CRISPR Gene Editing Has Repaired a Blood-Borne Disease


IN BRIEF
  • Researchers from Stanford have used the CRISPR-Cas9 gene editing technique to fix the gene defects that cause sickle cell disease.
  • Approximately 70,000 – 100,000 individuals in the United States have sickle cell disease and 3 million have sickle cell trait.

GENE THERAPY

Gene editing remains a widely controversial topic due to the large potential for both benefit and “accidents.” Despite this, scientists are still hard at work developing gene edits that can solve a wide variety of diseases.

A new study may have found the key to solving a painful, and potentially fatal, genetic defect. Researchers from Stanford used the CRISPR-Cas9 gene editing technique to fix the gene defects that cause sickle cell disease.

Sickle cell disease is actually a group of related diseases that cause the formation of hemoglobin S, or sickle-shaped hemoglobin. That shape results in red blood cells becoming tangled in each other. Enough tangling, and blood vessels could be blocked, causing pain and even death.

To correct the issue, one has to repair the genes that code for abnormal hemoglobin. Using CRISPR, the researchers were able to do this. They took hematopoietic stem cells from patients that have the disease and edited them to repair their genome.

After, the repaired stem cells are concentrated and injected to healthy mice. Once there, the stem cells find their way into bone marrow and start producing more of their healthy variants. Sixteen weeks after injection, the researchers found healthy red blood cells thriving in the bone marrow.

ONE OF MANY

This is just one of the increasing number of diseases that have met their match thanks to CRISPR. The gene editing technique is composed of an enzyme that can splice DNA sequences and guide RNA that take them to the specific sequences that need splicing.

“There’s already a lot of active research going on using the CRISPR technology to fix diseases like Duchenne muscular dystrophy or cystic fibrosis or Huntington’s disease,” says Jennifer Doudna, a pioneer of the technology, to CNBC.

But even if ways to cure using CRISPR are abound, researchers are treading lightly on implementation, since editing genes is risky and can have disastrous consequences. Ultimately, we will need to have a serious debate on how to go about the changing the very blueprint of our bodies.

‘Needle risk’ over beauty treatments.


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A health watchdog is concerned that people having beauty treatments like Botox could be at risk of infection from dirty needles.

The National Institute for Health and Care Excellence says growing numbers of people are injecting tanning agents, dermal fillers and Botox at home and in salons, and some are lax about hygiene.

Sharing needles can spread blood-borne diseases like HIV and hepatitis C.

Nice is updating its advice for England and Wales accordingly.

The guidelines, which are out for public consultation, aim to encourage people to use sterile needle and syringe programmes to stem the spread of infections.

Sharps bins

Most blood-borne diseases occur among people who inject drugs like heroin and anabolic steroids.

But NICE says people seeking out cosmetic fixes are also at risk.

“Start Quote

The dangers of sharing needles in cosmetic injectables are so great that any practitioner who does this should be considered guilty of a criminal offence and nothing less”

President of BAAPS Rajiv Grover

A spokeswoman said: “We are seeing an increasing issue with drugs that are used for vanity purposes.”

This includes the anti-wrinkle treatment Botox, dermal fillers and tanning agents.

Prof Mike Kelly, Director of the NICE Centre for Public Health Excellence, said: “Since we last published our guidance on needle and syringe programmes in 2009, we’ve seen an increase in the use of performance-and-image-enhancing drugs such as anabolic steroids, Botox, tanning agents and the use of dermal fillers like collagen.

“We’ve also heard anecdotal evidence that more teenagers are injecting these performance-and-image-enhancing drugs too.

“We’re updating our guidance – and our public consultation on the draft update is an important part of this process – to make sure all of these groups of people are considered in the planning and delivery of needle and syringe programmes.”

One of the recommendations proposed in the new guidelines is that local councils consider providing sharps boxes for people to dispose of used needles and syringes.

Rajiv Grover, consultant plastic surgeon and president of the British Association of Aesthetic Plastic Surgeons (BAAPS), said: “Due to the lack of regulation in the cosmetic sector it is impossible to know how many patients could be at risk of blood borne diseases from needle sharing with either Botox or fillers.

“These should be considered medical procedures and BAAPS has campaigned for over a decade to have this field of non-surgical cosmetic treatments tightly regulated. The dangers of sharing needles in cosmetic injectables are so great that any practitioner who does this should be considered guilty of a criminal offence and nothing less.”

Allergan, a healthcare company that provides Botox, said that Botox is a prescription-only medicine which should only be administered by a trained and qualified medical healthcare professional.

“These treatments should be carried out by appropriately trained and qualified medical practitioners, who have high levels of expertise in full-face anatomy and can provide sufficient aftercare and redress for the patient in the event of an adverse event,” said a spokesman.