A new way to make powerful changes at will to the DNA of humans, other animals and plants, much like how a writer changes words in a story, could usher in a transformation in genetic medicine.
Scientists are not just excited about this recently discovered technique because it can snip and edit DNA with precision. It can also do the job more easily and cheaply than other gene-editing methods, making possible research that has historically been difficult, experts say.
Now some of the biologists who unlocked this tool, derived from the immune system of bacteria, are forming companies around it. Although this molecular system, known as Crispr, is not fully understood, researchers believe it can be harnessed to create therapies for intractable genetic diseases.
“In principle, this is a technology that could enable correction of genetic mutations that would otherwise lead to disease,” said Doudna, a professor of chemistry and biochemistry and molecular biology, in a telephone interview. She was among several experts who spoke at a UC Berkeley conference on the subject last month.
But because the method is in its infancy and has little precedent with the agencies that regulate medicines, it will almost certainly be a long time before a Crispr-based therapy makes it to market.
Its potential risks also concern some bioethicists.
The Latest on: Editing DNA
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The Latest on: Editing DNA
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Scientists have figured out how to modify CRISPR's basic architecture to extend its reach beyond the genome and into what's known as the epigenome -- proteins and small molecules that latch onto DNA ...
- CRISPR base editor treats premature-aging syndromeon April 16, 2021 at 7:10 am
CRISPR-mediated adenine base editor (ABE) to repair mutations of the Hutchinson–Gilford progeria syndrome (HGPS or progeria), attenuate symptoms, and extend lifespan of mice (Fig. 1 ), 1 representing ...
- Behold the infinite possibilities of gene editingon April 16, 2021 at 6:29 am
CRISPR’s ability to alter genome sequences holds immense promise for medicine and other fields.The industrial revolution was all about using atoms for human advancement, and the internet revolution ...
- Structure-guided engineering of adenine base editor with minimized RNA off-targeting activityon April 16, 2021 at 4:35 am
Base editors can induce transcriptome-wide RNA off-target editing independent of gRNA. Here, the authors engineer ABEmax variants with minimized RNA off-target activities.
- A New Clinical Trial Will Explore Gene Editing For Sickle Cell Diseaseon April 15, 2021 at 7:22 pm
Researchers will conduct a first-in-human clinical trial of a CRISPR gene correction therapy on people with severe sickle cell disease.
- An on-off switch for gene editingon April 14, 2021 at 12:50 pm
A new gene editing technology called CRISPRoff allows researchers to control gene expression with high specificity while leaving the sequence of the DNA unchanged.
- New approach to scale up genome editing in tiny wormson April 13, 2021 at 8:59 pm
Understanding the effects of specific mutations in gene regulatory regions - the sections of DNA and RNA that turn genes on and off - is important to unraveling how the genome works, as well as normal ...
- Researchers call for greater awareness of unintended consequences of CRISPR gene editingon April 12, 2021 at 5:10 am
Researchers at the Francis Crick Institute have revealed that CRISPR-Cas9 genome editing can lead to unintended mutations at the targeted section of DNA in early human embryos. The work highlights the ...
- CRISPR-on, CRISPR-off: New game-changing gene editing technique is entirely reversibleon April 10, 2021 at 9:39 am
US researchers have unveiled a significant upgrade to the CRISPR gene editing system, which, unlike its predecessor, can target specific genes with high precision and make completely reversible ...
- Genetic Engineering 2.0: An On-Off Switch for Gene Editingon April 10, 2021 at 8:34 am
New, reversible CRISPR method can control gene expression while leaving underlying DNA sequence unchanged. Over the past decade, the CRISPR-Cas9 gene editing system has revolutionized genetic ...
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