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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.

The Redox Activity of Protein Disulphide Isomerase Functions in Non‐Homologous End‐Joining Repair to Prevent DNA Damage

Schematic diagram illustrating the protective role of protein disulphide isomerase (PDI) against DNA damage via non-homologous end-joining (NHEJ), which repairs double stranded DNA breaks (DSBs). Ind…

Two transparent worms shed light on evolution

Two species of worms have retained remarkably similar patterns in the way they switch their genes on and off despite having split from a common ancestor 20 million years ago, a new study finds.

The findings appear in the journal Science.

“It was just remarkable, with this evolutionary distance, that we should see such coherence in gene expression patterns,” said Dr. Robert Waterston, professor of genome sciences at the University of Washington School of Medicine in Seattle and a co-senior author of the paper. “I was surprised how well everything lined up.”

Eye-tracking exhibit helps map gaze behavior development across different life stages

Understanding how people visually browse their surroundings and direct their gaze in specific situations is a long-standing goal among psychology researchers. Past studies suggest that humans exhibit oculomotor biases, which are tendencies that guide the way they look at the world around them, for instance, preferentially directing their gaze around the center of what they are visually exposed to at a given time.

Researchers at Justus Liebig University Giessen in Germany recently carried out a study aimed at better understanding how these patterns in gazing develop throughout the human lifespan. Their findings, published in Nature Human Behaviour, suggest that scene viewing tendencies gradually develop over childhood and adolescence, while older people tend to observe the world following similar viewing and gaze fixation strategies.

“One of the key questions our lab is interested in is how gaze behavior—that is, where and how we look at natural scenes—develops as we grow up,” Marcel Linka, first author of the paper, told Medical Xpress.

Permanent magnet configurations outperform classical arrangement to deliver strong and homogeneous fields

Physicists Prof. Dr. Ingo Rehberg from the University of Bayreuth and Dr. Peter Blümler from Johannes Gutenberg University Mainz have developed and experimentally validated an innovative approach for generating homogeneous magnetic fields using permanent magnets.

Their method outperforms the classical Halbach arrangement—which is optimal only for infinitely long and therefore unrealizable magnets—by producing higher field strengths and improved homogeneity in compact, finite-sized configurations.

The study was published in Physical Review Applied, which shows significant advances in the applied sciences at the intersection of physics with engineering, materials science, chemistry, biology, and medicine.

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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✨ Support EasyPeasy!
Get early access, behind-the-scenes content, and suggest future topics:
👉 / @easypeasylearning.
👉 / supereasypeasy.
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