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Jennifer Doudna: The Promise and Peril of Gene Editing
Geneticist Jennifer Doudna on the controversy about Crispr-Cas9, a technique that could potentially cure genetic diseases

Jennifer Doudna, a professor of chemistry and molecular biology at the University of California, Berkeley. Photo: Drew Kelly for The Wall Street Journal; Hair & makeup by Veronica Sjoen

Alexandra Wolfe
March 11, 2016 2:33 p.m. ET

Geneticist Jennifer Doudna, 52, is waiting for the day when she reads about the first baby whose genes have been altered in a lab. “It’s only a matter of time,” says the professor of chemistry and molecular biology at the University of California, Berkeley—within the next 10 years, she thinks, or even sooner. The idea excites and worries her because she has been so deeply involved in the technology that would make it possible.

Called Crispr-Cas9, it is a way to edit DNA and potentially remove and replace genes for certain diseases, characteristics and capabilities. As scientists have become more comfortable with the technology, its reach has grown—as have ethical concerns about how it may be used.

In 2014, Chinese scientists successfully produced monkeys that had been genetically modified at the embryonic stage using Crispr-Cas9. And last year, another team of researchers in China published a paper about their experiments editing genes in human embryos. (The researchers tried but failed to repair a gene that causes a hereditary blood disease.) Last month, a U.K. regulatory group approved research using the technology on viable human embryos. Some scientists, including Dr. Doudna, have called for a moratorium on using Crispr and other gene-editing techniques to bring about any heritable genetic changes in humans.

UC Berkeley is in the midst of a patent battle over the technology’s invention, with billions of dollars at stake. In March 2013, Dr. Doudna and six other scientists filed a patent application with 155 claims related to Crispr-Cas9. In October of that year, scientist Feng Zhang of the Broad Institute and the Massachusetts Institute of Technology filed his own claims related to Crispr; his application was approved first. UC Berkeley contested his claims, and proceedings began last week at the federal Patent Trial and Appeal Board to determine who invented the technique first. The side that prevails could get all the rights to the technology.

“The Broad Institute looks forward to participating in this process as it provides an opportunity to bring clarity to the field,” said a spokesman.

Photo: Drew Kelly for The Wall Street Journal; Hair & makeup by Veronica Sjoen

Crispr stands for “clustered regularly interspaced short palindromic repeats”; Cas9 is the protein that makes the gene editing work. Basically, Cas9 helps to snip out a piece of DNA at any point in an organism—including, for instance, viral or faulty DNA—and then enables the cell to stitch the ends back together.

Dr. Doudna collaborated on her research with Emmanuelle Charpentier, a researcher now at the Max Planck Institute for Infection Biology in Berlin. Early on, she was excited by how efficient the technique was. One night, she went home to make dinner for her 13-year-old son and started laughing. “My son said, ‘What’s so funny?’ ” she remembers. “We have this crazy protein that they program to find and destroy viruses!” she told him. “It just struck me as so amazing that biology had come up with this.”

She and her colleagues published a paper about the technique in 2012 and then another in 2013, three weeks after Dr. Zhang published his own research. ( George Church, a geneticist at Harvard University, also published Crispr-Cas9 research at the same time.)

Related Video

Scientists have high hopes for a new gene-editing technology that could provide them with unprecedented power to rewrite the code of life. WSJ’s Monika Auger reports.

Growing up in Hilo, Hawaii, the daughter of two university professors, Dr. Doudna was always fascinated by the natural environment. She recalls wondering as a child, for instance, how science could explain the sky’s being blue. She went on to Pomona College in California, where she studied biochemistry. She is married to Jamie Cate, a biochemistry professor who is also at UC Berkeley.

For now, she thinks that scientists should hold off on editing genes in human embryos. Too much is still unknown about the long-term effects and its consequences, she says. “It should not proceed until we have a chance to understand better how the technology operates in those kinds of cells, as well as to provide time for societal consideration,” she says.

The ethical issues mainly center on the idea that people could edit genes in embryos to select for various heritable traits, from eye color to intelligence—uses that Dr. Doudna opposes.

She does support using the technology to cure genetic diseases such as cystic fibrosis and muscular dystrophy in adults and children. Less contentious are the technique’s agricultural applications, such as genetic editing to protect plants from fungal infections or to create drought and disease-resistant crops such as wheat or sorghum.

She hopes that Crispr—once it is fully tested—will eventually be as accepted as in vitro fertilization, which was also controversial when it was first introduced. “I wonder if over time people will get comfortable with the idea of human genome editing in embryos, at least if it were going to correct a mutation that would otherwise give rise to a debilitating lifetime disorder and maybe to even remove some kinds of debilitating bad diseases from the human genome completely,” she says.

As Dr. Doudna notes, the DNA modifications made to embryos could be transmitted to future generations. “It’s kind of a profound thing because if you really think about it,” she says, “it really means altering human evolution on some level.”
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