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An ancient RNA-guided system could simplify delivery of gene editing therapies By Corie Lok February 27, 2025 Breadcrumb Home An ancient RNA-guided system could simplify delivery of gene editing therapies The programmable proteins are compact, modular, and can be directed to modify DNA in human cells. Lisa Yang and Hock E.
The octopuses achieve this by editing their RNA, the messenger molecule between DNA and proteins. Researchers report that two-spot octopuses adapt to seasonal temperature shifts by producing different neural proteins under warm versus cool conditions.
Functional analysis based on these structures also revealed how a 'prime editor' could achieve reverse transcription, synthesizing DNA from RNA, without 'cutting' both strands of the double helix. New research has determined the spatial structure of various processes of a novel gene-editing tool called 'prime editor.'
The system can be formulated completely as RNA, dramatically simplifying delivery logistics compared to traditional systems that use both RNA and DNA. Investigators have developed STITCHR, a new gene editing tool that can insert therapeutic genes into specific locations without causing unwanted mutations.
Genes contain instructions for making proteins, and a central dogma of biology is that this information flows from DNA to RNA to proteins. But only two percent of the human genome actually encodes proteins; the function of the remaining 98 percent remains largely unknown.
The work appears today in Science. The scientists found a surprising number and diversity of CRISPR systems, including ones that could make edits to DNA in human cells, others that can target RNA, and many with a variety of other functions. Zhang’s team thinks the new systems could be adapted for diagnostic technologies as well.
Their new editors are more efficient and specialized than previous versions, and are able to modify DNA in cultured cells and in animals that have been difficult to edit, including in immune system cells and inside the brain. Jordan Doman and Smriti Pandey, graduate students in the Liu lab, are co-first authors of the study.
In the new work published today in Nature Biotechnology , the team adapted engineered virus-like particles (eVLPs) that they had previously designed to carry base editors — another type of precision gene editor that makes single-letter changes in DNA. Paper cited: An, M.
What I love most about science in general, and genetics in particular, is when new findings upend everything we thought we knew about something. That was so in 1977, when “intervening DNA sequences” – aka “introns” – were discovered to interrupt protein-encoding genes.
These tough nuts to crack in medical science—biological targets known to play roles in diseases but resistant to traditional drug design—are now seeing new strategies that shift the paradigm from "undruggable" to "druggable." Beyond Proteins: DNA and RNA Frontier The story doesn’t end with proteins.
By Leah Eisenstadt, Broad Communications October 23, 2024 Credit: Courtesy of the Broadbent family Brian and Julia Broadbent are raising their daughters Claire, top left, and Emma, seated, who is the first person to be diagnosed with a rare genetic disorder caused by the long noncoding RNA CHASERR.
Our overall mission at Arrakis is to expand the set of “druggable” targets for small-molecule medicines to include RNA. Today, we are pleased to announce that our article describing one such platform: “ PEARL-seq, A Photoaffinity Platform for the Analysis of Small Molecule-RNA Interactions ” was published in ACS Chemical Biology.
Although the researchers followed all of these events with color-labeled DNA, the details of how the cells at the injury site are reprogrammed into a state of stemness are still a mystery. Analyzing RNA Revealed the Regeneration The researchers analyzed RNA to reconstruct the steps to regeneration. Do humans have similar genes?
Durham, North Carolina-based Ribometrix announced a strategic collaboration deal with Genentech , a Roche company, to identify and advance novel RNA-targeted small molecule therapeutics. Targeting RNA is believed to be a way to develop therapeutics for so-called undruggable proteins. The first identifies the 3D RNA motifs.
After some time in that role and launching several products, I received a call from Bill Banyai and Bill Peck, or ‘The Bills’ as we call them, who were building a company around technology that creates DNA by ‘writing’ it on a silicon chip. They had the science and the technology but not the business model. It was right after synbio 1.0
Instead, this is what is called a splicing mutation; it occurs at the boundary between coding DNA that is read into RNA and protein, and noncoding DNA that is excluded. These boundaries need to be “spliced” or specifically cut so RNA and proteins only contain coding DNA.
million DNA bases. The exons are nestled amongst the much longer introns, which are DNA sequences that are not represented in the final protein. It delivers a shortened dystrophin gene, just 4,558 DNA bases. Proteins called histones wrap around DNA at regular intervals, winding the molecule into units that resemble tiny spools.
And unlike traditional DNA sequencers, which parse genetic material by breaking it up into fragments and interpreting it chunk-by-chunk, a nanopore device unspools a long strand of DNA and reads it all at once. A scientist can isolate DNA and load up a flow cell in fifteen minutes. Nanopore devices work incredibly fast.
The studies are from groups at the Broad, Allen Institute for Brain Science, the Salk Institute for Biological Studies, and other institutions that are part of the National Institutes of Health’s Brain Research Through Advancing Innovative Neurotechnologies ® Initiative, or The BRAIN Initiative — Cell Census Network ( BICCN ). and Virginia W.
Mount Sinai researchers have developed a new model that uses DNA and RNA sequencing data from hundreds of patients to identify specific genes and genetic alterations responsible for never-before-defined subtypes of a blood cancer called multiple myeloma. doi: 10.1126/sciadv.abg9551. Source link: [link].
Everything started in school with an experiment on isolating DNA from bananas. Making a little tangle of DNA visible to the eye and understanding that this is the basis of complex organisms, which might be altered in disease, was the defining moment for my future path in life sciences.
2 AlphaFold 3 has been built to model DNA, RNA and smaller molecules (ligands). Google DeepMind’s new AI can model DNA, RNA, and ‘all life’s molecules’. His passion is to help early to mid-stage life sciences companies achieve novel biological breakthroughs through the effective use of computational modelling.
Reporting in Science , researchers at Massachusetts General Hospital and the Broad Institute of MIT and Harvard have made a surprising discovery about these drugs. IDH1 normally facilitates the activity of enzymes called demethylases, which remove chemical flags called methylation marks from DNA, allowing genes to be transcribed into RNA.
Related news #WhyIScience Q&A: How a global collaboration is boosting science and public health in Senegal Why did you and your colleagues in Senegal decide to do this study? Tags: Infectious Disease DNA sequencing RNA sequencing Tags: Infectious Disease DNA sequencing RNA sequencing Paper cited Levine, Z.C.,
20, 2020 /PRNewswire/ — Caris Life Sciences® , a leading innovator in molecular science focused on fulfilling the promise of precision medicine, announced today that Winship Cancer Institute of Emory University (Winship) has joined Caris’ Precision Oncology Alliance (The Alliance/POA). . IRVING, Texas and ATLANTA , Oct.
This method was more about serendipity than science. Another promising avenue is the use of technologies like RNA interference and gene editing, which allow scientists to turn off the production of certain proteins altogether. But as molecular biology has advanced, so too has our approach to finding new drugs.
Photo courtesy of Science Advances. Their research – the first of its kind – suggests that the system can be utilized to address cancer in animals, according to Professor Dan Peer, whose peer-reviewed research was published in the Science Advances journal. Cas-9 represents the Cas9 protein, an enzyme that cuts foreign DNA.
The researchers searched the DNA of humans and other primates for clues to how we lost our tails. They discovered that a bit of repetitious DNA, called an Alu sequence, inserted into a gene associated with tail development early in the ape lineage. ” The post How the Human Lost Its Tail appeared first on DNAScience.
One thread that has run throughout it has been my passion for science as a lens for looking at the world. In particular, I love connecting science to daily life. Pfizer’s recent campaign around “ Science Will Win ” similarly appealed to the ethos of the power of science to help humanity. Remember life’s Central Dogma.
As soon as I learned about DNA and RNA, I wanted to be a molecular biologist. Last stops at RNA My last roles in biotech were where my original passion began: DNA and RNA. My last stop at Arrakis Therapeutics is with a company targeting RNA with small molecules. Arrakis is the capstone of my career.
Our skilled protein science team can provide in-house production of proteins to support all these assay formats if needed. MST is notably effective for DNA binding proteins, where it outperforms flow-based systems in multi-component assay setups. Explore this page to learn about the technologies we use and their key benefits.
These results were corroborated by an RNA sequencing analysis we carried out in parallel, which revealed that depending on the temperature, RBM3 had different isoforms created by differential self-splicing. RBM3 is an RNA-binding protein, and we needed an RNA biologist, which is when Deepak joined the project.
We each have two DNA copies of the KIF1A gene; each copy is called an allele and makes its own RNA, which is then translated into protein. It is a type of gene-based therapy for KIF1A mutations that reduces RNA expression of the mutant allele, without interrupting healthy KIF1A expression. What is an allele specific ASO?
When choosing my bachelor’s degree, the choice was between pursuing a career in biology or in computer science. As my studies were in Lebanon, one of the challenges I faced was the low funding dedicated to fundamental sciences in my home country. Awareness work should be done in schools, getting more girls interested in sciences.
Her research grew out of a project helmed by Andersson, Jones, Jesse Engreitz, and their collaborators at the Novo Nordisk Foundation Center for Genomic Mechanisms of Disease (NNFC) at Broad: building computational models to predict which specific stretches of DNA called enhancers regulate which genesand in which cell types.
The steps taken during the analytical phase are dependent on the type of biomarker, which can range from DNA, RNA, protein, peptide, biomolecular modification(s), or biochemical pathway(s). Assay technologies are used during the process of analytical evaluation and validation of biomarkers.
She has played a key role inbuilding the target identification platform and a proprietary database of transcriptome-wide, functional RNA structures. Rising from Research Scientist to Senior Scientist, I supported early-stage DD efforts within their Protein Science group, based in Cambridge, UK.
We will present a fully automated end-to-end solution inclusive of automation and reagents optimized for miniaturized NGS that is guaranteed to generate high-quality libraries.
The BioNeMo service is domain-optimized for chemical, proteomic, and genomic applications, designed to support molecular data represented in the SMILES notation for chemical structures, and FASTA for amino acid and nucleic acid sequences for proteins, DNA, and RNA.
Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.
The long-acting regimen of cabotegravir and rilpivirine is an investigational regimen for the treatment of HIV-1 infection in adults to replace the current antiretroviral regimen in patients who are virologically stable and suppressed (HIV-1 RNA less than 50 copies/mL). For further information please visit www.gsk.com/about-us.
Can you provide examples of efforts within the life sciences sector aimed at overcoming operational bottlenecks in cell and gene therapy manufacturing using AI? We are pursuing and encouraging more data partnerships to enable the required openness.
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