Which type of disease is LEAST likely to be able to be treated using genome editing with TALEN or CRISPR?

Question

A.  Trisomy-21
B.  Leukemia
C.  Cystic fibrosis
D.  Huntingtons

Clarify Question
· What is the key concept addressed by the question?
· What type of thinking is required?

  Gather Content
· What do you already know about genome editing?

  Choose Answer
· Given what you now know, what information and/or problem solving approach is most likely to produce the correct answer?
   Reflect on Process
· Did your problem-solving process lead you to the correct answer? If not, where did the process break down or lead you astray? How can you revise your approach to produce a more desirable result?

Answer 1

A.  Trisomy-21

Clarify Question
· What is the key concept addressed by the question?
        o This question addresses genome editing.
· What type of thinking is required?
        o This question is asking you to weigh and judge evidence, or evaluate, to choose the best of the possible answers.

  Gather Content
· What do you already know about genome editing?
        o Genome editing uses either a TALE protein or an RNA to target a nuclease to a particular gene in the genome.
        o The nuclease then cuts the targeted DNA, which can be repaired to leave a deletion, or repaired to incorporate a new desired sequence of DNA.

  Choose Answer
· Given what you now know, what information and/or problem solving approach is most likely to produce the correct answer?
        o All of the diseases listed originate with a single abnormal gene that could be targeted by genome editing, except for trisomy 21, which involves a whole extra chromosome.
        o It would be difficult to regulate the expression of a whole chromosome using the single gene targeting techniques of genome editing.
   Reflect on Process
· Did your problem-solving process lead you to the correct answer? If not, where did the process break down or lead you astray? How can you revise your approach to produce a more desirable result?
        o The question required you to weigh and judge evidence, or evaluate, to choose the best of the possible answers.
        o Did you recognize that genome editing works by targeting and modifying a particular gene in the genome? It would be difficult to use the technique to target multiple genes – or a whole chromosome – at once.