Researchers have released genetic and behavioral data from over 1,500 children and adolescents with autism who were hospitalized in psychiatric units across the U.S.
Category: genetics – Page 13
Cells assembled into Anthrobots become biologically younger than the original cells they were made from
Modern humans have existed for more than 200,000 years, and each new generation has begun with a single cell—dividing, changing shape and function, organizing into tissues, organs, and limbs. With slight variations, the process has repeated billions of times with remarkable fidelity to the same body plan.
Researchers at Tufts have been on a quest to understand the code guiding individual cells to create the architecture of a human being, and to create a foundation for regenerative medicine. As they learn more about that code, they are also looking at how to build living structures from human cells that have totally new forms and capabilities—without genetic manipulation.
To decipher that code, they took a cell from the human body and allowed it to grow in a novel environment to observe how the rules of self-organization play out.
Creating the World’s First CRISPR Medicine, for Sickle Cell Disease
When Vijay Sankaran was an MD-PhD student at Harvard Medical School in the mid-2000s, one of his first clinical encounters was with a 24-year-old patient whose sickle cell disease left them with almost weekly pain episodes.
“The encounter made me wonder, couldn’t we do more for these patients?” said Sankaran, who is now the HMS Jan Ellen Paradise, MD Professor of Pediatrics at Boston Children’s Hospital.
In 2008, Orkin, Sankaran, and colleagues achieved their vision by identifying a new therapeutic target for sickle cell disease.
In December 2023, through the development efforts of CRISPR Therapeutics and Vertex Pharmaceuticals, their decades-long endeavor reached fruition in the form of a new treatment, CASGEVY, approved by the U.S. Food and Drug Administration.
The decision has ushered in a new era for sickle cell disease treatment — and marked the world’s first approval of a medicine based on CRISPR/Cas9 gene-editing technology.
How a genetic insight paired with gene editing technology led to a life-changing new therapy.
Somatic gene delivery faithfully recapitulates a molecular spectrum of high-risk sarcomas
Sarcomas are a group of mesenchymal malignancies which are molecularly heterogeneous. Here, the authors develop an in vivo muscle electroporation system for gene delivery to generate distinct subtypes of orthotopic genetically engineered mouse models of sarcoma, as well as syngeneic allograft models with scalability for preclinical assessment of therapeutics.
🔬Binary Fission Uncovered: DNA Relay-Ratchet Mechanism + Septum Formation!
In this video, we take a deep dive into the fascinating process of binary fission, the primary mode of reproduction in prokaryotic cells like bacteria.
You’ll learn how:
🧬 DNA replication begins the cycle.
⚙️ The DNA relay-ratchet mechanism ensures accurate segregation of chromosomes, and.
🧱 A septum forms to physically divide the cell into two genetically identical daughter cells.
Whether you’re a student, teacher, or just curious about microbiology, this simplified explanation breaks down complex concepts into clear, visual steps.
📚 References & Further Reading:
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Get early access, behind-the-scenes content, and suggest future topics:
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Gene-editing system targets multiple organs simultaneously
A gene-editing delivery system developed by UT Southwestern Medical Center researchers simultaneously targeted the liver and lungs of a preclinical model of a rare genetic disease known as alpha-1 antitrypsin deficiency (AATD), significantly improving symptoms for months after a single treatment, a new study shows.
Gene-editing nanoparticle system targets multiple organs simultaneously
A gene-editing delivery system developed by UT Southwestern Medical Center researchers simultaneously targeted the liver and lungs of a preclinical model of a rare genetic disease known as alpha-1 antitrypsin deficiency (AATD), significantly improving symptoms for months after a single treatment, a new study shows. The findings, published in Nature Biotechnology, could lead to new therapies for a variety of genetic diseases that affect multiple organs.
“Multi-organ diseases may need to be treated in more than one place. The development of multi-organ-targeted therapeutics opens the door to realizing those opportunities for this and other diseases,” said study leader Daniel Siegwart, Ph.D., Professor of Biomedical Engineering, Biochemistry, and in the Harold C. Simmons Comprehensive Cancer Center at UT Southwestern.
Gene editing—a group of technologies designed to correct disease-causing mutations in the genome—has the potential to revolutionize medicine, Dr. Siegwart explained. Targeting these technologies to specific organs, tissues, or cell populations will be necessary to effectively and safely treat patients.
Palm-sized device detects disease markers in under 45 minutes without additional lab equipment
Scientists from the National University of Singapore (NUS) have developed NAPTUNE (Nucleic Acids and Protein biomarkers Testing via Ultra-sensitive Nucleases Escalation), a point-of-care assay that identifies trace amounts of disease-related genetic material, including nucleic acid and protein markers, in less than 45 minutes. Importantly, it accomplished this without the need for laboratory equipment or complex procedures.
Lying at the heart of many modern diagnostics, polymerase chain reaction (PCR) and real-time immunoassays provide high accuracy. However, they are hindered by lengthy processing time, the need for specialized thermal cyclers and skilled personnel. These constraints hamper rapid outbreak management, early cancer screening and bedside decision-making, especially in low-resource settings.
NAPTUNE tackles these challenges by replacing bulky amplification steps with a tandem nuclease cascade that converts biological signals directly into readily detectable DNA fragments, streamlining the diagnostic process.
Study reveals ‘switch-like’ behavior for hundreds of genes with links to human disease
Gene expression, where cells use the genetic information encoded in DNA to produce proteins, has been thought of as a dimmer light.
How much a particular gene gets expressed continually rises and falls, depending on the needs of a cell at any given time. It’s like adjusting the lighting of a room until it’s just right for your mood.
But University at Buffalo researchers have shown that a considerable portion of a human’s roughly 20,000 genes express more like your standard light switch—fully on or fully off.
DNA ‘glue’ could help prevent and treat diseases triggered by ageing
Macquarie University researchers have discovered a naturally occurring protein found in human cells plays a powerful role in repairing damaged DNA — the molecule that carries the genetic instructions for building and maintaining living things.
The discovery, published in the journal Ageing Cell, could hold the key to developing therapies for devastating age-related diseases such as motor neuron disease (MND), Alzheimer’s disease, and Parkinson’s disease.
Hope: Dr Sina Shadfar, pictured, and colleagues discovered a protein which they have shown for the first time acts like a ‘glue’, helping to repair broken DNA, which is widely accepted as one of the main contributors to ageing and the progression of age-related diseases.
The research, conducted by neurobiologist Dr Sina Shadfar and colleagues in the Motor Neuron Disease Research Centre, reveals a protein called protein disulphide isomerase (PDI) helps repair serious deoxyribonucleic acid (DNA) damage. This breakthrough opens new possibilities for therapies aimed at boosting the body’s ability to fix its own DNA — a process that becomes less efficient as we age.