Geneplex
Betting on the first disease
To be treated by gene editing
Alexandra Ossola
Special to CNBC.com
Wednesday, 15 Mar 2017
[... ] Mark Kay, a professor of pediatrics and genetics at Stanford University, is placing his bet on a treatment for rare immunodeficiency conditions, such as the "bubble boy" disease, which he says could come about in the next five years or even sooner.[1]
Why Genetic Modification
Is About To Get Easier
Is About To Get Easier
These diseases top researchers' lists because they are usually caused by a single, clearly defined mutation, giving researchers an obvious target to cut out or replace with functional DNA.
But anticipating when CRISPR could be used to develop a cure is more difficult to assess. [1]
There are not only technical hurdles to overcome but ethical ones.[1]
An influential U.S. science advisory board recently, and for the first time, gave cautious approval for gene editing in embryos to prevent diseases such as Huntington's and Tay-Sachs, but not for noncritical research efforts, such as the so-called designer babies or even for diseases for which there is an alternate treatment.[1]
"When people talk about a cure for whatever disease [with CRISPR], that's a tough one," said David Edgell, an associate professor of biochemistry at the University of Western Ontario.[1]
Scientists are first looking to use gene therapy tools on a certain number of cells to treat a disease, a more attainable goal.[1]
Some treatments, such as those for cancer involving modified T-cells and blood diseases with stem cells in bone marrow, allow scientists to remove pertinent cells from the body, modify them with CRISPR, and put them back in.[1]
Others, such as eye diseases or cystic fibrosis, have to be altered in the living patient's cells.[1]
Additional factors, like how many cells have to be affected for the symptoms to subside and how easily researchers can access the pertinent cells — such as whether they are in the eye or deep in the bone marrow — can also influence the techniques researchers use to treat disease.[1]
Read more in detail in:
Betting on the first disease to be treated by gene editing
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with CRISPR-edited cells in China
First time
scientists use CRISPR on a human
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Could Soon Be A Workplace Requirement
If Passed By GOP
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the Boss Get in Your Genes Act
that Would Undermine
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LINKS:
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to have access to genetic testing
HUGlican Bill Allows
Employers To Demand Employees To Get Genetic Testing
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Employers to Demand Genetic Information From Workers
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spark discrimination fears
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- conference keynote speaker
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WILL INCLUDE MANDATORY GENETIC TESTING
Your Genetic Information
Might be at Risk Soon with H.R. 1313?
the Genetic Information
NonDiscrimination Act?
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