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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.

Continue reading “Groundbreaking Discoveries About Human Brain and Neuronal Complexity” »

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.”

Sofia Hart has no pulse due to heart failure, and uses TikTok to document her life with an LVAD, a device that pumps her heart while she awaits a heart transplant.

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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Continue reading “23 Younger Biological Age: Supplements, Diet (Blood Test #6 in 2023)” »

One of the most popular dog breeds is the Golden Retriever. Unfortunately, these dogs are also at high risk for developing cancer. New research has investigated genetic factors that may be able to extend the lives of these beloved dogs. This work focused on longevity genes instead of those that have been associated with cancer, and led to the identification of gene variants that could extend the dogs’ lifespan by as much as two years. The findings have been reported in GeroScience.

While most golden retrievers are predisposed to cancer, some of these dogs can live to be as old as 15 or 16 years. So the researchers thought that there might be genetic factors that were mitigating the effect of the cancer-related genes, noted co-corresponding study author Robert Rebhun, Maxine Adler Endowed Chair in oncology at the UC Davis School of Veterinary Medicine. The gene that had this effect was HER4.

A study published today by scientists from Harvard University and epigenetic research company TruDiagnostic has shed light on the reasons why our bodies are aging on a cellular level, laying the foundations for medical based treatment options to reduce the risk of age-related death and disease in highly targeted ways.

Longevity. Technology: Age is the number one risk factor for most chronic diseases and death across the world. Epigenetics (or the way our genes are put to use throughout our bodies) has emerged as a crucial method of evaluating health, and while previous DNA methylation clocks could determine how advanced one’s body has aged, they have not yet been able to provide information to the reasons why someone might have accelerated or decelerated aging outcomes.

“In our research, we set out to create the best method to quantify the biological aging process. However, aging is extremely complex,” explains Harvard Medical School Associate Professor Dr Jessica Lasky-Su. “To solve this issue of complexity, our approach was to gather data across multiple sources of information. We chose to do this by building one of the most robust aging datasets in the world by quantifying patients’ proteomics, metabolomics, clinical histories and DNA methylation.”