Write a critical review for a final year gene regulation undergraduate assignment on the therapeutic potential of CRISPR/Cas9 in treatment of sickle cell disease with the following structure:
1. Introduction (brief ~10-15% of word count):
a. CRISPR/Cas9 technology
b. Types of CRISPR/Cas9 gene modification & how they differ
c. How the technologies work
d. Why it is potentially useful therapeutically
e. Briefly introduce TALENs and ZFNs as alternatives, but not too much detail.
f. Sickle cell disease – what it is, why is it important, why do we need new treatments?
2. Main Review: Critical review of primary research articles & clinical trial data on how CRISPR/Cas is being used to treat sickle cell disease?
a. Do these studies use CRISPR in the same way i.e., to target the same gene?
b. Do these studies support each other or do they have differing results?
c. How big are the clinical trials? What are the trials showing right now?
3. Limitations: Current limitations to clinical translation of CRISPR methodology and ways in which people are trying to address these with specific examples.
4. Conclusion (5-10% of word count)
Please:
- Include reviews of not only preclinical (i.e., cell culture based and/or animal studies) but also clinical trial data.
- Ensure all statements are backed up by evidence from the literature – not just from reviews, I need primary references as well
- Can include Table/Figures, e.g., images of how CRISPR/Cas works or illustrating sickle cell disease – but modify images from other sources and reference them
- Include 15 or more references