DAILY DOSE: CRISPR takes on sickle cell disease and is winning; Fusion energy slowly becoming a reality.


An expert panel has deemed a novel treatment for sickle cell disease, known as exa-cel, safe for clinical use, paving the way for a likely FDA approval by Dec. 8. This approval could herald a significant advancement in treating the over 100,000 Americans afflicted by this genetic disorder. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, exa-cel employs the CRISPR gene-editing technique to alleviate the painful symptoms of the disease. If approved, it will be the first CRISPR-based medicine for treating a genetic disorder, setting a precedent for more curative options. By Dec. 20, the FDA will also decide on another potential cure by Bluebird Bio. However, the high cost, which is yet to be disclosed by Vertex, and the intensive treatment process, including chemotherapy and a month-long hospital stay, may pose challenges. Moreover, the historical healthcare inequities could affect the treatment’s adoption within the Black community, which is predominantly affected by sickle cell disease. Despite these hurdles, the new treatment represents a monumental step towards curing this debilitating disease. (New York Times)


The journey towards feasible fusion energy saw progress with the activation of Japan’s JT-60SA, the newest and largest fusion reactor, last week. Utilizing superconducting coils’ magnetic fields, it encases a plasma within a doughnut-shaped vessel, aiming to trigger hydrogen nuclei fusion to unleash energy. This behemoth is engineered to sustain a 200 million degrees Celsius plasma for around 100 seconds, surpassing previous tokamaks. This stride underscores the reactor’s fundamental functionality, says Sam Davis of Fusion for Energy, an EU entity collaborating with Japan’s National Institutes for Quantum Science and Technology (QST) on this and related ventures. It’s anticipated to take two more years for JT-60SA to generate the enduring plasmas essential for substantial physics experiments, mentions Hiroshi Shirai of QST. Furthermore, findings from JT-60SA will be instrumental for ITER, a gigantic fusion reactor being assembled in France, aiming to validate fusion’s energy output surpassing its input. Despite its substantial delay, the JT-60SA venture is a stepping stone towards realizing fusion energy, with plans like DEMO slated for 2050, bridging the gap between JT-60SA, ITER, and commercial fusion power. (Science)

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On October 27, the Department of Defense disclosed its intent to develop a new B61-13 nuclear gravity bomb variant, a continuation of the B61 design legacy since 1963, repurposing old warheads instead of new construction. Unlike its predecessors carried by fighter jets, B61-13 is for nuclear-capable bombers likely including the B-21 Raider and B-2 Spirit stealth bombers, replacing the existing B61-7s. Despite its modern safety and accuracy features akin to the B61-12, its yield remains between 10 to 360 kilotons. The B61-13 aims to provide the President additional options against large, tough military targets, pending Congress approval, with no particular threat prompting this development. Experts argue its necessity, citing the reduced importance of nuclear gravity bombs due to the risks to bombers and the evolving capabilities of long-range missiles. This step represents a policy choice by the Biden administration, reflecting an ongoing effort to modernize the US nuclear arsenal amidst a complex global security landscape. (Popular Science)


A 58-year-old man with heart failure, who received a genetically altered pig heart, passed away nearly six weeks post-transplant at the University of Maryland Medical Center. Lawrence Faucette was the second person to undergo such a procedure aimed at addressing organ donor shortages. The first patient, David Bennett, died last year, two months after his transplant, facing multiple complications including a pig virus in the new heart. Unlike Bennett, Faucette showed initial signs of heart rejection. Despite the setbacks, both cases contribute valuable insights into xenotransplantation, a field that’s seen recent advancements, especially in transplanting modified pig kidneys into brain-dead patients to study organ rejection and functionality. Over 100,000 Americans are living with end-stage organ disease, with a dire shortage of human donor organs, urging medical centers to fervently explore xenotransplantation to bridge the gap between organ demand and availability, potentially saving many lives in the future. (New York Times)

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cOAlition S, the group behind the open-access initiative Plan S, has proposed a bolder plan to transform research publishing. They advocate for all versions of an article and its peer-review reports to be openly published from the start, without any fees, and for authors to decide the publishing details. This proposal aims to shift towards a “community-based” and “scholar-led” system, transitioning publishers from gatekeepers to service providers. While previously, cOAlition S, consisting of influential funding agencies, pressured journals for immediate open-access publishing, this new proposal outlines a broader transformation. It’s part of an ongoing initiative to enhance the accessibility and dissemination of research, aligning with some earlier recommendations by the European Union. The proposal has been endorsed on a small scale but represents a significant departure from current mainstream scholarly communication systems. It’s now under a six-month review process to gather global research community feedback. Supporters view it as a positive step towards equitable open-access publishing, but concerns remain regarding its execution and the continuation of Article Processing Charges (APCs) which may limit open access to those with sufficient funding. (Nature)


Demis Hassabis, co-founder of Google DeepMind, advises against the “Move fast and break things” approach of older tech giants, urging for a cautious path in AI’s evolution due to its significance. Speaking before the UK’s AI safety summit, Hassabis outlined AI threats into three broad categories: generating misinformation and bias, misuse by malicious actors, and controlling future advanced AI. The summit, held at Bletchley Park, aims to discuss maximizing AI benefits while minimizing risks, focusing on extreme threats like bio-terrorism and cyber-attacks, with notable attendees including US VP Kamala Harris and EC President Ursula von der Leyen. Despite critiques of the event’s US-centric guest list and overshadowing announcements on AI safety from the US and G7, Hassabis sees a pivotal role for the UK in shaping global AI discussions. The discussions emphasize the necessity of regulatory frameworks to ensure AI’s safe and responsible deployment, underscoring the industry’s shift towards prioritizing safety and ethics in AI development. (BBC)


The emergence of climate change-driven ghost towns is a growing concern as increasing environmental disasters force communities to abandon their homes. While historically, economic factors led to the creation of ghost towns, climate change now drives this with floods, wildfires, and extreme temperatures causing displacement. The phenomena is already noticeable with recent wildfires and floods globally. Relocating communities is a complex and costly affair, often termed as “managed retreats” or “planned relocations”, where government aids in resettlement. Over 20 million people are displaced annually due to extreme weather, with projections suggesting billions may face unlivable conditions by century’s end, disproportionately affecting impoverished and communities of color. Besides, climate change threatens tourism-reliant areas, yet could spur “dark tourism” to abandoned places, offering educational insight into climate change repercussions. Examples of climate-induced ghost towns include Vunidogoloa in Fiji, Isle de Jean Charles in Louisiana, and Chacaltaya Ski Resort in Bolivia, showcasing the devastating, real-time effects of climate change on human settlements. (CNN)


Nut-bearing trees like oaks and black walnuts experience “on” and “off” years, known as mast years and off years respectively. During mast years, trees vigorously produce nuts, which in turn influences wildlife populations as these nuts are a food source for various animals, leading to increased breeding. The cycle timelines vary among species, with factors like temperature and rainfall believed to influence these cycles, although the exact cause remains uncertain. The phenomenon not only ensures the propagation of tree species but also impacts the ecological system, affecting animal populations even in subsequent years. While mast years may present challenges like increased yard maintenance, they play a crucial role in the ecosystem balance, emphasizing the interconnectedness of nature’s elements and their broader implications on wildlife and perhaps even larger ecosystems. (Associated Press)

Thanks for reading. Let’s be careful out there.

WORDS: The Biology Guy.

IMAGE CREDIT: Vertex Pharmaceuticals.

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