On the Cusp of a Scientific Revolution: A Message from Anthony J. Angelo, MD Chief Medical Officer

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  •  September 7, 2017
  •  News

It almost sounds like science fiction, but what I am about to describe is real science. I’m referring to a gene-editing tool known as CRISPR – the acronym for Clustered Regularly Interspaced Short Palindromic Repeats.

Why should we pay attention to CRISPR? Just as the Internet revolutionized the way we communicate, today more than 10,000 companies are studying CRISPR to learn how to better treat chronic and complex diseases. It is my belief that current research and its implications have the potential to profoundly change the lives of the individuals that we at Federation serve.

At some point within the next decade (maybe as soon as five years from now), the CRISPR tool may be used to help treat schizophrenia, depression, autism, and developmental disabilities in fundamentally new ways.

No matter how you view the notion of genetic modification – a part of science that sets out to change the genetic makeup of cells – I encourage you to become familiar with what is happening in this area of science.

To understand CRISPR, think of it as a piece – or, snippet – of a chromosome in a bacterium that is being invaded by a virus. The snippet takes a piece of viral DNA (or RNA) and copies it into its own DNA. The snippet is then used to detect and destroy DNA from further attacks by similar viruses.

Without going into a deeper explanation of the science, let’s focus on some recent discoveries from this tool.

Scientists are working on a fungus-resistant wheat that can feed and nourish 100 times more people than the capacity of current wheat producers. Drought-resistant corn is also being tested. Sugars are being converted into fuel.

When it comes to tropical diseases and the deadly Zika virus, scientists are figuring out how to modify the reproductive ability of mosquitos that carry these diseases. If successful, this will be a huge breakthrough in eliminating the spread of such diseases.

The CRISPR tool is giving hope to people with genetic conditions. Scientists are experimenting to snip out genetic heart conditions, blindness, muscular dystrophy, and multiple sclerosis. Scientists have been successful in eliminating the HIV virus in mouse cells using CRISPR/Cas9. An even more precise tool, the CRISPR/Cpf1, which cuts on a bias and allows chromosomes to reknit themselves, decreasing the possible entry of unwanted chromosomes, is being used to determine how to destroy cancer cells that have been resistant to treatment.

What are the implications for individuals with developmental disabilities? We all know that most genes are the same in all people, but a small number of genes are slightly different between people. The small differences contribute to each person’s unique physical features. What if gene modification could help people who suffer from depression or schizophrenia and a whole range of developmental disabilities?

We at Federation know the challenges that people with developmental disabilities face. Scientists are working to safely and naturally help alleviate suffering. I, for one, am very excited about the possibilities.

For now, however, we must focus on our work at hand. Let us all continue to work diligently to help the individuals and families who come to us seeking more fulfilling lives. And let us hope that, in the near future, science will ease their burdens and ultimately make their lives much easier.

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