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Category: biotech/medical – Page 110

Two remarkable innovations coming together to tackle prion disease: AAVs that leverage human receptors to cross the blood-brain-barrier + a way of epigenetically silencing the gene encoding prions. I recall reading those cited papers and both are amazing!

BOSTON and NEW YORK, Feb. 28, 2025 /PRNewswire/ — Apertura Gene Therapy, a biotechnology company focused on innovative gene therapy solutions, supports the Broad Institute of MIT and Harvard, and the Whitehead Institute in advancing a gene therapy approach for the treatment of prion disease. The project is led by the Vallabh-Minikel lab at the Broad Institute which is focused on finding a cure for prion disease, and their approach leverages two cutting-edge technologies developed at the Institutes of both the Broad and Whitehead: the CHARM platform designed in Dr. Jonathan Weismann’s lab, and TfR1 capsid, an engineered AAV designed in the lab of Dr. Ben Deverman, Director of Vector Engineering at the Broad Institute and scientific founder of Apertura.

Prion disease is a rare, fatal, neurodegenerative disorder caused by misfolded proteins. The new gene therapy aims to address the root cause by using CHARM (Coupled Histone tail for Autoinhibition Release of Methyltransferase) to target and silence the gene that codes for the disease-causing protein1. This payload will be combined with Apertura’s TfR1 capsid, an adeno-associated virus (AAV) capsid engineered to efficiently cross the blood-brain barrier by binding to the human TfR1 receptor, which facilitates iron transport into brain cells2. Together, these technologies represent a transformative approach to tackling CNS diseases.

“We are thrilled to see the progress being made in the development of this innovative therapy for prion disease,” said Dr. Sonia Vallabh, co-leader of the group at the Broad working on preventative therapies for prion disease. “The collaborative efforts between Apertura, the Broad Institute and the Whitehead mark a significant milestone toward addressing unmet needs in neurodegenerative disorders.”

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Bile duct cancer, also called cholangiocarcinoma, is a rare disease in which cancer cells form in the bile ducts. Learn more about this cancer and treatment options.

External and internal radiation therapy are used to treat bile duct cancer and may also be used as palliative therapy to relieve symptoms and improve quality of life.

Learn more about Radiation Therapy to Treat Cancer and Radiation Therapy Side Effects.

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“Of these children, 85% are going to beat their cancer, but it’s a win at a cost,” says Armstrong. “We know that these kids will have shortened lifespans. They often die young of chronic diseases like heart disease, stroke or secondary cancers which present much earlier. And we discovered about a decade ago that this is because they’re ageing much faster than their chronological age.”

In particular, this is reflected not just in their biology, but in physical frailty. When Kirsten Ness, a physical therapist and clinical epidemiologist at St Jude, assessed a group of childhood cancer survivors aged 24–41, she noted that when it came to heart function, flexibility, respiratory capacity and range of motion, they resembled people decades older. “We showed that at 30, they have physiological frailty that resembles people in their 70s and 80s, and it’s getting worse over time,” says Ness.

The underlying cause of this is senescence, a state in which cells cease to continue dividing as normal, but instead simply linger, refusing to die. Because of this quality, senescent cells have sometimes been described as “zombie cells” and they are now regarded as a driving force and a reflection of ageing. Over the course of a lifetime, our bodies incur increasing amounts of damage which in turn makes many of our cells, distributed throughout our body, more likely to become senescent.

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Frequency: Daily, 7-Day.

Indeed calculates the index change in seasonally-adjusted job postings since February 1, 2020, the pre-pandemic baseline. Indeed seasonally adjusts each series based on historical patterns in 2017, 2018, and 2019. Each series, including the national trend, occupational sectors, and sub-national geographies, is seasonally adjusted separately. Indeed switched to this new methodology in December 2022 and now reports all historical data using this new methodology. Historical numbers have been revised and may differ significantly from originally reported values. The new methodology applies a detrended seasonal adjustment factor to the index change in job postings. For more information, see Frequently Asked Questions regarding Indeed Data.

Copyrighted: Pre-approval required. Contact Indeed to request permission to use the data at their contact information provided here.

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Discount Links/Affiliates:
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NAD+ Quantification: https://www.jinfiniti.com/intracellular-nad-test/

Continue reading “A Low RDW Is Associated With A Longer Lifespan (Clip)” | >

Preclinical trial reveals how beta-glucan, a compound found in all fungi, can ‘reprogram’ immune cells to combat lung inflammation.

A recent study suggests that a common fungal component may help protect against flu-related lung damage.

Led by Professor Maziar Divangahi from McGill’s Faculty of Medicine and Health Sciences and the Research Institute of the McGill University Health Centre, the research team found that beta-glucan, when given to mice before influenza exposure, reduced lung damage, improved lung function, and lowered the risk of severe illness and death.

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Researchers studying a protein linked to a rare, severe disease have made a discovery that sheds light on how cells meet their energy needs during a severe metabolic crisis. The findings could lead to new treatments for the disease and open new avenues of research for other conditions involving impaired fat metabolism.

When scientists at the Centre for Genomic Regulation (CRG) in Barcelona first identified a handful of protein-coding genes called TANGO in 2006, they had no idea that one of them, TANGO2, would eventually be linked to a life-threatening disorder in children. In 2016, the researchers found that mutations in TANGO2 cause a now officially recognized as TANGO2 Deficiency Disorder (TDD).

There are about 110 known patients with TDD worldwide, though there are thought to be an estimated six to nine thousand undiagnosed patients in total.

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Dear Colleagues.

In the context of an ageing world population, certain pathologies that are exacerbated in this process of ageing, such as osteoarthritis (OA), will become more prevalent in the coming years. Moreover, OA is one of the main causes of chronic pain and physical disability in the elderly. It is therefore of great relevance to gain a deep understanding on the pathophysiology of this disease, and also to identify potential prognostic and diagnostic tools along with novel promising therapeutic targets for OA.

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