based one this rationale recommend me the title of this reprot Rationale
The purpose of this research investigation is to gather secondary evidence to find out how the use of Crispr technology results in an increase in foetal haemoglobin (HBF) against a single gene disorder: beta thalassemia. The chosen claim is: CRISPR technology offers new hope in medicine with a developed research question: How does CRISPR-Cas9 editing of the gene BCL11A affect the clinical nature of thalassemia, as measured by foetal haemoglobin levels and clinical outcomes in patients with transfusion-dependent β-thalassemia?
Crispr, Clustered Regularly Interspaced Short Palindromic Repeats, which is a component of the “bacterial” immune system, plays a role as molecular scissors, cutting DNA and reediting the gene or any transpiration factors (Synthego, 2024). Crispr-Cas9 is a revolutionary gene-editing technology that is an endonuclease that cuts specific parts of the genome by allowing precise alternations, alteration, insertion, or deletion of double-stranded DNA. It was chosen considering addressing the claim the best (Redman, King, Watson, King, 2016) and (Your Genome, 2024). Haemoglobin is a protein inside the red blood cells that facilitates oxygen to the tissues and returns carbon dioxide from the tissues back to the lung to be exhaled (National Cancer Institute, 2011). The differences between adult haemoglobin (HBA) and foetal haemoglobin (HBF) levels in terms of structural differences HBF contains two alpha and gamma subunits, whereas HBA contains two alpha and beta subunits (Kaufman, Khattar, and Lappin, 2023).
The single gene disorder, beta thalassemia, occurs when the normal body is not producing enough beta-globin, leading to symptoms like extreme tiredness. pale skin. shortness of breath (Numerous Kidshelath, 2022). “Pathophysiology of β-thalassemia: an imbalance in chain synthesis and an excess of freed α-globin chains accumulate within erythroid cells. The levels of HBF are affected by increased erythropoietin levels, leading to bone marrow expansion and increased F-cell production, combined with ineffective erythropoiesis, which gives a survival advantage to F cells” (Nienhuis and Nathan, 2012).
BCL11A is a transcriptional repressor that is crucial in brain and hematopoietic system development, as well as fatal-to-adult haemoglobin switching. In thalassemia, BCL11A represses y-globin expression and foetal haemoglobin in thyroid cells (Frangoul, Altshuler, Cappellini, Chen, Domm, Eustace, Foell, de la Fuente, Heandgretinger, Kattamis, Kernytsky, Lekstrom-Himes, Locatelli, Mapara, Montalembert, Rondelli, and Sharama, 2020).
The Crispr would treat thalassemia by transplanting the modified cells to correct genetic mutations in the form of haemoglobin F and red blood cells, causing hematopoietic stem cells (Goodman, 2024).
Swee Lay Thein's study demonstrated that genetic modifiers can influence the severity of sickle cell disease and beta-thalassemia. This impact was measured by HbF levels and alpha-globin genotypes. Therefore, the following research question has been developed (Thein, 2017).
Based on the provided rationale, a suitable title for your report could be:
"Transformative Potential of CRISPR-Cas9 in Beta-Thalassemia: Impact on Foetal Haemoglobin Levels Through BCL11A Gene Editing"
This title encapsulates the core focus of the research investigation, emphasizing the transformational potential of CRISPR-Cas9 technology in beta-thalassemia treatment by targeting the BCL11A gene to elevate foetal haemoglobin levels.
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