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<p>Gene therapy is the therapeutic delivery of nucleic acid into a patient&rsquo;s cell as a drug to treat disease. There are 3 main methods for gene therapy.</p>

<p style="margin-left:20.0pt">Using adenovirus vector, new gene can be inserted in patient&rsquo;s cell. It can transduce both non-diving and dividing cells. Carry up to 8Kbp heterologous DNA, and ensure high levels of transgene expression.</p>

<p style="margin-left:20.0pt">But this adenovirus vector has disadvantages. 1) It is highly immunogenic. 2) Vector genome does not integrate into the host genome, so it is not permanent therapy. 3) Hard to control expression levels 4) Hard to &ldquo;Knock-out&rdquo; host gene.</p>

<p style="margin-left:20.0pt">RNA interference (RNAi) is a biological process in which RNA molecules inhibit gene expression or translation by neutralizing targeted mRNA molecules. It can effectively silence specific target gene. In August 2018, FDA approved the first RNAi drug (Onpattro). RNAi can be used even if the mRNA is not translated to protein. If GWAS data gives a information about specific mRNA is associated with a disease, RNAi can inhibit it.</p>

<p style="margin-left:20.0pt">&nbsp;</p>

<p style="margin-left:20.0pt">CRISPR (<u>C</u>lustered <u>R</u>egularly <u>I</u>nterspaced <u>S</u>hort <u>P</u>alindromic <u>R</u>epeats) / Cas9 (<u>C</u>RISPR- <u>as</u>sociated protein 9) is a unique technology that enables geneticists and medical researchers to edit parts of the genome by removing, adding or altering sections of the DNA sequence. It edits host genome, so therapy with CRISPR/Cas9 is permanent. Both &ldquo;Knock-out&rdquo; and &ldquo;Knock-in) are possible. It can edit specific loci. So, I think this gene editing is very powerful tool to adjust treatment from GWAS data.</p>

<li><a href="https://www.yourgenome.org/facts/what-is-crispr-cas9">https://www.yourgenome.org/facts/what-is-crispr-cas9</a></li>

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