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CRISPR-Cas9: Developments in Genomic Editing

Started by Recusant, November 28, 2015, 05:00:48 AM

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Recusant

I heard a story on the radio about this--it's a technique that allows scientists to edit genes in a precise way. Though it's fairly early days yet, this discovery is very likely to have a huge impact in the future. The video below gives a pretty clear explanation of the technique.



For more detail, a story about the background of CRISPR and how it was developed: "The Genesis Engine" | Wired

Some amazing things could be created with this technique, but as with any powerful tool, extreme care must be exercised. It's simple enough to do that control over what is done with it may not be easy.
"Religion is fundamentally opposed to everything I hold in veneration — courage, clear thinking, honesty, fairness, and above all, love of the truth."
— H. L. Mencken


Icarus

A recent article described some cutting edge experiments with mosquito DNA. Mosquitos transmit malaria as is well known. Replacing a certain DNA element with a different one eliminates the ability to carry the malaria element. The modified mosquito is now unable to transmit the carrier trait to its eggs. That means that new hatches will not be malaria carriers. Malaria is one of the top causes of  human deaths.

There are both humanitarian and sociological implications with this. Millions of lives and miseries will be saved, but annual human population will be substantially expanded. 



 

Recusant

"Religion is fundamentally opposed to everything I hold in veneration — courage, clear thinking, honesty, fairness, and above all, love of the truth."
— H. L. Mencken


Recusant

Here we go.

"Human gene editing gets green light" | Science News

QuoteHuman gene-editing research, even on embryos, is needed and should go ahead, with one major caveat: No pregnancies can result, leaders of an international summit on the topic said December 3.

In recent years, scientists have devised increasingly precise molecular scissors for cutting and pasting DNA. These tools, especially the guided scissors known as CRISPR/Cas9, have become so cheap and easy to use that it may be possible to use them to correct genetic diseases.

Many see the technology as a medical boon; others, though, say that the prospect of designer babies and tinkering with the DNA of future generations should be out of bounds (SN: 5/30/15, p. 16). The U.S. National Academies of Sciences and Medicine, the Chinese Academy of Sciences and the United Kingdom's Royal Society convened the summit to discuss the state of the science as well as ethical, legal and regulatory considerations surrounding gene-editing technology.

Gene editing of human body, or somatic, cells, which do not pass genetic information to future generations, is already in clinical trials. Most of those studies have involved older technologies and cells that were edited outside the body and then given to a patient later, such as a baby with leukemia treated with edited immune cells (SN: 12/12/15, p. 7).

[Continues . . .]
"Religion is fundamentally opposed to everything I hold in veneration — courage, clear thinking, honesty, fairness, and above all, love of the truth."
— H. L. Mencken


Crow

Sweet! This could be interesting in a few decades.
Retired member.

Recusant

Quote from: Crow on December 06, 2015, 09:47:56 PM
Sweet! This could be interesting in a few decades.

It may be sooner than that.

"CRISPR Successfully Treats Genetic Disorder in Adult Mammal" | Science World Report

QuoteCRISPR, the gene editing tool, has long been touted as something that could change the way we treat genetic disorders. Now, scientists have used CRISPR to treat an adult mouse model of Duchenne muscular dystrophy.

In this latest study, the researchers used CRISPR to correct genetic mutations in cultured cells from Duchenne patients, and other labs had corrected genes in single-cell embryos in a laboratory environment. But the latter approach is currently unethical to attempt in humans, and the former faces many obstacles in delivering treated cells back to muscle tissues.

Another approach, which involves taking CRISPR directly to the affected tissues through gene therapy techniques, also faces challenges, particularly with delivery.

In this latest study, the researchers overcame several of these obstacles by using a non-pathogenic carrier called adeno-associated virus, or AAV, to deliver the gene-editing system.

[Continues . . .]
"Religion is fundamentally opposed to everything I hold in veneration — courage, clear thinking, honesty, fairness, and above all, love of the truth."
— H. L. Mencken


Icarus

A recent NPR program with a guest who is a CRISPR authority said that bacteria, within humans, often have smaller bacteria or perhaps viruses that attack them. That is reminiscent of the mathematical infinite regression concept....or the cute little poem about the little demons that bitem' and to ad finitem'........

Recusant

The following isn't really a science story--it's almost entirely about the intersection of science and money. Anyway, it gives some indication of how important and valuable investors and biotech companies believe the CRISPR technique will be. "The Battle Over CRISPR Could Make Or Break Some Biotech Companies" | FiveThirtyEight
"Religion is fundamentally opposed to everything I hold in veneration — courage, clear thinking, honesty, fairness, and above all, love of the truth."
— H. L. Mencken


Recusant

It's looking like the first safety trial of the CRISPR technique on humans will start soon. Amazing.

"First human CRISPR trial given go-ahead: your questions answered" | New Scientist

QuoteThe CRISPR gene editing revolution is happening even faster than we expected. Many thought human trials of therapies using the technique were still years away. But yesterday, a US federal committee gave its nod of approval – meaning the first trial could start later this year. The therapy is designed to treat cancer but the main purpose of this first trial is safety. If it succeeds, it will encourage many other groups to start testing treatments that involve CRISPR.

[Continues . . .]
"Religion is fundamentally opposed to everything I hold in veneration — courage, clear thinking, honesty, fairness, and above all, love of the truth."
— H. L. Mencken


Asmodean

Somehow, I thought this would be the domain of nanorobots. Perhaps it will though, no? After all, in theory, those could be a formidable vessel for delivering treatment where it's required.

For the aforementioned reason of unsustainable population growth, I do hope any successful treatment involving gene therapy remains hugely expensive until the less civilized societioes learn to control their birth rates. Still, color me fascinated.
Quote from: Ecurb Noselrub on July 25, 2013, 08:18:52 PM
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Recusant

The CRISPR-Cas9 gene manipulation technique apparently isn't as precise as it might be.

"CRISPR gene editing can cause hundreds of unintended mutations" | PhysOrg

QuoteAs CRISPR-Cas9 starts to move into clinical trials, a new study published in Nature Methods has found that the gene-editing technology can introduce hundreds of unintended mutations into the genome.

"We feel it's critical that the scientific community consider the potential hazards of all off-target mutations caused by CRISPR, including single nucleotide mutations and mutations in non-coding regions of the genome," says co-author Stephen Tsang, MD, PhD, the Laszlo T. Bito Associate Professor of Ophthalmology and associate professor of pathology and cell biology at Columbia University Medical Center and in Columbia's Institute of Genomic Medicine and the Institute of Human Nutrition.

CRISPR-Cas9 editing technology—by virtue of its speed and unprecedented precision—has been a boon for scientists trying to understand the role of genes in disease. The technique has also raised hope for more powerful gene therapies that can delete or repair flawed genes, not just add new genes.

The first clinical trial to deploy CRISPR is now underway in China, and a U.S. trial is slated to start next year. But even though CRISPR can precisely target specific stretches of DNA, it sometimes hits other parts of the genome. Most studies that search for these off-target mutations use computer algorithms to identify areas most likely to be affected and then examine those areas for deletions and insertions.

"These predictive algorithms seem to do a good job when CRISPR is performed in cells or tissues in a dish, but whole genome sequencing has not been employed to look for all off-target effects in living animals," says co-author Alexander Bassuk, MD, PhD, professor of pediatrics at the University of Iowa.


[Continues . . .]
"Religion is fundamentally opposed to everything I hold in veneration — courage, clear thinking, honesty, fairness, and above all, love of the truth."
— H. L. Mencken


Tank

Better to know now than later. But, bugger it!
If religions were TV channels atheism is turning the TV off.
"Religion is a culture of faith; science is a culture of doubt." ― Richard P. Feynman
'It is said that your life flashes before your eyes just before you die. That is true, it's called Life.' - Terry Pratchett
Remember, your inability to grasp science is not a valid argument against it.

Dave

Yeah, have to wait a bit longer to get to the sci-fi level of human gene sculpting and setting exact specs for babies . . .

But also for all the therapeutic stuff as well.
Tomorrow is precious, don't ruin it by fouling up today.
Passed Monday 10th Dec 2018 age 74

Recusant

The CRISPR technique has been tested for use in eliminating genetic disease in vitro for human embryos. It appears to have worked.

"Human embryos edited to stop disease" | BBC

QuoteScientists have, for the first time, successfully freed embryos of a piece of faulty DNA that causes deadly heart disease to run in families.

It potentially opens the door to preventing 10,000 disorders that are passed down the generations.
The US and South Korean team allowed the embryos to develop for five days before stopping the experiment.

The study hints at the future of medicine, but also provokes deep questions about what is morally right.

Science is going through a golden age in editing DNA thanks to a new technology called Crispr, named breakthrough of the year in just 2015.

Its applications in medicine are vast and include the idea of wiping out genetic faults that cause diseases from cystic fibrosis to breast cancer.

[Continues . . .]

The full paper is available through a link ("described in the journal Nature") in the BBC story.
"Religion is fundamentally opposed to everything I hold in veneration — courage, clear thinking, honesty, fairness, and above all, love of the truth."
— H. L. Mencken


Arturo

https://www.technologyreview.com/s/608350/first-human-embryos-edited-in-us/

Here is a related article. I couldn't read because my phone won't load it but it gets around.
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