Hi, my name is Timothy, and today, I would persuade you to give me the grant money for Hemophilia. So, what is hemophilia? Hemophilia is caused when the person lacks clotting factor 14, or in other words, the F8 gene on the 28th chromosome have been mutated, which causes it to malfunction so the protein coagulation factor VIII cannot be produced.
So there is no clotting factor, what does that mean? Well, with no or little clotting factor, Hemophilia victims tend to bruise easily, have excessive bleeding, spontaneous bleeding, and bleed in there joints. This may seem terrible, but in reality, we only see this disease on a basic level. We cant see the troubles it brings us. Currently, the treatment for hemophilia for a year is about 40,000 american dollars, but since these victims would need special care and special facilities, it would cost around
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But first, let me explain what some of terms. CRISPR-CAS9 is a gene editing tool that enables scientists to freely edit the human genome by changing or editing the gene sequence. CRIPSR-CAS9 is currently the simplest, most flexible, and most precise method of gene editing. So how does CRISPR work? The CRISPR system has 2 key parts. Part one is an enzyme called CAS-9, this is basically a pair of molecular scissors that can cut the two strands of DNA at a specific location in the genome so that bits of DNA can then be added or removed. The second part is a piece of RNA called guide RNA, this includes a small piece of pre-designed RNA sequence located within a longer RNA strand. The strand part binds to DNA and the pre-designed sequence ‘guides’ Cas9 to the right part of the genome. This makes sure that the Cas9 enzyme cuts at the right point in the genome. The guide RNA is designed to find and bind to a specific sequence in the DNA. This means that, the guide RNA will only bind to the target sequence and no other targets, therefore making it completely accurate and