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Super-Precise CRISPR Tool Enters US Clinical Trials

Nature | Noncommunicable Disease

Super-Precise CRISPR Tool Enters US Clinical Trials

“A high-precision successor to CRISPR genome editing has reached a milestone: the technique, called base editing, has made its US debut in a clinical trial. The trial tests more complex genome edits than those performed in humans so far.

Trial organizers announced on 5 September that the first participant had been treated using immune cells with four base-edited genes, equipping the cells to better target and destroy tumours. The hope is that the approach can tame trial participants’ difficult-to-treat form of leukaemia and serve as a gateway to more complex edits in the future.”

From Nature.

New York Post | Communicable Disease

Researchers Discover Cheap Way to Shorten Children’s Colds

“For the study, researchers taught parents how to make and apply salt water drops to their children’s noses. Three drops were administered to each nostril at least four times a day until the 150 young children got better.

The remaining 151 kids were given their typical cold treatment.

‘We found that children using salt water nose drops had cold symptoms for an average of six days where those with usual care had symptoms for eight days,’ Cunningham said. ‘The children receiving salt water nose drops also needed fewer medicines during their illness.’

Only 46% of nose drop households reported family members catching the child’s cold, versus 61% of the other households.

Eighty-two percent of parents said the nose drops helped their child get better quickly, with 81% declaring that they would use drops in the future.”

From New York Post.

Irish Examiner | Vaccination

First 100,000 Doses of Mpox Vaccine Reach DRC

“The first batch of mpox vaccine has arrived in the Democratic Republic of the Congo’s capital, the country’s authorities said.

It comes three weeks after the World Health Organisation (Who) declared mpox outbreaks in 12 African countries a global emergency.

The 100,000 doses of the MVA-BN vaccine, manufactured by the Danish company Bavarian Nordic, have been donated by the European Union through Hera, the bloc’s agency for health emergencies.

Another 100,000 are expected to be delivered on Saturday.”

From Irish Examiner.

Our World in Data | Noncommunicable Disease

HIV Antiretroviral Therapy Saves over a Million Lives Each Year

“Four decades ago, when HIV was first identified, it was an invariably fatal disease: nearly 100% of those infected died, typically within a few years.

The virus spread rapidly around the world — especially in Africa, where almost two million were dying every year by the millennium.

Thankfully, medical advances and global public health efforts have entirely changed this course. Modern antiretroviral therapy is very effective in both treating HIV — returning people with HIV to near-normal life expectancy — and preventing the virus from spreading to others.

The chart above shows this impact based on data from UNAIDS. Over a million people’s lives are now saved by antiretroviral therapy each year.”

From Our World in Data.

Washington Post | Noncommunicable Disease

Diabetes Took over Her Life, until a Stem Cell Therapy Freed Her

“Smith, a 35-year-old nurse from London, Ontario, has Type 1 diabetes, which wipes out critical islet cells within the pancreas that produce insulin. Without them, Smith relied on vials of insulin from a pharmacy and constant vigilance to stay alive. ‘You have to pay attention to your diabetes, or you die.’

On Valentine’s Day 2023, doctors transplanted replacement islet cells, grown in a lab from embryonic stem cells, into a blood vessel that feeds Smith’s liver. By August, she no longer needed insulin. Her new cells were churning it out…

Smith is one of a dozen patients who have received a full dose of islet cells generated in a laboratory from stem cells. Eleven of the patients in the clinical trial drastically reduced taking insulin or stopped altogether, according to data presented at an American Diabetes Association meeting in June.

Despite the promise, the therapy developed by Vertex Pharmaceuticals remains in early stages, and many experts consider it a major step forward, not the finish line.

No one knows how long these cells will keep churning out insulin or whether the therapy is safe long-term until it is tested and followed up in more patients, who must take immune-suppressing drugs to prevent their body from rejecting the foreign cells.”

From Washington Post.