Question

In: Biology

1. Iris reads a media article in the New York times that describes how CRISPR is...

1. Iris reads a media article in the New York times that describes how CRISPR is being used to treat genetic disease. She asks her friend if CRISPR could someday be used to cure Wilson’s disease. For CRISPR to be a viable option for curing Wilson’s disease, which of the following must be possible? SELECT ALL

The full sequence of the mutated atp7b gene must be known.

The full sequence of the normal ATP7B gene must be known.

Patients’ liver cells would have to be able to be removed and later reintroduced into the body.

The STRs flanking either side of the mutated atp7b gene must be known.

2.

At which point during ATPB7B gene expression does the copper-transporting ATPase 2 ER signal get manufactured? What would be the result if the SRP is inhibited such that it cannot bind to the ER signal?

Before transcription. So transcription would not occur.

After transcription…during pre-mRNA processing. So processing would not occur.

After pre-mRNA processing. So processing would not be completed.

After pre-mRNA processing…during translation. So the secondary and tertiary structure of the protein would not be made.

After translation. So the protein wouldn’t be specialized.

Solutions

Expert Solution

1. Iris reads a media article in the New York times that describes how CRISPR is being used to treat genetic disease. She asks her friend if CRISPR could someday be used to cure Wilson’s disease. For CRISPR to be a viable option for curing Wilson’s disease, which of the following must be possible?

The full sequence of the mutated atp7b gene must be known.

(CRISPR need to know the sequence of mutated DNA in order to make changes in the guide RNA and thus needs to be introduced to the target cells)

CRISPR :

Clustered regularly interspaced short palindromic repeats

It is a family of DNA sequences found in the genomes of prokaryotic organisms such as bacteria and archaea.

They are derived from DNA fragments of bacteriophages that had previously infected the prokaryote.

Used to detect and destroy DNA from similar bacteriophages during subsequent infections.

Wilson disease

It is an autosomal recessive inherited disorder of copper metabolism

The responsible gene for Wilsons disease, ATP7B

2. At which point during ATPB7B gene expression does the copper-transporting ATPase 2 ER signal get manufactured? What would be the result if the SRP is inhibited such that it cannot bind to the ER signal?

After transcription…during pre-mRNA processing. So processing would not occur.


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