The technique, which used genetically healthy donor cells, prolonged life and function in mice with a disease similar to Tay-Sachs. It may help with other neurodegenerative diseases like Alzheimer’s.
Category: genetics – Page 7
CRISPR-GPT Turns Novice Scientists into Gene Editing Experts
CRISPR technology has revolutionized biology, largely because of its simplicity compared to previous gene editing techniques. However, it still takes weeks to learn, design, perform, and analyze CRISPR experiments; first-time CRISPR users often end up with low editing efficiencies and even experts can make costly mistakes.
In a new study, researchers from Stanford University, Princeton University, and the University of California, Berkeley, teamed up with Google DeepMind to create CRISPR-GPT, an artificial intelligence (AI) tool that can guide researchers through every aspect of CRISPR editing from start to finish in as little as one day.1 The results, published in Nature Biomedical Engineering, demonstrate that researchers with no previous CRISPR experience could achieve up to 90 percent efficiency in their first shot at gene editing using the tool.
CRISPR-GPT is a large language model (LLM), a type of AI model that uses text-based input data. Led by Le Cong of Stanford University and Mengdi Wang of Princeton University, the team trained the model on over a decade of expert discussions, as well as established protocols and peer-reviewed literature. They designed it to cover gene knockout, base editing, prime editing, and epigenetic editing systems, and benchmarked the tool against almost 300 test questions and answers.
First gene-edited islet transplant in a human passes functional trial
Uppsala University Hospital-led investigators report that gene-edited donor islet cells survived 12 weeks inside a man with long-standing type 1 diabetes without any immunosuppressive medication.
Intensive insulin therapy can delay complications and improve life expectancy. Early-onset type 1 diabetes remains linked to reduced quality of life, serious cardiovascular risk, and shortened lifespan. Toxicity from lifelong immune suppression also drives morbidity and mortality in organ recipients.
In the study, “Survival of Transplanted Allogeneic Beta Cells with No Immunosuppression,” published in the New England Journal of Medicine, researchers conducted a first-in-human open-label trial to test whether hypoimmune-engineered islet cells could evade rejection.
Chinese Scientists Unveil Major Breakthrough in Large-scale DNA Editing
Chinese scientists have developed a gene editing technology capable of precisely manipulating large DNA segments ranging from thousands to millions of base pairs in both plant and animal cells, marking a significant advance in the field of life sciences.
The research team from the Institute of Genetics and Developmental Biology at the Chinese Academy of Sciences announced the new technology in a study published online Monday in the journal Cell.
The method, called PCE (Programmable Chromosomal Engineering), combines three innovative techniques to enable programmable editing of large chromosome segments. In lab tests, researchers successfully inserted an 18,800-base-pair DNA fragment, replaced a 5,000-base-pair sequence, inverted a 12-million-base-pair chromosomal region, deleted a 4-million-base-pair segment, and even relocated entire chromosomes.
Ultra-short RNA insertions offer scalable, cost-effective gene silencing for agriculture
A team of researchers from the Spanish National Research Council has made a significant advance in plant biotechnology by developing a new method for silencing genes. The novel technique uses ultra-short ribonucleic acid (RNA) sequences carried by genetically modified viruses to achieve genetic silencing, allowing the customization of plant traits. The work, published in the Plant Biotechnology Journal, opens up new avenues for crop improvement, functional genomics, and sustainable agriculture.
Viral vector technology involves modifying viruses, removing the genetic material that causes disease, to turn them into vehicles that carry the RNA sequence to be introduced into an organism. This technique, when applied to plants, has already proven effective under experimental conditions in inducing flowering and accelerating the development of improved crop varieties, modifying plant architecture to facilitate adaptation to mechanization, improving drought tolerance, and producing metabolites beneficial to human health, among other applications.
Now, the method developed by the CSIC, together with the Valencian University Institute for Research on the Conservation and Improvement of Agrodiversity (COMAV) and the Italian Department of Applications and Innovation in Supercomputing (Cineca), represents an optimization of technological platforms to accelerate the development and validation of agricultural applications based on viral vectors.
Scientists map the genes behind diet and dementia risk
Concordance was high between imputed and sequenced APOE genotypes. Moreover, the researchers replicated known GWAS associations with diet-related biomarkers.
The authors also noted several limitations to provide context for future research. These include that the study population was predominantly of European ancestry, which may limit the generalizability of findings, and that the specific participant criteria (e.g., overweight, family history of dementia) mean the resource is not representative of the general population. They also advise that potential batch effects from specimen type and study site should be accounted for in future analyses.
This genetic resource enables analyses of genetic contributions to variability in cognitive responses to the MIND diet, supporting integrative analysis with other data types to delineate underlying biological mechanisms. The data will be made available to other researchers via The National Institute on Aging Genetics of Alzheimer’s Disease Data Storage Site (NIAGADS).
Scientists just cracked the code to editing entire chromosomes flawlessly
A group of Chinese scientists has created powerful new tools that allow them to edit large chunks of DNA with incredible accuracy—and without leaving any trace. Using a mix of advanced protein design, AI, and clever genetic tweaks, they’ve overcome major limitations in older gene editing methods. These tools can flip, remove, or insert massive pieces of genetic code in both plants and animals. To prove it works, they engineered rice that’s resistant to herbicides by flipping a huge section of its DNA—something that was nearly impossible before.
Is Altos Labs gearing up for clinical trials?
Longevity biotech giant Altos Labs has appointed Dr Joan Mannick as its Chief Medical Officer and head of product development, signaling a shift toward advancing clinical programs based on the company’s cellular rejuvenation technology. As Life Biosciences reportedly prepares to enter clinical trials with its partial epigenetic reprogramming candidate, is Altos about to join the party?
Altos, which launched with $3 billion in funding in 2022, is focused on reversing disease and age-related decline by restoring cellular health through partial epigenetic reprogramming, a technique inspired by the work of Nobel laureate Shinya Yamanaka and Juan Carlos Izpisua Belmonte. The company’s approach, which reverts cells toward a youthful state without altering their identity, has demonstrated benefits in animal models, extending both lifespan and healthspan in mice.
Although Altos has not yet launched human trials, the appointment of Mannick, who has significant experience designing and running clinical programs in aging biology, indicates the company is shifting into clinical applications of its technology. She will operate within Altos’ Institute of Medicine, collaborating with discovery and development teams to shape the clinical direction of its therapies.
Human CLOCK gene enhances brain connectivity and mental flexibility in mice, study finds
Clock genes are a set of genes known to contribute to the regulation of the human body’s internal 24-hour cycle, also known as the circadian rhythm. One of these genes is the so-called CLOCK gene, a protein that regulates the activity of other genes, contributing to recurrent patterns of sleep and wakefulness.
Past findings suggest that this gene is also expressed in the neocortex, a brain region that supports important cognitive abilities, including reasoning, decision-making and the processing of language. However, the gene’s possible contribution to these specific brain functions remains poorly understood.
Researchers at UT Southwestern Medical Center recently carried out a study on genetically modified mice aimed at better understanding how the expression of the CLOCK gene in the human neocortex influences cognitive functions. Their findings, published in Nature Neuroscience, suggest that the gene plays a role in the formation of connections between neurons, which in turn influence mental and behavioral flexibility.