS1
Put these in order: diagnosis, treatment, testing, patient complaint.
Patient complaint → testing → diagnosis → treatment
A disease is caused by a defective gene. Which treatment category could potentially address the underlying genetic cause rather than only managing symptoms?
Gene therapy
What are some current therapies for hyperlipidemia (high cholesterol)?
Oral medicines, biologics - PCKS9 inhibitors
How many nucleotides were mutated in baby KJ that caused his disease? (nucleotide = letters in the DNA)
just one!