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CRISPR Will Change Our Lives

By Shlomo Maital

   Some time ago, I wrote about a technology known as Blockchain that will undoubtedly change our lives – and has already. Blockchain is simply a way to record transactions, that is secure, unhackable and ‘disintermediated’ (no need for banks or other financial middlemen). It is now widely used to create digital money.

   Now comes CRISPR. It stands for Clustered Regularly Interspaced Short Palindromic Repeats.     What is it? Simply, a way of editing genes – like we edit blogs or emails. Erase this group of words. Replace it with another, better one.   In the case of DNA: “Erase” (snip away) this (bad) piece of DNA, a gene that causes problems – and replace with a ‘good’ gene, that will not cause disease or problems.

   A palindrome is a word that reads the same forward and backward.   E.g. “civic”. So pieces of DNA are inserted into longer pieces, such that the inserted pieces read the same forward or backward, so it doesn’t matter which way they are inserted. The method originated with studying how viruses ‘snip’ DNA – and using viruses to do the same in constructive way.

     We now know the genetic causes of many diseases. But until now we have not had the ability to repair bad genes. Now CRISPR makes it possible. This will create an entirely new branch of medicine, immunotherapy, in which gene therapy is used to both treat illness, when identified, and mainly, to prevent it — an individual can now have his or her genome analyzed, and potential ‘bad’ genes identified. No point in doing this, so far, because there was no real way to ‘edit’ bad genes. Now, with CRISPR, there is.

     I would like to mention one of the scientists responsible for CRISPR, the young MIT scientist Feng Zhang. He was born in China and is only 36; he does research at the famous Broad (pronounced Brode) institute in Cambridge, MA. Increasingly bright foreign students are encountering US visa problems and are going elsewhere, e.g. Canada. It is America’s loss.

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Fixing Bad Genes: A First!

By Shlomo Maital

Shoukhrat Mitalipov  

   Virtually at the same time — President Trump proposes legislation to limit immigration to the U.S. and an immigrant from Kazakhstan named Shoukhrat Mitalipov manages, for the first time, to ‘edit’ mutated genes in embryos and ‘cure’ genetic disease, enabling the babies to be born healthy.

     This is hugely significant. Parents who undergo genetic testing, and who test their unborn babies, are at times tragically told: Your unborn infant carries a ‘bad’ gene that will make him sick or even die.

       The parents ask: What can we do?

       The tragic answer, until now — Nothing. Abort or —     accept its fate.

         There have been 3 previous published instances of gene editing, all in China.   According to MIT Technology Review:   “Now Mitalipov is believed to have broken new ground both in the number of embryos experimented upon and by demonstrating that it is possible to safely and efficiently correct defective genes that cause inherited diseases. Although none of the embryos were allowed to develop for more than a few days—and there was never any intention of implanting them into a womb—the experiments are a milestone on what may prove to be an inevitable journey toward the birth of the first genetically modified humans.”

   How did he do it?

   Mitalipov and his team used CRISPR Clustered regularly interspaced short palindromic repeats. CRISPR is now the dominant technology for gene editing. Try as I might, I do not know enough biology to explain how it works. The initial work was done in Japan, at Osaka University.

     CRISPR often makes mistakes – disastrous if you are trying to heal a bad gene. Mitalipov figured out how to avoid those mistakes.

   Who is he?

   “Born in Kazakhstan when it was part of the former Soviet Union, Mitalipov has for years pushed scientific boundaries. In 2007, he unveiled the world’s first cloned monkeys. Then, in 2013, he created human embryos through cloning, as a way of creating patient-specific stem cells.”

       Is it coincidence that Mitalipov is an immigrant? I don’t think so. By definition an outsider,   such ‘outsiders’ try daring things that those comfortably within the establishment might not.

     Is there any reason to worry?

   Yes.  We can genetically modify embryos. We can do this to cure gene defects – or to modify embryos to make taller kids (NBA), smarter kids (Einsteins), or handsomer kids, or blonde kids… etc.

       “Some critics say germline experiments could open the floodgates to a brave new world of “designer babies” engineered with genetic enhancements—a prospect bitterly opposed by a range of religious organizations, civil society groups, and biotech companies.  The U.S. intelligence community last year called CRISPR a potential “weapon of mass destruction.”

 

 

Blog entries written by Prof. Shlomo Maital

Shlomo Maital
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