Science is where curiosity, ambition, and innovation meet.
Liz Parrish, founder and CEO of BioViva Science, is spearheading a campaign against the greatest killer on the planet. She stands, unvexed by criticism and convention, in the vanguard of bringing tomorrow’s treatments to those who need them today.
Her journey began when her son was diagnosed with type-1 diabetes.
Through years of engineering gene-editing systems, researchers have developed a suite of tools that enable the modification of genomes in living cells, akin to “genome surgery.” These tools, including ones based on a natural system known as CRISPR/Cas9, offer enormous potential for addressing unmet clinical needs, underscored by the recent FDA approval of the first CRISPR/Cas9-based therapy.
A relatively new approach called “prime editing” enables gene-editing with exceptional accuracy and high versatility, but has a critical tradeoff: variable and often low efficiency of edit installation. In other words, while prime edits can be made with high precision and few unwanted byproducts, the approach also often fails to make those edits at reasonable frequencies.
In a paper that appeared in print in the journal Nature on April 18, 2024, Princeton scientists Jun Yan and Britt Adamson, along with several colleagues, describe a more efficient prime editor.
SCIENTISTS are developing a DNA test to tell if a man’s prostate cancer will come back.
Patients who are at genetic risk of tumour cells flying under the radar and surviving radiotherapy could get extra treatment to make sure they are all wiped out.
Scientists at the Leibniz Institute of Plant Biochemistry (IPB) have succeeded for the first time in stably and precisely inserting large gene segments into the DNA of higher plants very efficiently. To do this, they optimized the gene-editing method CRISPR/Cas, commonly known as “genetic scissors.”
The improved CRISPR method offers great opportunities for the targeted modification of genes in higher plants, both for breeding and research. The study, led by Prof. Alain Tissier and Dr. Tom Schreiber, has been published in Molecular Plant.
CRISPR/Cas is a method with enormous potential for the targeted modification of individual genes. However, this does not apply to all kinds of genetic modifications that breeders and scientists have on their wish lists. While the genetic scissors are ideal for knocking out genes, i.e., switching off or removing existing genes, they do not work well for precisely inserting genes or replacing gene segments. To date, genetic scissors have been too inefficient and therefore of little use for the targeted insertion of genes into the DNA of higher plants.
Paralog synthetic lethals have been assessed with multiple CRISPR-based methods, but systematic comparison among these platforms is unavailable. Here, the authors systematically compare combinatorial perturbation platforms and establish the in4mer CRISPR/Cas12a multiplex knockout platform.
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A recent study published in the journal Nature by an international team of 279 scientists, including three biologists from the University of Michigan, provides the latest insights into the flowering plant tree of life.
Using 1.8 billion letters of genetic code from more than 9,500 species covering almost 8,000 known flowering plant genera (ca. 60%), this achievement sheds new light on the evolutionary history of flowering plants and their rise to ecological dominance on Earth.
Led by scientists at the Royal Botanic Gardens, Kew, the research team believes the data will aid future attempts to identify new species, refine plant classification, uncover new medicinal compounds, and conserve plants in the face of climate change and biodiversity loss.
Single-cell multiplexing techniques (cell hashing and genetic multiplexing) combine multiple samples, optimizing sample processing and reducing costs. Cell hashing conjugates antibody-tags or chemical-oligonucleotides to cell membranes, while genetic multiplexing allows to mix genetically diverse samples and relies on aggregation of RNA reads at known genomic coordinates. We develop hadge (hashing deconvolution combined with genotype information), a Nextflow pipeline that combines 12 methods to perform both hashing-and genotype-based deconvolution. We propose a joint deconvolution strategy combining best-performing methods and demonstrate how this approach leads to the recovery of previously discarded cells in a nuclei hashing of fresh-frozen brain tissue.
MEDIA ADVISORY: An international team of researchers, led by NCI scientists, has identified 50 new areas across the human genome that are associated with the risk of developing kidney cancer.
In a new analysis of genetic susceptibility to kidney cancer, an international team of researchers has identified 50 new areas across the genome that are associated with the risk of developing kidney cancer. These insights could one day be used to advance our understanding of the molecular basis of kidney cancer, inform screening efforts for those at highest risk, and identify new drug targets. The study was led by scientists at the National Cancer Institute (NCI), part of the National Institutes of Health (NIH).
A previous genome-wide association study (GWAS) of people of European ancestry identified 13 regions of the genome that are associated with kidney cancer risk. However, the study population was not diverse. To identify additional regions, researchers conducted a GWAS in participants of many different genetic ancestries that included 29,020 people with kidney cancer and 835,670 people without kidney cancer. Analysis of the data, which came from published studies, biobanks, and a new study, resulted in the identification of 50 new regions associated with the risk of developing kidney cancer, bringing the total number of such regions to 63.
Continue reading “New study provides genomic insights into kidney cancer risk” »
A unique genetic mutation in two siblings – that has never been seen in anyone else – has been discovered by UK researchers at the University of Exeter, pointing the way towards new treatment options for type 1 diabetes.
The mutation is in the gene for a protein called programmed death-ligand 1 (PD-L1), and a new study explains how it may be responsible for the autoimmune form of diabetes that the children developed at a very young age.
“We searched the globe, looking at all the large-scale datasets that we know of, and we haven’t been able to find another family,” says molecular geneticist Matthew Johnson, from the University of Exeter in the UK.
