Mutations in the DNA can affect the structure and function of proteins. Some mut

Question

Mutations in the DNA can affect the structure and function of proteins. Some mutations may even cause genetic diseases. Scientists and doctors can intervene at different points during gene expression to develop treatments for such genetic diseases—or genetic medicine. Let’s learn about the genetic medicines that are being developed.

Select the “Genetic Medicine” tab located on the top right of the screen. Click on the tab corresponding to the genetic medicines, or scroll through the interactive and click on the pink “+” sign labeled with that genetic medicine. Read the “Genetic Medicine” tab material, watch the video, and read the information in the “Learn more” link. Pick 3 genetic medicines you are interested in and complete the rows below in your own words.

Genetic Medicine

Short Summary

Write a one-sentence summary in your own words of how this genetic medicine works.

Detailed Description

Describe in your own words how this genetic medicine would be used to treat a genetic disease. (For example, mention how it would fix the disease-causing mutation and/or result in a functioning protein.)

CRISPR-

Cas9

Gene Therapy

Gene Switches

Exon Skipping

RNA

Interference

Small Molecule Drug

Genetic Medicine

Short Summary

Write a one-sentence summary in your own words of how this genetic medicine works.

Detailed Description

Describe in your own words how this genetic medicine would be used to treat a genetic disease. (For example, mention how it would fix the disease-causing mutation and/or result in a functioning protein.)

CRISPR-

Cas9

Gene Therapy

Gene Switches

Exon Skipping

RNA

Interference

Small Molecule Drug

Explanation / Answer

Since the question is asking for any 3 I will be concentrating on the current trends in this field.

CRISPR cas9 - it is a molecular scissor unique to bacteria that contain short segments of sequences that correspond to the virus which attack the bacteria. During the attack the bacteria can idenfy the virus and eliminate it using this system.

Application

Knockdown mechanism of CRISPR used dead cas 9 which doesnot have cutting capability. However targeting capability of the system still remains. So epigenetic modification of the desired gene through methylation allows the blockage of transcription of the gene. By using this method we can stop a derised gene from being transcribed at all and thus the protein (if harmful) will not be generated. This is a good way to silence specific genes. This can be used in progpraming a foetus that should traits for a genetic disorder carried forward from its parents. Conversely this system is reversible and transcription can be activated using CRISPR mediated activators.

Gene therapy- or gene replacement therapy is when you introduce a normal gene into the cell to replace a defective or missing gene.

Application - most common example would be that of the CFTR gene associated to cystic fibrosis. In cystic fibrosis, the mucosal lining of the respiratory system thicken and thereby block the passage of air due to the non functioning of the CFTR gene. In this form of gene therapy, the functioning copy of the gene is inserted into a suitable vector and then introduced in the nasal epithelium. This allows for the clearing og the nasal passage and will allow the subject to breathe. However this process needs to be administered repetitively.

RNAi- occurs when RNA molecules can inhibit gene expression by by suppressing the mRNA through complementarity.

Application- In this method a double stranded RNA (sense and anti sense) complementry to the sequence of the gene that we want to silence is introduced to the cell. Eg. we can target cancer specific genes. the cell recognizes the insert as exogenous material and activates its RNAi pathway. The RISC factor would get associated to the corresponding siRNA and then allow for the silencing of the target mRNA and thus the gene won't be expressed.

Related Questions

Navigate

• Browse (All)
• Browse M
• Subjects

Integrity-first tutoring: explanations and feedback only — we do not complete graded work. Learn more.