Aussie scientists have developed a new gene-editing technique that could be a major breakthrough. It could allow scientists to make accurate and more significant changes to DNA.
#Science #GeneEditing #genes
A team led by NCI researchers has developed an artificial intelligence (AI) tool that uses data from individual cells inside tumors to predict whether a person’s cancer will respond to a specific drug. Learn more about how these findings hold promise for optimally matching cancer drugs to patients:
Precision oncology, in which doctors choose cancer treatment options based on the underlying molecular or genetic signature of individual tumors, has come a long way. The Food and Drug Administration has approved a growing number of tests that look for specific genetic changes that drive cancer growth to match patients to targeted treatments. The NCI-MATCH trial, supported by the National Cancer Institute, in which participants with advanced or rare cancer had their tumors sequenced in search of genetic changes that matched them to a treatment, has also suggested benefits for guiding treatment through genetic sequencing. But there remains a need to better predict treatment responses for people with cancer.
A promising approach is to analyze a tumor’s RNA in addition to its DNA. The idea is to not only better understand underlying genetic changes, but also learn how those changes impact gene activity as measured by RNA sequencing data. A recent study introduces an artificial intelligence (AI)-driven tool, dubbed PERCEPTION (PERsonalized single-Cell Expression-based Planning for Treatments In ONcology), developed by an NIH-led team to do just this.1 This proof-of-concept study, published in Nature Cancer, shows that it’s possible to fine-tune predictions of a patient’s treatment responses from bulk RNA data by zeroing in on what’s happening inside single cells.
This may be about as wildly entertaining, disruptive and philosophically profound as legitimate scientific research gets. Michael Levin’s work in cellular intelligence, bioelectrical communication and embodied minds “is going to overturn everything.”
Giorgia Marucci of HORIBA explains how Jennifer Doudna, Emmanuelle Charpentier and their research teams revolutionized genetic engineering with their CRISPR-Cas9 discovery. Their groundbreaking approach to DNA editing elevated these two scientists to Nobel Laureate status when they received the Nobel Prize in Chemistry in 2020.
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MIT researchers have innovated a method to observe the interaction between genes and enhancers by monitoring their activation times, helping to pinpoint drug targets for genetic disorders. This technique also enhances understanding of eRNA’s function in gene regulation and disease treatment.
Gene Expression and Enhancer Mapping
Although the human genome contains about 23,000 genes, only a fraction of those genes are turned on inside a cell at any given time. The complex network of regulatory elements that controls gene expression includes regions of the genome called enhancers. These are often located far from the genes that they regulate.
The CRISPR gene-editing technique has revolutionised biology, but now an even more powerful system called bridge editing could let us completely reshape genomes.
Neurogenetic disorders, such as neurofibromatosis type 1 (NF1), are diseases caused by a defect in one or more genes, which can sometimes result in cognitive and motor impairments. Better understanding the neural underpinning of these disorders and how they affect motor and cognitive abilities could contribute to the development of new treatment strategies.
Researchers at Stanford University and Washington University School of Medicine recently performed a study on mice aimed at investigating the impact of Nf1 gene mutations, which cause the NF1 neurogenetic disorder, on oligodendroglial plasticity, an adaptive brain process known to contribute to cognitive and motor functions.
Their findings, published in Nature Neuroscience, provide strong evidence that Nf1 mutations delay the development of oligodendroglia, a type of glial cells that support the functioning of the central nervous system, causing disruptions in motor learning.
An immunotherapy drug given before surgery instead of chemotherapy meant that over ten times more patients with a certain genetic profile were cancer-free after surgery, according to clinical trial results presented by researchers at UCL and UCLH.
The findings, presented at the American Society of Clinical Oncology (ASCO) Annual Meeting 2024, are interim results from the NEOPRISM-CRC phase II clinical trial assessing whether the immunotherapy drug pembrolizumab can improve outcomes for patients with stage two or stage three MMR deficient/MSI-High bowel cancer. The trial was a collaboration among UCL, UCLH, the Christie NHS Foundation Trust in Manchester, St. James’s University Hospital in Leeds, University Hospital Southampton and the University of Glasgow.
Bowel cancer is the fourth most common cancer in the UK, with around 42,900 cases a year. Though still predominantly a cancer that affects older people, cases among the under 50s have been increasing in recent decades.
The enzyme telomerase can prevent telomere attrition from happening by extending the length of telomeres. However, in most multicellular organisms, including humans, telomerase expression is switched off, except in germ cells, some types of stem cells, and certain white blood cells. While this might play a role in preventing cancer, as most cancerous cells must switch telomerase expression back on via mutations to enable runaway replication, numerous studies have shown that increasing telomerase through TERT delays aging and increases longevity of model organisms [1].
The small molecule that could
In the lab, this is usually done by introducing genetic vectors carrying a working copy of the gene that codes TERT. It’s this gene that is switched off in somatic cells. However, gene therapies are complex and expensive, and they are just entering the medical mainstream. What if we could do the same using a small molecule?
In molecular biology, few molecules have had as profound an impact as Cas9. Madeleine King, Kayla Perry, Mitchell McAndrew and Audrone Lapinaite discuss how this multifunctional molecular tool of genetic engineering is revolutionizing multiple fields.
