You wish to perform gene therapy to correct a mutated gene to wildtype in an adult...

You wish to perform gene therapy to correct a mutated gene to wildtype in an adult organism. You decide to use the new CRISPR system to accomplish this task. Three important CRISPR-related components must be included in the virus that will be used to treat the subject. What are these three components, and what is the function of each?

Solutions

Expert Solution

CRISPR “spacer” sequences are transcribed into short RNA sequences (“CRISPR RNAs” or “crRNAs”) capable of guiding the system to matching sequences of DNA. When the target DNA is found, Cas9 – one of the enzymes produced by the CRISPR system – binds to the DNA and cuts it, shutting the targeted gene off.

CRISPR relies on just two components: the molecular scissors, a CRISPR-associated (Cas) nuclease, and the GPS guiding it to the appropriate site, the guide RNA (gRNA). In natural, the gRNA consists of two distinct segments of RNA: CRISPR RNA (crRNA) and transactivating CRISPR RNA (tracrRNA).

Cas nuclease Cas9 (CRISPR associated protein 9) is a protein which plays a vital role in the immunological defense of certain bacteria against DNA viruses and plasmids and which is heavily utilized in genetic engineering applications. Its main function is to cut DNA and therefore it can alter a cell's genome.

The guide RNA is a specific RNA sequence that recognizes the target DNA region of interest and directs the Cas nuclease there for editing. ... The CRISPR-associated protein is a non-specific endonuclease.

gladiator answered 1 year ago

Next > < Previous

Related Solutions

Gene therapy attempts to correct a genetic deficiency by inserting a normal version of the affected...

Gene therapy attempts to correct a genetic deficiency by inserting a normal version of the affected gene into the patient's cells. For this to be effective, the cells need to be _______cells that will not passed on to offspring, but they also need to be cells that will keep dividing for the lifetime of the patient. Currently, ______ cells, which produce all the blood and immune cell types of the body, are a preferred target.What limits the use of gene therapy...

Construct a recombinant bacteria strain with a gene mutated. You need to design and suggest the...

Construct a recombinant bacteria strain with a gene mutated. You need to design and suggest the steps. 1) cloning of gene 2) mutation (how to do it) 3) which homologous recombinant method you use 4) how to verify the strain EXPLAIN THE STEPS IN DETAIL.

1) The WILDTYPE DNA/gene has the base sequence: 5’GTACTGCAT3’ (the antisense strand of the gene is...

1) The WILDTYPE DNA/gene has the base sequence: 5’GTACTGCAT3’ (the antisense strand of the gene is shown) The gene has undergone a mutation. The mutant DNA/gene has the base sequence: 5’GTACTCCAT3’ (the mutation is in BOLD) based on this information, answer the following 1a)What is the base sequence of mutant mRNA? Label the ends of the mRNA 1b) this mutation is most likely to be caused by: a. soot b. analogues c. X-Ray d. RNA polymerase e. ribosome (pick one)...

The following sequences show the wildtype and mutant alleles of part of a gene. What is...

The following sequences show the wildtype and mutant alleles of part of a gene. What is the name of this type of mutation? Wildtype DNA: 5’ AAC – AGC – CTG – CGT – ACG – GCT – CTC 3’ Wildtype protein: Asn – Ser – Leu – Arg – Thr – Ala – Leu Mutant DNA: 5’ AAC – AGC – CTG – CTT – ACG – GCT – CTC 3’ Mutant protein: Asn – Ser –...

Genetics: How does gene therapy work? What are the challenges of gene therapy? Identify a disease...

Genetics: How does gene therapy work? What are the challenges of gene therapy? Identify a disease state that could be treated with gene therapy. There are several ethical issues surrounding gene therapy. Pick one and state whether you agree or disagree with the position. Explain your answer. There are both highs and lows in gene therapy. Research the stories of Ashanti de Silva and Jesse Gelsinger. Based on the outcomes of these treatments, do you think experimental gene therapy trials...

Describe gene therapy? Choose any human condition that is currently being treated with gene therapy. Identify and...

Describe gene therapy? Choose any human condition that is currently being treated with gene therapy. Identify and describe the genetic deficiency that the strategy is intended to address. Describe the proposed gene therapy procedure. Identify successes, failures, or observed adverse consequences that have been associated with this condition in gene therapy in the past or currently. 100 words minimum

If A a transcription factor or B a response element in a gene were mutated and...

If A a transcription factor or B a response element in a gene were mutated and not functional, how could transcriptomics by RNA-seq or cDNA microarrays be used to find which gene(s) was/were affected? Which method (RNA-seq or microarrays) is better and why? Explain your answers for both A and B in detail and be sure to address how the results of A versus B would likely differ from each other.

One of the genes that is commonly mutated in skin cancer is the gene that codes...

One of the genes that is commonly mutated in skin cancer is the gene that codes for the p53 protein. If both copies of the gene are mutated by UV light and the p53 protein is no longer functioning in a skin cell, the cell is not necessarily cancerous despite these mutations. However, descendants of that cell are likely to develop into cancer. Explain why having defective p53 proteins might lead to cells with many mutations present – a cancer-prone cell.

The normal (wildtype) size of the brat gene is 1500 base pairs (bp). In the mutant...

The normal (wildtype) size of the brat gene is 1500 base pairs (bp). In the mutant variant you identified, there is a deletion of 500 bps in the last exon of the gene. How would you recognize the two versions based on size? List what fragment sizes you would expect to see on a gel.

1) You wish to clone a gene into a bacterial plasmid for the production of the...

1) You wish to clone a gene into a bacterial plasmid for the production of the E protein to generate antibodies, use in vaccine production, and for biochemical analysis. A. What expression plasmid will you use and why? B. What primers to facilitate cloning into this expression vector? C. What essential features (DNA sequence elements) must the plasmid have to ensure: i. Plasmid maintenance/amplification in bacteria ii. Selection of cells transformed with your plasmid iii. Controlled/inducible expression of Gene E...

Subjects