Classic myeloproliferative neoplasms, including essential thrombocythemia, polycythemia vera, and primary myelofibrosis, are chronic, clonal hematopoietic stem-cell disorders. These disorders are driven by gain-of-function mutations in the genes Janus kinase 2 (JAK2), calreticulin (CALR), or the thrombopoietin receptor (MPL) that activate cytokine signaling. These mutations arise decades before clinical disease develops and confer a clonal advantage that is further shaped by comutations in epigenetic, splicing, or signaling genes. Inflammation enhances clonal dominance, favoring the development of myelofibrosis and thrombotic complications. Disease evolution may culminate in secondary acute myeloid leukemia, which has a poor prognosis.
Human migration and isolation appear to shape genetic and language diversity in opposite ways.
The finding may seem counterintuitive at first. Regions shaped by migration and population mixing often have higher genetic diversity, so it might seem logical that their languages would be more varied too. Instead, the study finds the reverse pattern.
“We were struck by how robust this inverse relationship is across the globe,” says Anna Graff, lead author of the study and linguist at the University of Zurich. “Places where people have mixed more tend to be genetically diverse, but their languages are structurally more similar. In contrast, places with long-term isolation show less genetic diversity, yet much greater diversity in how languages are structured. Crucially, this relationship holds after adjusting for a wide range of confounding factors, including deep population history such as the timing of continental settlement.”
It is not yet approved, and this does not mean we have “cured cancer.” But it is an extraordinary proof of concept: sequencing + AI/computational biology + mRNA + immunotherapy can be combined to create a treatment tailored to the genetic fingerprint of an individual patient’s tumor.
Moderna and Merck said on Wednesday that a personalized mRNA cancer vaccine reduced the risk of recurrence and spread of melanoma in a late-stage trial, a major success in a new field of cancer treatment that sent Moderna’s shares surging as much as 160%.
Every cell in the body contains essentially the same DNA, yet a brain cell behaves differently from a muscle cell or an immune cell. The difference lies largely in how each cell reads its genetic instructions.
Proteins called transcription factors bind specific DNA sequences and help control when and where genes are active. They direct processes ranging from embryonic development to immune function. When this regulation goes wrong, disease can result. But although the human genome contains around 1,600 transcription factors, the DNA-binding preferences of many have remained unknown.
An international collaboration led by Timothy Hughes at the University of Toronto has now filled many of these gaps in a study published in Nature. The researchers combined five experimental platforms with computational analyses, performing more than 4,800 experiments and identifying DNA-binding motifs for 177 transcription factors that were previously poorly characterized. The work added around 130 distinct motifs to the known vocabulary of human gene regulation.
The first human trial of a therapy designed to make older cells behave more like younger ones has begun. But did it reverse aging? No result has been reported yet.
In this video, Dr Deepa explains what was actually injected, why researchers started with only one eye, how ER-100 uses partial epigenetic reprogramming, and what this Phase 1 trial is really designed to discover.
We also examine a separate Klotho gene therapy being promoted through overseas clinics. What evidence supports it? Why don’t promising animal studies prove that people will live longer? And what risks should anyone understand before considering an experimental anti-aging treatment?
Covered in this video: • ER-100 and partial epigenetic reprogramming. • OSK and the Yamanaka factors. • Why the treatment was injected into one eye. • The doxycycline activation system. • What researchers are monitoring for five years. • Klotho and Minicircle gene therapy. • Regulated trials versus treatments offered overseas. • Tumour, immune and manufacturing risks. • Questions to ask before considering experimental treatment.
The first participant was dosed in a Phase 1 study involving ER-100 for open-angle glaucoma and NAION. The primary purpose is to evaluate safety and tolerability—not to prove that human aging has been reversed.
Dr. Trevor Martin, Ph.D. — Co-Founder & CEO, Mammoth Biosciences
Most of us think of medicine as something we take every day — a statin, a blood pressure pill, or perhaps a monthly injection. But what if those lifelong treatments eventually become obsolete? What if one carefully targeted gene edit could dramatically reduce your risk of heart disease for decades?
Dr. Trevor Martin, Ph.D. is the Co-Founder and Chief Executive Officer of Mammoth Biosciences (https://mammoth.bio/), a pioneering biotechnology company developing next-generation CRISPR genome editing technologies for potentially curative medicines. He co-founded the company in 2017 alongside Nobel Prize-winning CRISPR pioneer Dr. Jennifer Doudna and an interdisciplinary team of leading scientists with the goal of building more precise, versatile, and clinically practical gene editing systems.
Dr. Martin earned his undergraduate degree in Molecular Biology with honors from Princeton University before completing a Ph.D. in Genetics and Statistics at Stanford University, where his research focused on quantitative genetics, genomics, and computational biology. His scientific background in statistics and genetics has shaped Mammoth’s unique approach to developing smaller CRISPR enzymes capable of reaching tissues and diseases that have been difficult to target with first-generation technologies.
Under Dr. Martin’s leadership, Mammoth has grown into one of the world’s leading CRISPR companies, raising more than $465 million, achieving a valuation exceeding $1 billion, and establishing strategic collaborations with companies including Vertex Pharmaceuticals, Bayer, and Regeneron Pharmaceuticals.
Dr. Jessica Dymond, Ph.D. — Vice President of Technology, In-Q-Tel.
The same technologies that could transform medicine, agriculture, and manufacturing could also reshape global security. Understanding the future of biotechnology requires scientists who can bridge discovery, engineering, and responsibility.
Today on Progress, Potential, and Possibilities, we’re joined by a true pioneer at the nexus of biotechnology and national security. Dr. Jessica Dymond, Ph.D is the Vice President of Technology at In-Q-Tel (https://www.iqt.org/), a not-for-profit venture fund that invests in companies advancing the strategic priorities of U.S. Intelligence Community, where she provides technical and strategic leadership to accelerate emerging biotechnologies that address some of the most critical challenges to national and global security.
Before In-Q-Tel, Dr. Dymond served as Chief Scientist for Physical and Life Sciences at the Johns Hopkins University Applied Physics Laboratory, where she led an interdisciplinary portfolio spanning biological sensing, genomic surveillance, microbiome engineering, and synthetic biology. She founded the Lab’s Biological Sciences group and spearheaded initiatives to anticipate, assess, and mitigate emerging biological threats while strengthening global health security.
Formally trained as a synthetic biologist, Dr. Dymond earned her Ph.D. from the Johns Hopkins University School of Medicine, completed a postdoctoral fellowship in functional genomics at the U.S. Department of Agriculture, and has contributed to groundbreaking research, including the design of synthetic yeast genomes and combinatorial genomic diversity. She also serves on the editorial board of Synthetic Biology from Oxford University Press.
Marcus’ legacy lives on, and his philanthropic foundation now nods to one of his original passions: medicine.
https://marcusfoundation.org/” href=“https://marcusfoundation.org/”>The Marcus Foundation, established in 1989 by Bernie and his wife, Billi, donated $28.7 million to Dr. Chuck Murry, director of the University of Southern California’s Stem Cell Center, to advance clinical trials of stem cell-based therapies to regenerate damaged tissue after a heart attack.
“Murry and his team have advanced gene-edited cellular therapy to the point that the time is right to support cells as living medicine in this new form of treatment,” Jonathan Simons, chief science officer of The Marcus Foundation, said in a statement.
You et al. show that size-dependent proteomic remodeling originates at the transcriptomic level. Protein and mRNA turnover contribute minimally to the proteomes of large cells, implicating transcription as the primary driver of size-dependent gene expression. Moreover, mRNA synthesis scales with size through a proportional increase in burst length.
