🌞 Cure for Beta-Thalassemia

Daily Upsider - Saturday, May 10th, 2025

Saturday, May 10th, 2025

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Today’s Upside

Health Science

Cure for Beta-Thalassemia

Rahemeen Nabeel – credit, Childrens Hospital of Philadelphia, released

In 2020, researchers began exploring CRISPR gene editing to treat beta-thalassemia, a hereditary blood disorder that impairs the body's ability to produce hemoglobin. Fast forward to today, a promising new gene therapy called Zynteglo has shown real-world success. Developed at the Children’s Hospital of Philadelphia (CHOP), Zynteglo has already been used to treat a 12-year-old patient named Rahemeen Nabeel, with additional cases underway.

Zynteglo works by reprogramming a patient’s own cells. Doctors extract cells and convert them into stem cells, which are then genetically modified to correct the faulty gene responsible for beta-thalassemia. The patient undergoes chemotherapy to prepare the body for the modified cells. Dr. Timothy Olson, who oversaw Rahemeen’s treatment, emphasized that this approach aims for lasting change—and in her case, it appears to have succeeded. Once dependent on frequent blood transfusions and plagued by pain, Rahemeen is now considered cured.

The therapy marks a major advance over traditional treatments, such as bone marrow transplants, which require a matched donor and come with significant risks and limitations. Zynteglo instead uses so-called “Yamanaka factors,” named after Nobel laureate Shinya Yamanaka, to convert adult cells into pluripotent stem cells capable of becoming nearly any cell type in the body. This breakthrough could redefine what’s possible for patients with genetic blood disorders.

Culture

‘Smiling’ Amphibian Thrives Again

A captive-bred leucistic Axolotl – credit, LaDame Bucolique, via Pixabay

In Mexico, a groundbreaking captive-breeding program has successfully released 18 critically endangered axolotls into restored wetland habitats—and all 18 have survived. Recaptured after release, each animal had gained weight, suggesting they are not only adapting but actively hunting in their new environment. “The amazing news is that they all survived, and not only that, but the ones that we recaptured had gained weight—so they’re hunting,” said Dr. Alejandra Ramos, one of the scientists leading the effort.

Unlike many endangered species in Latin America, whose populations have declined due to poaching and deforestation, the axolotl’s downfall began centuries ago. When Spanish colonizers drained the vast lake system that once fed the Valley of Mexico—now Mexico City—they destroyed the axolotl’s final stronghold. Though nearly extinct in the wild, axolotls are common in labs and aquariums due to their extraordinary biology. They retain juvenile features into adulthood, do not undergo metamorphosis, and can regenerate limbs and organs, making them invaluable to medical research.

The released axolotls now inhabit a blend of artificial wetlands, remnants of Lake Xochimilco, and newly restored natural zones. Local farmers helped with the habitat restoration, and natural filtration systems were added to improve water quality. For Dr. Ramos and co-lead scientist Luis Zambrano, the project is as much about conservation as it is about optimism. “If we can restore this [wetland] habitat and restore the axolotl’s population in a city of more than 20 million people,” Dr. Zambrano told the BBC, “I feel that we have hope for humanity.”

World News

Oldest Star Log

A rubbing of the Song Dynasty stone star chart at Suzhou – credit, public domain

Using digital analysis of ancient records, scientists now argue that Chinese astronomer Shi Shen—not the Greeks—created the world’s first star catalogue. Known as the Star Manual of Master Shi, the document likely dates to around 335 BCE and includes the names and spherical coordinates of 120 stars. However, the absence of a timestamp has long complicated efforts to authenticate its age, especially given Earth’s axial precession, which gradually alters star positions over thousands of years.

To overcome this, researchers from the National Astronomical Observatories under the Chinese Academy of Sciences employed a shape-detection algorithm based on the Hough transform. By comparing Shi’s catalogue with star maps from the Tang and Yuan dynasties—spanning over 1,300 years—they tested 10,000 potential creation dates. The best match aligned with how the stars would have appeared around 335 BCE, during Shi Shen’s lifetime.

The analysis also revealed that the chart was revised in 125 BCE by Zhang Heng, the Han Dynasty’s Grand Astronomer. Using China’s first armillary sphere, Zhang recorded 2,500 stars and 124 constellations. Researchers Zhao Yongheng and He Boliang found 59 overlapping stars between Zhang’s and Shi’s charts, suggesting Zhang expanded upon Shi’s original work. These findings offer compelling evidence that ancient Chinese astronomers were the first to systematically map the heavens using coordinate-based methods—predating Greek efforts by more than a century.

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