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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.
One company leading the charge in this revolution is Alltrna , whose pioneering work in engineered transfer RNA (tRNA) therapeutics is offering new hope for patients with rare genetic diseases. we are first focused on engineering tRNAs to restore protein production in diseases caused by premature termination codons.
The dynamic behaviour of RNA transcripts, the regulation and modification of proteins, the lipid composition of membranes, the structural organisation of cells and the spatial context of tissues all play critical roles in disease progression and therapeutic response.
Founding Vision: Harnessing RNA Editing for Transformative Medicine Korro Bio’s story began in 2018, co-founded by a remarkable group: Jean-François Formela, M.D. Academic specializing in RNA editing). The ability of modifying RNA enabled us to sidestep many of the potential risks associated with permanent DNA editing.
Certain CRISPR components can add short DNA sequences from the genomes of defeated viruses into the bacterium’s own genome, creating a type of protective “memory.” The core of the CRISPR immune response is a guide RNA (gRNA) that binds to a CRISPR-associated (Cas) protein. Visit our website to get a copy.
Some fluoroquinolones — a class of antibiotics that inhibits DNA replication — may even cause psychosis. Testing the engineered cells against four common drugs, I found that the M. By inserting the gene into a harmless, related species called M. The researchers were left with 40 drug candidates.
In the four years I’ve been working as a machine learning engineer at Dyno Therapeutics , a biotechnology company in Boston, I’ve become increasingly convinced that biologists have an almost masochistic tolerance for difficult and protracted work. Yet many disciplines have yet to benefit from such dramatic acceleration.
Scientists are already building a model that can, for example, look at which RNA molecules are expressed in a cell at t=0 and predict how those molecules will change at t=1. Synthesizing a single human protein-coding gene costs several hundred dollars and even a simple PCR machine (used for amplifying DNA) costs between $1,500 and $50,000.
There might be 100, 200, or 5,000 different kinds of parts in a steam engine or a jumbo jet. This makes mechanistic thinking different from just offering descriptions, and makes science different from engineering. For example, a gear is one of the entities you find in an engine. Even so, you know that the number is finite.
A team of researchers at the Broad Institute of MIT and Harvard has engineered virus-like particles to deliver prime editors to cells in mice at a high enough efficiency to rescue a genetic disorder. Prime editing, described in 2019 by Liu’s group, can make longer and more diverse types of DNA changes than other types of editing.
When faced with a viral threat, bacterial cells developed an immune response by capturing and copying DNA fragments of viruses. This allowed bacteria to recognise subsequent attacks and cleave the viral DNA to stop the viral infection. It was also discovered that the Cas enzyme was responsible for DNA cleavage.
By Leah Eisenstadt June 28, 2023 Credit: Courtesy of the Zhang lab Cryo-EM map of a Fanzor protein in complex with ωRNA and its target DNA. They showed that Fanzor proteins use RNA as a guide to target DNA precisely, and that Fanzors can be reprogrammed to edit the genome of human cells. said Zhang.
Now researchers at the Broad Institute of MIT and Harvard have used cutting-edge continuous laboratory evolution and engineering methods to develop improved versions of the gene-editing tool. Reverse transcriptase proteins that copy RNA templates into strands of DNA are found naturally in all plant and animal cells and in many viruses.
Cas9 ancestor engineered into a compact genome editing tool By Tom Ulrich May 14, 2025 Breadcrumb Home Cas9 ancestor engineered into a compact genome editing tool Researchers adapt a compact RNA-guided enzyme from bacteria for a variety of DNA editing tasks in human cells.
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 showed that two of these systems could make short edits in the DNA of human cells.
At Alkermes , our interdisciplinary team of protein engineers, immunologists, pharmacologists, and analytical scientists is investigating the biology of several immunomodulatory cytokines including IL-12 and IL-18 to develop novel versions of these molecules with the goal of harnessing their therapeutic potential.
The reasons for this are multifaceted, including concerns over the safety of directly altering DNA sequences and subsequent regulatory restrictions that have arisen as a result. The epigenome (meaning ‘above the genome’) is a system of reversible marks regulating how the DNA is read, translated and used. What is epigenetic editing?
iPSC-derived lymphocytes, eg, T cells and natural killer (NK) cells, engineered to express targeting molecules such as chimeric antigen receptors (CARs) have shown clinical promise to treat haematological malignancies. We believe this lays the groundwork for engineering these kinds of myeloid cells to potentially target any cancer antigen.
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.
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.
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. Because I was put into the highest tier, I was automatically mapped to the engineering track.
The other study was led by senior authors Xiao Wang , a Broad core institute member, a Merkin Institute Fellow, and an assistant professor of chemistry at MIT, and Jia Liu, an assistant professor of engineering at Harvard University. Related news Researchers map brain cell changes in Alzheimer’s disease Courtesy of the Chen and Macosko labs.
Spectrum’s self-contained saliva collection kit provides critical sample consistency while suspending and neutralizing viral RNA transcripts completely inactivating the live virus. Qualified commercial RNA extraction chemistries include Perkin Elmer, Thermo Fisher, Roche, and Qiagen. ABOUT SPECTRUM SOLUTIONS AND SPECTRUM DNA.
Learn more COVID-19 portals and datasets Broad data scientists and software engineers have developed several portals and datasets to support COVID-19-related research. Learn more Genome Analysis Toolkit (GATK) Industry standard for identifying SNPs and indels in germline DNA- and RNA-seq data.
"These findings reveal a completely unexpected and fascinating mechanism that explains the efficacy of the drug," Manguso, who is a principal investigator at MGH, a co-director of the Tumor Immunology Discovery Engine (TIDE) group, and a Cancer Program associate member at Broad, said.
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.
The company’s suite of market-leading molecular profiling offerings assesses DNA, RNA and proteins to reveal a molecular blueprint that helps physicians and cancer patients make more precise and personalized treatment decisions.
Transforming Genomics Research Next-generation sequencing (NGS) enables comprehensive genome sequencing and has transformed genomics research, offering vastly improved capabilities for analyzing DNA and RNA in a high-throughput and cost-effective manner. Let’s Connect.
My interest in STEM came from a natural curiosity into how things work, as well as from my parents and grandfather who did STEM studies (physics, biology, engineering). However, there’s still a notable underrepresentation in fields like computer and engineering occupations.
She has played a key role inbuilding the target identification platform and a proprietary database of transcriptome-wide, functional RNA structures. Harini joined Serna Bio in early 2021 and has been an integral part of the multidisciplinary teamworking on target ID platform development to drug discovery.
Several viral vectors are being used currently, in addition to non-viral vectors, such as oligonucleotides, naked DNA, and lipoplexes and polyplexes. Oligonucleotide-Based Techniques Most oligonucleotide therapies act through antisense mechanisms and are directed against various RNA species. Asia, and Europe.
Unlike gene editing, this “epigenetic” editing does not modify the underlying DNA sequence, but it should switch the gene off permanently, which means that this could be a one-time treatment. They used an engineered adeno-associated virus (AAV) that crosses the blood-brain barrier after intravenous administration.
Kamal Nahas highlights the biological limits on cell division speed and explores how we might engineer cells to grow faster. However, they plod along as they clone plasmids—the loops of DNA that biologists use to manipulate and study organisms—because propagating them relies, in part, on the pace at which cells grow and divide.
It also gave me a new appreciation for biology, and the incredible exactitude that one must have to engineer it — let alone engineer it successfully. of a Boeing 737 jet engine. If scientists wanted to engineer cells to divide faster, then, they should start with the ribosomes. Always free. Richly storied.
Scientists from Northwestern Medicine have demonstrated that RNA interference could have a crucial role in the onset and development of Alzheimer’s disease (AD). sRNAs Cellular functions rely on numerous protein-coding and noncoding RNAs and the RNA-binding proteins associated with them, which form ribonucleoprotein complexes (RNPs).
Tissue engineering techniques using induced pluripotent stem cells (iPSC), which are differentiated into cardiomyocytes, are being developed to create functional cardiac tissues from patient-derived cells. RNA-based therapies, including mRNA and RNA interference (RNAi), are being explored to modulate gene expression in heart cells.
This is Codon Digest, a weekly roundup of research papers, news articles, and industry highlights about engineered biology. But now, by studying DNA extracted from microbes in the blood of almost 10,000 healthy people, this paper shows that there is no such thing. Read Transcription factors bind to DNA and control gene expression.
In 1952, when the famous Hershey-Chase experiment indicated that DNA—and not protein—was the source of genetic material, just 285 papers mentioned E. 2 After five years of work, Lee published a paper that reported the first molecular tools used to grow, transform, and engineer V. natriegens. coli using calcium chloride.
in physics at Princeton University, he had a remarkable idea: What if it were possible to build a circuit out of DNA, rather than electronics, and use it to “program” a living cell? The RNA Exporter , which was developed by Felix Horns and others in my lab, is one example. In other words, they could “program biology.”
Experiments at the Wuhan facility were done at the lowest two of the four standard biosafety levels, which were established at the dawn of recombinant DNA research in the 1970s. The Likely Predecessor of SARS-CoV-2 – From my Past Blog Posts I wrote 100+ DNA Science blog posts during the pandemic about the virus.
Together, Haining, Manguso , and Yates went on to found the Tumor Immunotherapy Discovery Engine ( TIDE ) at the Broad Institute of MIT and Harvard in 2017 with support from Calico as a collaborating partner. These cells also express the Cas9 protein, which helps disable the genes targeted by the guide RNAs.
Scientists from the Massachusetts Institute of Technology (MIT) and the University of Massachusetts Medical School (UMass), US, have collaborated to create a novel type of nanoparticle that can deliver messenger RNA that encodes for beneficial proteins to the lungs.
What are the key findings of Circio’s in vivo proof-of-concept for its circVec circular RNA platform technology compared to conventional mRNA-based expression with DNA vectors? Circular RNA (circRNA) has two major advantages versus mRNA in a vector-expression context. DNA vectors in mouse models?
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