CRISPR is Making News Again

Below is an excerpt of a good article on the National Public Radio website. Clink on the title below to read the entire article.

First U.S. Patients Treated With CRISPR As Human Gene-Editing Trials Get Underway

"...scientists have long hoped CRISPR — a technology that allows scientists to make very precise modifications to DNA — could eventually help cure many diseases. And now scientists are taking tangible first steps to make that dream a reality.

For example, NPR has learned that a U.S. CRISPR study that had been approved for cancer at the University of Pennsylvania in Philadelphia has finally started. A university spokesman on Monday confirmed for the first time that two patients had been treated using CRISPR.
The revelation comes as several other human trials of CRISPR are starting or are set to start in the U.S., Canada and Europe to test CRISPR's efficacy in treating various diseases.

"2019 is the year when the training wheels come off and the world gets to see what CRISPR can really do for the world in the most positive sense," says Fyodor Urnov, a gene-editing scientist at the Altius Institute for Biomedical Sciences in Seattle and the University of California, Berkeley.

Here are highlights of the year ahead in CRISPR research, and answers to common questions about the technology..."

So what's happening now with new or planned trials?

"We've finally reached the moment when CRISPR is moving out of the lab and into the clinic around the world..."

"...And yet another study, sponsored by Editas Medicine of Cambridge, Mass., will try to treat an inherited form of blindness known as Leber congenital amaurosis.

That study is noteworthy because it would be the first time scientists try using CRISPR to edit genes while they are inside the human body. The other studies involve removing cells from patients, editing the DNA in those cells in the lab and then infusing the modified cells back into patients' bodies.

Finally, several more U.S. cancer studies may also start this year in Texas, New York and elsewhere to try to treat tumors by genetically modifying immune system cells..."

When might we know whether any of these experimental CRISPR treatments are working?

"...All of these studies are very preliminary and are primarily aimed at first testing whether this is safe. That said, they are also looking for clues to whether they might be helping patients. So there could be at least a hint about that later this year. But it will be many years before any CRISPR treatment could become widely available..."

New Research Could Fine-Tune the Gene Scissors CRISPR

If you are a follower of this blog, you know I am following CRISPR research closely in regards to a potential cure for Kennedy's Disease (SBMA). If this process could reduce the number of CAG Repeats, we might have the cure. One issue that researchers are looking at is how to identify and minimize the potential side effects. Below is a link to the latest article on the fine tuning of the process.

New Research Could Fine-Tune the Gene Scissors CRISPR

GENETIC RESEARCH

When researchers and doctors use the tool CRISPR to correct genetic errors, it may have side effects on the human genome. Now, researchers from the University of Copenhagen have learned how the molecular machinery behind CRISPR works and thus expect to be able to fine-tune CRISPR and remove the undesired effects.

The introduction of the tool for gene editing, the so-called gene scissors CRISPR, in 2007 was a revolution within medical science and cell biology. But even though the perspectives are great, the launch of CRISPR has been followed by debate, especially focussing on ethical issues and the technology’s degree of accuracy and side effects.

However, in a new study published in the scientific journal Cell researchers from the Novo Nordisk Foundation Center for Protein Research have described how one of the CRISPR technologies, the so-called Cas12a, works – all the way down to the molecular level. This makes it possible to fine-tune the gene-editing process to only achieve the desired effects.

‘If we compare CRISPR to a car engine, what we have done is make a complete 3D map of the engine and thus gained an understanding of how it works. This knowledge will enable us to fine-tune the CRISPR engine and make it work in various ways – as a Formula 1 racer as well as an off-road truck’, says Professor Guillermo Montoya from the Novo Nordisk Foundation Center for Protein Research....

Molecular Film 

The researchers have used a so-called cryo-electron microscope to map the technology. The recently inaugurated cryoEM facility at the University of Copenhagen has established the state-of-the-art technology enabling the researchers to take photographs of the different shapes of the molecule when CRISPR-Cas12a cuts up the DNA strand.

They combined it with a fluorescent microscopy technique called ‘single molecule FRET’ that directly observes the motions of the molecules and the sequence of events for each individual protein.

Among other things, this sequence of events revealed to the researchers that three “pieces” of the CRISPR tools must change form for the DNA to be cut properly.

‘Our new study shows the precise series of events in the genome leading to gene editing. These three “pieces” that change, work like airport security checks. You have to complete all checks and in the right order to proceed’, says Associate Professor Nikos Hatzakis from the Department of Chemistry and the Nano-Science center.

[Click on the link above to read the entire article]

Gene Editing Fixes Muscular Dystrophy in Dogs

As my readers know, I have been following the development of CRISPR for years. There is a short article written by Alice Park in TIME concerning CRISPR and Duchenne muscular dystrophy. There is also a short video on the page explaining hos CRISPR works. Click on the title below to go to the article.

 What bothers me is that I love beagles.

CRISPR Gene Editing Fixes Muscular Dystrophy in Dogs. Are Humans Next?

The powerful gene editing technology CRISPR is one small step closer to treating a human disease.

In a new paper published in Science, researchers led by Eric Olson, professor and chair of molecular biology at UT Southwestern Medical Center, reported that he and his team successfully used CRISPR to correct the genetic defect responsible for Duchenne muscular dystrophy in four beagles bred with the disease-causing gene. It’s the first use of CRISPR to treat muscular dystrophy in a large animal. (Previous studies had tested the technology on rodents.) In varying degrees, the genetic therapy halted the muscle degradation associated with the disease.

Duchenne is caused by mutations in the dystrophin gene, which codes for a protein essential for normal muscle function. People born with the disease are often eventually confined to wheelchairs as their muscles continue to weaken, and in the later stages, many rely on ventilators to breathe as their diaphragm muscles stop working. Eventually, they develop heart and respiratory failure.

A Crispr Conundrum

I am trying to keep up on the evolution of CRISPR and its possible performance in curing or treating Kennedy's Disease and other progressive neuromuscular disorders. Here is the latest from The New York Times. To read the entire article, click on the link above or the heading below.

A Crispr Conundrum: 

How Cells Fend Off Gene Editing

Human cells resist gene editing by turning on defenses against cancer, ceasing reproduction and sometimes dying, two teams of scientists have found.

The findings, reported in the journal Nature Medicine, at first appeared to cast doubt on the viability of the most widely used form of gene editing, known as Crispr-Cas9 or simply Crispr, sending the stocks of some biotech companies into decline on Monday.

Crispr Therapeutics fell by 13 percent shortly after the scientists’ announcement. Intellia Therapeutics dipped, too, as did Editas Medicine. All three are developing medical treatments based on Crispr.

But the scientists who published the research say that Crispr remains a promising technology, if a bit more difficult than had been known.

“The reactions have been exaggerated,” said Jussi Taipale, a biochemist at the University of Cambridge and an author of one of two papers published Monday. The findings underscore the need for more research into the safety of Crispr, he said, but they don’t spell its doom.

“This is not something that should stop research on Crispr therapies,” he said. “I think it’s almost the other way — we should put more effort into such things.” ...

... A team of scientists at the Novartis Institutes for Biomedical Research in Cambridge, Mass., got similar results with a different kind of cells, detailed in a paper also published Monday.

They set out to develop new versions of Crispr to edit the DNA in stem cells. They planned to turn the stem cells into neurons, enabling them to study brain diseases in Petri dishes.

Someday, they hope, it may become possible to use Crispr to create cell lines that can be implanted in the body to treat diseases.

When the Novartis team turned Crispr on stem cells, however, most of them died. The scientists found signs that Crispr had caused p53 to switch on, so they shut down the p53 gene in the stem cells. ...

...“We should definitely be cautious,” said George Church, a geneticist at Harvard and a founding scientific adviser at Editas.

He suspected that p53’s behavior would not translate into any real risk of cancer, but “it’s a valid concern.”

And those concerns may be moot in a few years. The problem with Crispr is that it breaks DNA strands. But Dr. Church and other researchers are now investigating ways of editing DNA without breaking it.

“We’re going to have a whole new generation of molecules that have nothing to do with Crispr,” he said. ...

Using CRISPR to Edit Genes

I have written about CRISPR gene editing several times over the years. I can see  it someday removing the defective CAG repeats in Kennedy's Disease.

Last night, there was a CBS program ’60 Minutes’ featuring CRISPR. The segment was well done. It broke down the actual process to understandable bites of information.

If you missed the program, you can read about it and watch most of it on 60 Minutes Overtime.

I was impressed to learn 2,300 labs across the globe are currently experimenting with CRISPR. And, they have used it to correct a genetic disorder that causes blindness (mouse models only). There is the first U.S. clinical trial using CRISPR for patients with a certain cancer. I also found it interesting that scientists have to consider what could be any unexpected results of correcting the defective DNA.

Below are excerpts of the CBS article:

“Scientists are excited about using CRISPR to treat genetic disease, but the devil's in the details.

Take, for example, treating cystic fibrosis. Scientists know that the hereditary disease is caused by the misspelling of a particular gene. Genes are made up of DNA, which is composed of chemical bases abbreviated by the letters A, T, G, and C. A genetic mutation that causes a disease like cystic fibrosis reads like a typo in the genetic instructions. If scientists can identify the typo, they can program CRISPR to try to correct it with the right sequence.

This week on 60 Minutes, correspondent Bill Whitaker reports on the gene-editing tool. Feng Zhang, a molecular biologist at the Broad Institute of MIT and Harvard, who has been working on CRISPR for seven years, explains how it works.” ...

"You can understand why I want to say, 'It's amazing,' and you also have to be careful and every single cure is going to take a lot of people working together to deliver it," Lander tells Whitaker in the video above. "But you couldn't have imagined such a cure. It wasn't even on your to-do list before CRISPR." ...

"Preventing disease, I think, is a little more complicated because it depends on how we're trying to prevent the disease. Some of them may have undesirable side effects that we don't fully understand," he explains in the clip above.”

US scientists try 1st gene editing in the body

In recent years I have reported several times about CRISPR and gene editing. I am not a scientist, but, to a layman, this sounds like the best possibility for curing Kennedy's Disease. In the article referenced below, it discusses the potential benefits and possible dangers of gene editing.

There is much more research that needs to be done, but the possibility of a cure is now closer than ever before.

US scientists try 1st gene editing in the body

"...This time, the gene tinkering is happening in a precise way inside the body. It’s like sending a mini surgeon along to place the new gene in exactly the right location.

“We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending,” said Dr. Sandy Macrae, president of Sangamo Therapeutics, the California company testing this for two metabolic diseases and hemophilia. “It becomes part of your DNA and is there for the rest of your life.”

That also means there’s no going back, no way to erase any mistakes the editing might cause.

“You’re really toying with Mother Nature” and the risks can’t be fully known, but the studies should move forward because these are incurable diseases, said one independent expert, Dr. Eric Topol of the Scripps Translational Science Institute in San Diego.

Protections are in place to help ensure safety, and animal tests were very encouraging, said Dr. Howard Kaufman, a Boston scientist on the National Institutes of Health panel that approved the studies.

He said gene editing’s promise is too great to ignore. “So far there’s been no evidence that this is going to be dangerous,” he said. “Now is not the time to get scared.”..."

A short APNews animation video does a good job of explaining the process. 

(AP Animation/Marshall Ritzel)

This is another article snipped from the NIH.gov blog. Since Huntington’s and Kennedy’s Disease  both have extra repetitions of the CAG DNA, this is another advancement in treating and possibly curing these type diseases. As I mentioned in my previous post, the process is still experimental, but it is moving in the right direction. If you haven't read the previous update on gene editing, you can find it here.

To read the entire article, click on the title below.

Huntington’sDisease: Gene Editing Shows Promise in Mouse Studies

Posted on June 27, 2017 by Dr. Francis Collins

“…But years of basic science advances, combined with the promise of innovative gene editing systems such as CRISPR/Cas9, are providing renewed hope that we will someday be able to treat or even cure Huntington’s disease, along with many other inherited disorders.

My own lab was part of a collaboration of seven groups that identified the Huntington’s disease gene back in 1993. Huntington’s disease occurs when a person inherits from one parent a mutant copy of the huntingtin (HTT) gene that contains extra repetitions, or a “stutter,” of three letters (CAG) in DNA’s four-letter code. This stutter leads to production of a misfolded protein that is toxic to the brain’s neurons, triggering a degenerative process that, over time, leads to mood swings, slurred speech, uncontrolled movements, and, eventually, death. In a new study involving a mouse model of Huntington’s disease, researchers were able to stop the production of the abnormal protein by using CRISPR tools to cut the stutter out of the mutant gene.

The progress, reported in the Journal of Clinical Investigation [1], comes from the NIH-supported team of Su Yang, Renbao Chang, Xiao-Jiang Li, and colleagues at Emory University School of Medicine, Atlanta. The group’s previous work showed that halting the production of mutated (or even healthy!) HTT protein in mature neurons doesn’t hurt the cells or cause obvious neurological problems in mice [2]. So, the researchers now wanted to see if halting HTT production in millions of neurons in the striatum, which is a part of the inner brain that controls motor skills, could reverse early signs of disease that typically appear in affected mice before the age of 9 months.

To get their answers, the researchers injected millions of inactivated viral particles directly into the striatum of a few 9-month-old mice, engineered to produce the mutant form of HTT protein. Each particle, like a Trojan horse, delivered to the neurons one of the two pieces of the CRISPR/Cas9 editing system: either a short guide RNA sequence to mark for removal the HTT gene’s CAG repeats or a scissor-like Cas9 enzyme to snip out the repeats. In this strategy, both the health and abnormal copies of the HTT gene were “knocked out,” resulting in the production of no HTT protein.

Remarkably, three weeks later, the researchers found that the CRISPR/Cas9 gene editing had reversed the disease process in their mouse model. Neurons in the striatum had stopped making the HTT protein. What’s more, the toxic, abnormal HTT protein that had already clumped together in and around the neurons—and which likely would have would have killed them—had begun to clear to varying degrees in the mice. The same went for other protein abnormalities associated with the progression of Huntington’s disease.

There was even better news to come. The Emory team repeated the CRISPR/Cas9 injections into the striatum of a dozen 9-month-old mice and got a similar protein-clearing outcome. Then, over the next three months, the researchers found that the animals’ balance, muscle coordination, and mobility had improved compared to mice given sham shots of CRISPR/Cas9. Interestingly, the degree of improvement in their motor skills corresponded with the amount of toxic protein that had been cleared from the striatum…”

“…This utilization of CRISPR/Cas9 to pursue a cure for Huntington’s disease is one more example of how this powerful new technology might be applied to the thousands of diseases due to a specific mutation in DNA; efforts are already underway for other conditions like sickle cell disease and muscular dystrophy. Given the promise, the NIH Common Fund is actively exploring ways in which this approach could be accelerated.”

The following article was snipped from NIH.gov’s blog. It describes another improvement in the gene editing research going on around the world. The process is still experimental, but it is moving in the right direction. Read my next article on Huntington's Desease. Kennedy's Disease could be right around the corner. 

To read the entire article, click on the title below.

GeneEditing: Gold Nanoparticle Delivery Shows Promise

Posted on October 10, 2017 by Dr. Francis Collins

…”NIH-funded researchers have developed a highly versatile approach to CRISPR/Cas9-based therapies. Instead of relying on viruses to carry the gene-editing system into cells, the new approach uses tiny particles of gold as the delivery system!

In order to fix a disease-causing mutation like the expanded DNA repeat that causes FA (Friedreich’s ataxia), researchers must create a CRISPR/Cas9 system that contains a scissor-like Cas9 enzyme and a synthetic guide RNA, which steers Cas9 to the specific part of the genome that needs to be snipped out. If a very precise correction is to be made, a repair template that contains the desired DNA code must also be included.

The challenge is delivering all these components into the appropriate tissues in a safe and efficient manner. Currently, most researchers use inactivated, non-disease-causing viruses to ferry various parts of the CRISPR/Cas9 system into cells. However, because of size constraints, it’s not possible to fit all three components into a single virus. Also, because of the large number of viral particles needed to carry CRISPR/Cas9 components in separately, there are concerns that viral delivery systems could trigger immune responses in people. Not only could such immune responses pose a safety hazard to patients, they could also reduce the effectiveness of the viral delivery system.

Because of these challenges, there’s been great interest in developing better ways to deliver CRISPR/Cas9 therapeutics. In the new study recently reported in Nature Biomedical Engineering, Irina Conboy and Niren Murthy at the University of California, Berkeley, decided to try a delivery vehicle they call CRISPR-Gold [1].

Gold might seem like an odd choice, but gold nanoparticles possess a special ability to penetrate cell membranes and have been considered for use in delivering therapies for cancer, rheumatoid arthritis, and many other conditions. In addition, gold is generally well tolerated by the human body and has the advantage of linking easily to DNA.

The CRISPR-Gold system—which consists of a DNA-linked gold nanoparticle containing Cas9, guide RNA, and a DNA repair template—is designed to enter cells through endocytosis, a process in which the cell engulfs outside molecules. A special polymer that encases the CRISPR-Gold system helps to ensure the gene-editing tools reach the cell’s genome in an active state.

In a series of tests, the researchers showed that CRISPR-Gold could enter a variety of cell types in laboratory culture, including immune cells, muscle cell progenitors, human induced pluripotent stem cells, and human embryonic stem cells. Once inside the cells, CRISPR-Gold could successfully find and edit a target gene in a non-toxic manner. Similar success occurred when CRISPR-Gold was injected into the muscles of living lab mice.

The next big challenge was to test CRISPR-Gold’s potential in a model of human disease. So, researchers turned to a mouse model of Duchenne muscular dystrophy (DMD), a fatal disorder characterized by progressive muscle weakening and caused by a mutation in the gene that codes for the protein dystrophin. They injected CRISPR-Gold containing a template for a healthy dystrophin gene into the leg muscles of young DMD mice. At the same time, they injected a toxin intended to encourage muscle cells to multiply because, for CRISPR editing to work optimally, cells must be actively dividing.

The outcome was encouraging. After one injection of CRISPR-Gold, about 5 percent of the dystrophin genes in the muscle tissue of the DMD mice had been corrected. What’s more, the animal’s muscles produced functional dystrophin protein, and they performed better on tests of muscle strength.

There was also good news on the safety front. The DMD mice didn’t appear to have a strong immune reaction to the treatment. The researchers also didn’t find evidence that CRISPR-Gold caused much, if any, unintended “off target” damage to the animals’ DNA.

Taken together, the findings suggest that, pending further replication, optimization, and careful testing, CRISPR-Gold might have promise for treating humans with DMD.

What makes this approach especially exciting is that it also holds potential for treating or even curing many other genetic diseases …”

CRISPR - Gene Editing Explained

A man living with Kennedy's Disease posted this on Facebook. The short YouTube video (link below) does a good job of explaining what CRISPR is, how it works, and its potential. Those of us living with KD are very interested in the potential tools like CRISPR offer for future generations.

Biologist Explains One Concept in 5 Levels of Difficulty - CRISPR

Video published May 24, 2017

CRISPR is a new area of biomedical science that enables gene editing and could be the key to eventually curing diseases like autism or cancer. WIRED has challenged biologist Neville Sanjana to explain this concept to 5 different people; a 7 year-old, a 14 year-old, a college student, a grad student and a CRISPR expert.

Gene Editing Followup

A followup article in the New York Times by Pam Belluck dismisses some of the fears people have regarding Gene Editing. Since Huntington’s Disease is mentioned, that is a good sign for those of us with families living with Kennedy’s Disease. To read the entire article, follow the title link.

Gene Editing for ‘Designer Babies’? Highly Unlikely, Scientists Say

 “Now that science is a big step closer to being able to fiddle with the genes of a human embryo, is it time to panic? Could embryo editing spiral out of control, allowing parents to custom-order a baby with Lin-Manuel Miranda’s imagination or Usain Bolt’s speed?

News that an international team of scientists in Oregon had successfully modified the DNA of human embryos has renewed apprehensions that babies will one day be “designed.” But there are good reasons to think that these fears are closer to science fiction than they are to science.

Here is what the researchers did: repair a single gene mutation on a single gene, a defect known to cause — by its lonesome — a serious, sometimes fatal, heart disease. …”

“ … So are most physical diseases and psychiatric disorders. The genetic message is not carried in a 140-character tweet — it resembles a shelf full of books with chapters, subsections and footnotes.

So embryonic editing is unlikely to prevent most medical problems.

But about 10,000 medical conditions are linked to specific mutations, including Huntington’s disease, cancers caused by BRCA genes, Tay-Sachs disease, cystic fibrosis, sickle cell anemia, and some cases of early-onset Alzheimer’s. Repairing the responsible mutations in theory could eradicate these diseases from the so-called germline, the genetic material passed from one generation to the next. No future family members would inherit them.

But testing editing approaches on each mutation will require scientists to find the right genetic signpost, often an RNA molecule, to guide the gene-snipping tool.

In the study reported this week, it took 10 tries to find the right RNA, said Juan Carlos Izpisua Belmonte, a co-author and geneticist at the Salk Institute.

Dr. Greely noted that while scientists work to get human embryonic editing ready for clinical trials (currently illegal in the United States and many countries), alternate medical treatments for these diseases might be developed. They may be simpler and cheaper. …”

Photo:  https://rhapsodyinwords.com/2017/01/

The Last - I can only hope

A picture above my desk called, “The Last,” has a dual meaning for me. First off, I love the lithograph because it is so well done. To me it reflects the dying off of the American Indian culture. The second meaning is much more personal. I hope and pray I am the last male in my mother’s family with Kennedy’s Disease.

A NewYork Times article written by Pam Belluck reports on a recent study and potential milestone in genetic engineering. Nature,the International Weekly Journal of Science published the study this week. A portion of the article is shown below. Follow the links above to read the entire article and the study.

In Breakthrough, Scientists Edit a Dangerous Mutation From Genes in Human Embryos

“”Scientists for the first time have successfully edited genes in human embryos to repair a common and serious disease-causing mutation, producing apparently healthy embryos, according to a study published on Wednesday.

The research marks a major milestone and, while a long way from clinical use, it raises the prospect that gene editing may one day protect babies from a variety of hereditary conditions.

But the achievement is also an example of human genetic engineering, once feared and unthinkable, and is sure to renew ethical concerns that some might try to design babies with certain traits, like greater intelligence or athleticism.

Scientists have long feared the unforeseen medical consequences of making inherited changes to human DNA. The cultural implications may be just as disturbing: Some experts have warned that unregulated genetic engineering may lead to a new form of eugenics, in which people with means pay to have children with enhanced traits even as those with disabilities are devalued. …”

“… Scientists at Oregon Health and Science University, with colleagues in California, China and South Korea, reported that they repaired dozens of embryos, fixing a mutation that causes a common heart condition that can lead to sudden death later in life.

If embryos with the repaired mutation were allowed to develop into babies, they would not only be disease-free but also would not transmit the disease to descendants.

The researchers averted two important safety problems: They produced embryos in which all cells — not just some — were mutation-free, and they avoided creating unwanted extra mutations.

“It feels a bit like a ‘one small step for (hu)mans, one giant leap for (hu)mankind’ moment,” Jennifer Doudna, a biochemist who helped discover the gene-editing method used, called CRISPR-Cas9, said in an email. …”

"Desistance" is my current story I am in the final stages of editing. It is a Sci-Fi that takes place sixty years in the future. One of the storylines is the development of almost super-humans whose DNA has been edited to remove most of the known diseases. Perhaps it won’t be Sci-Fi after all.

Human Gene Editing

I have written posts a few times in the past about CRISPR, a technology that allows the editing of human genes. In my current book I'm writing, the idea of gene editing is an important aspect of creating super-healthy children.

The New York Times published the following article today. As with anything revolutionary in nature, their is controversy surrounding it.

Human Gene Editing Receives Science Panel’s Support

"An influential science advisory group formed by the National Academy of Sciences and the National
Academy of Medicine on Tuesday lent its support to a once-unthinkable proposition: the modification of human embryos to create genetic traits that can be passed down to future generations.

This type of human gene editing has long been seen as an ethical minefield. Researchers fear that the
techniques used to prevent genetic diseases might also be used to enhance intelligence, for example, or to create people physically suited to particular tasks, like serving as soldiers.

The advisory group endorsed only alterations designed to prevent babies from acquiring genes known to cause “serious diseases and disability,” and only when there is no “reasonable alternative.” The report provides an explicit rationale for genetic research that the federal government has avoided supporting until now, although the work is being pursued in countries like Sweden and China.

So-called germ line engineering might allow people to have biological children without fear that they have passed on the genes for diseases like Huntington’s, Tay-Sachs and beta thalassemia, and without discarding embryos carrying the disease-causing mutations, as is often done now. Though such cases are likely to be rare, the report says they should be taken seriously. ..."

Follow the link above to read the rest of the article.

More on CRISPR

A friend sent me this seven minute CBS video that does a good job of reporting what CRISPR is, and what are its potential benefits and concerns on its use. Genes and Dreams

CRISPR, gene editing technology, is light years ahead of other technology in this one area. Its potential uses are almost unbelievable. Yet, it is still in its infancy, and therefore needs strong parenting and further guidance to insure the technology is not abused.

Yet, for me, the hope grows stronger that a treatment, and potentially a cure, for Kennedy's Disease, SBMA, will become available in the not to distant future. It might not be available for my generation, but it sure appears it will be there for my children, and their children.

Photo: MIT News

CRISPR is in the news again

Last year I posted four articles on CRISPR. The technology is new and fascinating. There appears to be endless possibilities as to what it might be able to do someday. For those of us living with a neuromuscular condition (including Kennedy’s Disease), it offers hope. 

In this latest article written by Kate Lunau for McLean’s Magazine, researchers have taken another step forward in this quest for a treatment or cure.

Gene editing spurs hope for muscular dystrophy cure

For the first time, a breakthrough technique called CRISPR has been used to treat a genetic disease inside a living mammal

Kate Lunau  January 4, 2016 

“Of the many diseases that afflict us, those that strike young children seem especially cruel. One is Duchenne muscular dystrophy, which typically appears before a child’s sixth birthday. Sufferers (almost always boys) struggle with muscle weakness, and have trouble walking. By 12, most will be in a wheelchair; few live beyond their thirties. There’s no cure, and it affects about one in 3,500 males born worldwide. Suddenly, with the arrival of a powerful gene editing technique, new treatments seem to be on the horizon. In three independent papers, published in the journal Science, U.S. scientists snipped out defective DNA in mice with the disease—the first time this tool, called CRISPR, has been used to treat any genetic disease inside a living mammal—and restored some of their muscle function. ...”

Clickhere to read the entire article