The use of CRISPR- cas has revolutionized the gene therapy field.One of the first uses of CRISPR- cas to edit genes in vivo was done at MIT.What disease did they cure?
A) B- thalassemia
B) RDEB
C) type 1 tyrosinemia
D) HIV
Correct Answer:
Verified
Q1: A serious concern about gene therapies and
Q2: Which gene therapy is currently approved in
Q3: Treatment of ADA- SCID is often considered
Q4: Despite the promise of gene therapy treating
Q5: ZFNs and TALENs have high specificity for
Q7: The gene PD- 1 plays an important
Q8: Gene therapy to treat Leber congenital amaurosis
Q9: CTL019 was the first gene edited CAR
Q10: What is the target of the first
Q11: Hematopoietic stem cells HSCs are particularly useful
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