Gene therapy slows Huntington's disease progression by 75%; High Seas Treaty protections advance; TRAPPIST-1E atmosphere hints; paracetamol pregnancy safety clarified; gravitational waves explained

Executive snapshot

The Naked Scientists cover five major science stories: a gene therapy approach that slows Huntington's disease progression by 75% through direct brain delivery of a suppression gene, with notable patient improvements and manageable safety concerns; the UN High Seas Treaty moving toward formal international law to protect oceanic ecosystems and migratory species; the James Webb Space Telescope's tantalizing hints that TRAPPIST-1E may possess a nitrogen-rich atmosphere, a potential prerequisite for habitability; a careful, evidence-based update on paracetamol use during pregnancy and autism risk, arguing for continuing current guidance; and a clear explanation of gravitational waves, energy conservation over cosmic distances, and redshift as observed in the data from LIGO and related experiments.

Gene therapy for Huntington's disease

In this edition, the show reports a breakthrough in Huntington's disease (HD) treatment. A gene therapy approach uses a harmless viral vector (AAV) to deliver a DNA payload into striatal neurons, with the aim of reducing the production of the mutated Huntington protein. In a three-year follow-up involving around 30 patients, 12 of whom received a high dose, the researchers observed a 75% slowing of disease progression, vastly exceeding prior expectations. The intervention requires direct brain delivery via a neurosurgical procedure lasting 12 to 18 hours. Importantly, there was no placebo group due to ethical considerations; instead, treated participants were compared with a large observational cohort to estimate expected progression. A minority (~10%) experienced an inflammatory reaction that was managed with short-term anti-inflammatory treatment, with no lasting adverse effects reported. Patient stories included improved functional status and one case of returning to work, underscoring the potential real-world impact of this therapy.

High Seas Treaty advances

The program then covers the UN High Seas Treaty, which has been ratified by several nations and will enter into international law in January. The treaty seeks to protect ocean ecosystems beyond national jurisdictions, including migratory species such as tunas, seabirds, and large pelagic species, by enabling the creation of marine protected areas (MPAs) on the high seas. While many countries anticipate full adherence, some, including major players, have not yet ratified the agreement. The discussion highlights the difference between signing and ratification and notes that even signatories face geopolitical pressures as global fisheries and potential deep-sea mining activities continue to threaten biodiversity. The potential to safeguard critical migratory corridors and hotspots of biodiversity is framed as a watershed moment for marine conservation.

TRAPPIST-1E and exoplanet habitability

The program shifts to space science, reporting that TRAPPIST-1E, an Earth-sized world 40 light-years away, shows signs of an atmosphere that may be nitrogen-rich, reminiscent of Earth's air. This finding comes from transmission spectroscopy using the James Webb Space Telescope, which looks for gas-specific absorption features as starlight passes through a planet's atmosphere during transits. The analysis focuses on whether thick hydrogen or CO2 atmospheres are present; their absence would imply a thinner, potentially Earth-like atmosphere. While promising, the researchers caution that red dwarf stars like TRAPPIST-1 are prone to stellar flares, complicating habitability assessments. More data will be needed to determine atmospheric density and surface conditions, including the potential for liquid water and a stable climate.

Paracetamol and pregnancy safety

A separate segment tackles paracetamol use in pregnancy amid political controversy. Experts explain that observational studies linking paracetamol to autism are challenging due to confounding factors such as underlying infections or fever during pregnancy, which themselves may influence outcomes. A Swedish sibling study found no difference in autism risk between pregnancies exposed to paracetamol and those not exposed, strengthening the position that paracetamol remains the safest available analgesic during pregnancy when needed. The discussion also notes the risks of alternative pain relievers such as ibuprofen in pregnancy and the potential unintended consequences of scaremongering that could reduce appropriate treatment for maternal discomfort or illness.

Gravitational waves QOTW

Finally, the podcast presents a question-and-answer segment on gravitational waves, explaining energy conservation across cosmic distances and how detectors like LIGO measure minute spacetime distortions. The energy released in events like black hole mergers is enormous, but the waves attenuate over distance, requiring exquisite detector sensitivity. The concept of redshift for gravitational waves is discussed, clarifying that unlike electromagnetic spectra, gravitational waves lack emission lines and redshift must be inferred via the origin of the source or by combining multimessenger signals when available. The discussion reinforces how these observations illuminate fundamental physics and the dynamic universe.