Dec 12, 2023
Bull of the Day: CRISPR Therapeutics (CRSP)
Posted by Paul Battista in categories: biotech/medical, genetics
FDA approval for revolutionary Sickle Cell Disease gene-editing treatment promises a bright future.
FDA approval for revolutionary Sickle Cell Disease gene-editing treatment promises a bright future.
Scientists have grown a tiny brain-like organoid out of human stem cells, hooked it up to a computer, and demonstrated its potential as a kind of organic machine learning chip, showing it can quickly pick up speech recognition and math predictions.
As incredible as recent advances have been in machine learning, artificial intelligence still lags way behind the human brain in some important ways. For example, the brain happily learns and adapts all day long on an energy budget of about 20 watts, where a comparably powerful artificial neural network needs about 8 million watts to achieve anything remotely comparable.
What’s more, the human brain’s neural plasticity, its ability to grow new nervous tissue and expand existing connective channels, has granted it an ability to learn from noisy, low-quality data streams, with minimal training and energy expenditure. What AI systems accomplish with brute force and massive energy, the brain achieves with an effortless elegance. It’s a credit to the billions of years of high-stakes trial and error that delivered the human brain to the state it’s in today, in which it’s chiefly used to watch vast numbers of other people dancing while we’re on the toilet.
Mini brains grown in a lab from stem cells spontaneously developed rudimentary eye structures, scientists reported in a fascinating paper in 2021.
On tiny, human-derived brain organoids grown in dishes, two bilaterally symmetrical optic cups were seen to grow, mirroring the development of eye structures in human embryos.
This incredible result will help us to better understand the process of eye differentiation and development, as well as eye diseases.
A team of chemists, microbiologists and physicists at the University of Cambridge in the U.K. has developed a way to use solid-state nanopores and multiplexed DNA barcoding to identify misfolded proteins such as those involved in neurodegenerative disorders in blood samples. In their study, reported in the Journal of the American Chemical Society, the group used multiplexed DNA barcoding techniques to overcome problems with nanopore filtering techniques for isolating harmful oligomers.
Prior research has shown that the presence of harmful oligomers in the brain can lead to misfolding of proteins associated with neurodegenerative diseases such as Parkinson’s and Alzheimer’s disease. Medical researchers have been looking for a way to detect them in the blood as a way to diagnose neurodegenerative disease and to track the progression once it has been confirmed.
Unfortunately, finding them in complex mixtures such as blood has proven to be a daunting task. One approach has shown promise—using nanopore sensors—but to date, they cannot track target oligomers as they speed through customizable solid-state nanopore sensors. In this new effort, the research team overcame this problem by using customizable DNA nanostructures.
Some chemical tags on DNA, called epigenetic factors, that are present at a young age can affect the maximum life spans of mammal species.
A system that integrates brain cells into a hybrid machine can carry out tasks such as voice recognition.
Rising levels of respiratory viruses, including flu and COVID-19, are being reported this season. CDC urges the public to get vaccinated, with fewer than two in five adults and children having received the flu vaccine. Visitor restrictions are in place at Riley Hospital due to the surge in illnesses.
As Vertex and CRISPR Therapeutics’ exa-cel and Verve Therapeutics’ VERVE-101 move forward, questions remain about possible drawbacks of such therapies.
There’s an unfortunate irony in cell therapy that holds it back from its full potential: Regenerating tissues often must be damaged to know if the treatment is working, such as surgically removing tissue to see if rejuvenation is occurring beneath.
The alternative isn’t much better: Patients can choose to wait and see if their health improves, but after weeks of uncertainty, they might find that no healing has taken place without a clear explanation as to why.
Jinhwan Kim, a new assistant professor of biomedical engineering at the University of California, Davis, who holds a joint appointment with the Department of Surgery at UC Davis Health, wants to change all of that. In his research program, he combines nanotechnology and novel bioimaging techniques to provide non-invasive, real-time monitoring of cellular function and health.
Biotech stocks could be at a major turning point after FDA approval of the first gene editing drug using CRISPR technology to treat sickle cell disease. Gove…