Lifeboat Foundation

This gene therapy treats LCA1, causing early childhood vision loss, affecting under 100,000 people:

“One patient reported for the first time being able to navigate at midnight outdoors only with the light of a bonfire,” said Cideciyan, who is also co-director of the Center for Hereditary Retinal Degenerations.

Continue reading “Gene therapy restores sight with up to 10,000-fold vision improvement” »

For the study, the researchers analysed blood samples and medical information from 27,939 healthcare providers living in the US, who participated in the Women’s Health Study. The women were on average aged 55 at the study’s start (1992−1995) and followed for 30 years.

During the worst days of the COVID-19 pandemic, many of us became accustomed to news reports on the reproduction number R, which is the average number of cases arising from a single infected case. If we were told that R was much greater than 1, that meant the number of infections was growing rapidly, and interventions (such as social distancing and lockdowns) were necessary. But if R was near to 1, then the disease was deemed to be under control and some relaxation of restrictions could be warranted. New mathematical modeling by Kris Parag from Imperial College London shows limitations to using R or a related growth rate parameter for assessing the “controllability” of an epidemic [1]. As an alternative strategy, Parag suggests a framework based on treating an epidemic as a positive feedback loop. The model produces two new controllability parameters that describe how far a disease outbreak is from a stable condition, which is one with feedback that doesn’t lead to growth.

Parag’s starting point is the classical mathematical description of how an epidemic evolves in time in terms of the reproduction number R. This approach is called the renewal model and has been widely used for infectious diseases such as COVID-19, SARS, influenza, Ebola, and measles. In this model, new infections are determined by past infections through a mathematical function called the generation-time distribution, which describes how long it takes for someone to infect someone else. Parag departs from this traditional approach by using a kind of Fourier transform, called a Laplace transform, to convert the generation-time distribution into periodic functions that define the number of the infections. The Laplace transform is commonly adopted in control theory, a field of engineering that deals with the control of machines and other dynamical systems by treating them as feedback loops.

The first outcome of applying the Laplace transform to epidemic systems is that it defines a so-called transfer function that maps input cases (such as infected travelers) onto output infections by means of a closed feedback loop. Control measures (such as quarantines and mask requirements) aim to disrupt this loop by acting as a kind of “friction” force. The framework yields two new parameters that naturally describe the controllability of the system: the gain margin and the delay margin. The gain margin quantifies how much infections must be scaled by interventions to stabilize the epidemic (where stability is defined by R = 1). The delay margin is related to how long one can wait to implement an intervention. If, for example, the gain margin is 2 and the delay margin is 7 days, then the epidemic is stable provided that the number of infections doesn’t double and that control measures are applied within a week.

UC Merced researchers have found that the protein OTUD6 can alter protein production in cells, potentially affecting lifespan and cancer, with future research aimed at exploiting this for therapeutic benefits.

Researchers at UC Merced used fruit flies to uncover a cellular process shared by many organisms, which could significantly advance the understanding of cancer and aging.

Department of Molecular and Cell Biology Professor Fred Wolf, then-graduate student Sammy Villa, and Genentech Vice President and Senior Fellow in Physiological Chemistry and Research Biology Vishva Dixit, discovered a mechanism that cells use to tune how much protein they make through the process of translating RNA into protein.

New gene therapy breakthrough from the University of Hawai’i offers safer, efficient treatments for genetic disorders like Hemophilia.

The crystal structure the scientists developed can enable computational-based drug discovery to be used to target the i-motifs from the insulin gene, because when scientists know the specific 3D shape, they can design molecules digitally and model them to see whether they will fit.

Scientists can then develop new drugs using particular chemicals when they know which ones will fit the drug target best—a process called rational design.

As the first crystal structure of this type, the researchers say it will also be useful as a model for other targets in the genome, besides the insulin gene, which form this shape of DNA.

This is another episode in our series on the great figures in developmental bioelectricity. We interview (~1 hour) Mustafa Djamgoz, who has made pivotal discoveries about the role of bioelectricity in the problem of cancer and its eventual solution.

Mustafa’s book: / beat-cancer.

Summary: A new gene therapy has restored vision in patients with Leber congenital amaurosis type I (LCA1), a rare genetic condition causing blindness. In a small trial, those receiving the highest dose saw up to a 10,000-fold improvement in light sensitivity and significant gains in reading and navigation abilities.

The therapy, developed by researchers, uses a virus-based system to deliver a functioning gene into the retina’s light-sensitive cells. The results show promise for expanding this treatment, with further trials planned to confirm safety and efficacy.

Researchers said the study showed nanobots had the potential to transport drugs to precise locations.