Fyodor Urnov, PhD, is a pioneer in the field of genome editing and one of the scientists most invested in expanding the availability and utility of CRISPR-based therapies to the broadest possible population. He envisions a world in which genome editing can treat the nearly 400 million people who are suffering from one of the 7,000 diseases brought on by gene mutations.
We know that migraines, which are recurrent and sometimes debilitating headaches, have some genetic basis, but the link with our DNA isn’t entirely clear. Newly identified genetic variants could help in developing treatments.
By Chen Ly
A type of cell once only thought to exist in the gills of freshwater fish and the skin of frogs, but recently found in humans lungs, has given scientists new insight into the underlying cause of cystic fibrosis (CF).
CF is a progressive, genetic disease that impacts the lungs and other organs, sometimes causing severe symptoms that can be life-threatening.
The disease is marked by the absence or mutation of a protein in the lungs called the cystic fibrosis transmembrane conductance regulator (CFTR).
The only cure for painful sickle cell disease today is a bone marrow transplant. But soon there may be a new cure that attacks the disorder at its genetic source.
On Tuesday, advisers to the Food and Drug Administration will review a gene therapy for the inherited blood disorder, which in the U.S. mostly affects Black people. Issues they will consider include whether more research is needed into possible unintended consequences of the treatment.
If approved by the FDA, it would be the first gene therapy on the U.S. market based on CRISPR, the gene editing tool that won its inventors the Nobel Prize in 2020.
A team of researchers has developed a software tool called DANGER (Deleterious and ANticipatable Guides Evaluated by RNA-sequencing) analysis that provides a way for the safer design of genome editing in all organisms with a transcriptome. For about a decade, researchers have used the CRISPR technology for genome editing. However, there are some challenges in the use of CRISPR. The DANGER analysis overcomes these challenges and allows researchers to perform safer on-and off-target assessments without a reference genome. It holds the potential for applications in medicine, agriculture, and biological research.
Their work is published in the journal Bioinformatics Advances on August 23, 2023.
Genome editing, or gene editing, refers to technologies that allow researchers to change the genomic DNA of an organism. With these technologies, researchers can add, remove or alter genetic material in the genome.
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Hello and welcome! My name is Anton and in this video, we will talk about recent discoveries about human brain and various types of neuronal cells.
Links:
https://www.nature.com/articles/d41586-023-03192-2
https://nemoarchive.org/
https://www.science.org/collections/brain-cell-census.
https://www.science.org/doi/10.1126/science.adc8810
https://news.rub.de/english/press-releases/2022-06-0…ir-neurons.
https://www.nature.com/articles/s41559-022-01933-6
https://www.nature.com/articles/s41586-023-06502-w.
https://www.nature.com/articles/s41593-023-01284-w.
https://elifesciences.org/articles/76143
Previous video on major discoveries: https://youtu.be/iGdFh3ENjzc.
More about Neanderthals: https://youtu.be/BvrBl9-TbBs.
#brain #neuron #neuroscience.
0:00 Recent papers on the human brain.
1:00 Human brain atlas and 3,000 new types of cells.
2:00 What was this collaboration for?
2:40 Unexpected complexity of cells in certain brain parts.
4:00 Are there a lot of individual differences? Yes!
4:40 Physical structure appears same across species.
5:10 Genetic activity is very different though.
5:35 Human disorders are unique to humans.
6:30 Unusual layers protecting the brain — SLYM
7:38 Axons turned out to be more unusual, especially in other species.
9:35 Shape of the brain suggests apes and humans are similar only until adolescence.
12:15 Hippocampus in humans is unique focusing on vision…explaining art?
13:48 New memory cell discovered.
15:45 Limitations.
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Researchers at Baylor College of Medicine have identified a small molecule named 5D4 that can suppress the growth of breast and ovarian cancers in animal models. 5D4 works by binding to TopBP1 protein in cancer cells, disrupting its interactions with several pathways that promote cancer growth. Combining 5D4 with another cancer inhibitor, talazoparib, enhances the effectiveness of the anti-cancer activity.
The study, published in the Proceedings of the National Academy of Sciences, strongly supports continuing the investigation toward further developing this strategy for clinical use.
“Cancer development involves many steps of genetic alterations and signaling pathway deregulation. About 10 years ago, our team discovered that protein TopBP1 is at a convergent point of multiple cellular pathways involved in cancer growth and progression, making it a potential candidate for targeted cancer therapy,” said corresponding author Dr. Weei-Chin Lin, professor of medicine-hematology and oncology and of molecular and cellular biology at Baylor. He also is a member of Baylor’s Dan L Duncan Comprehensive Cancer Center. “Our idea was to identify molecules that would bind to TopBP1 and interfere with its interactions with molecular pathways that promote cancer growth.”
A threat actor who claimed responsibility for the compromise of the 23AndMe site earlier this month has released a new dataset, including the records of more than 4 million people’s genetic ancestry.
The cybercriminal, known by the handle Golem, alleges in a cybercrime Dark Web forum the stolen data includes information on, “the wealthiest people living in the US and Western Europe,” according to reports.
23andMe spokesperson Andy Kill said in a statement the organization is still trying to confirm whether the most recently leaked data is genuine.
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Fyodor Urnov, PhD, is a pioneer in the field of genome editing and one of the scientists most invested in expanding the availability and utility of CRISPR-based therapies to the broadest possible population. He envisions a world in which genome editing can treat the nearly 400 million people who are suffering from one of the 7,000 diseases brought on by gene mutations.