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By adding synergistic self-adjuvanting properties to Covid-19 RNA vaccines, researchers showed they could significantly boost the immune response generated in mice.
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.
Researchers applied a new technology to generate the full inventory of mutations in the bacterial species Escherichia coli where the antibiotic rifampicin attaches to and disables an essential bacterial enzyme known as RNA polymerase (RNAP).
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. They also delivered prime editors to the mouse brain, and did not detect any off-target editing.
To speed up the annotation step, the Broad Institute’s Data Sciences Platform (DSP) has developed a new search engine that automates much of this process by using machine learning to search data on more than 50 million annotated single cells. It’s basically a search engine. How did you build the search engine? How does CAS work?
Using a synthetic guide RNA (gRNA), scientists can target a specific DNA sequence and employ Cas9 to cut it. Today, however, we will explore a different application of CRISPR: microbiome engineering… 1 Over the last decade, CRISPR-Cas9 has proven immensely valuable in drug discovery and drug manufacturing.
The Access to Advanced Health Institute Receives $18 Million Award to Develop a Temperature Stable, Single-Dose Chikungunya RNA Vaccine Through a Phase 1 Clinical Trial Credit: Delaney Brown Photography The Access to Advanced Health Institute Receives $18 Million Award to Develop a Temperature Stable, Single-Dose Chikungunya RNA Vaccine Through a Phase (..)
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. The new study demonstrates that RNA-guided DNA-cutting mechanisms are present across all kingdoms of life. said Zhang.
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.
Have you ever designed a CRISPR guide RNA and wondered why it is limited to only 20 bases, or why it’s so important to choose a target sequence with a nearby protospacer-adjacent motif (PAM) ?
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.
Forging the toolkit for subcellular omics Cellular components known as organelles, consisting of RNA and protein, exert vital influences on human wellbeing and ailments by sustaining equilibrium, managing growth and ageing, and producing energy.
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.
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. The new systems could potentially be harnessed to edit mammalian cells with fewer off-target effects than current Cas9 systems.
Streamlining the Path from Discovery to Patient – Simpler RNA Manufacturing with Aldevron’s Codex® HiCap RNA Polymerase swheeler Mon, 07/01/2024 - 17:17 Wed, 08/07/2024 - 10:00 Resource Type Webinar Kyle Studey, M.S. Codex ® is a trademark of Codexis, Inc. Listing Image aldevron 250x190.png
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.
At PEGS Europe, Drug Target Review spoke to Victoria Goldenshtein, a PhD candidate in Biomedical Engineering at Duke University, US, who presented an engaging poster on her lab’s novel in vitro library display platform for directed protein evolution termed GRIP Display, which entails Gluing RNA to Its Proteins.
By Allessandra DiCorato May 16, 2024 Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor. This engineered capsid opens up a world of possibilities."
“Sam has an exceptional talent in software engineering, and his contributions reflect a deep understanding of both the technical and biological aspects required for bioinformatics tool development,” says Laura Luebbert, now a postdoctoral fellow in the Sabeti lab at the Broad Institute of MIT and Harvard and Harvard University.
Senior Director, R&D Strategy, will be delivering a presentation highlighting how Strateos' smart lab platform accelerated riboswitch engineering to develop novel biosensors. Owing to their modular organization, riboswitches are ideal for engineering de novo sensing elements for use in synthetic biology.
This level of control is not achievable with gene editing and is a severe limitation in applications such as T-cell engineering, where precise epigenetic remodelling during differentiation 3 fundamentally shapes the phenotype and function of the cell. RNA-guided transcriptional silencing in vivo with S. Molecular Therapy.
Although this ancestral replicase appears to have been lost, key aspects of RNA-catalyzed RNA replication can be studied by proxy with the use of modern RNA enzymes (ribozymes) generated by in vitro evolution. However, none of these RPRs was capable of self-replication.
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.
In this regard, BioDAOs can be viewed as collaborative research translation engines and a novel form of biotech organization that develops IP through an online-native approach. By fostering a community of stakeholders with aligned incentives and diverse backgrounds, BioDAOs facilitate the translation of technologies.
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.
On the other hand, if you have a platform designed to be an engine for a standalone enterprise, capable of generating dozens of future drugs, then you might plot a course that brings you into the close orbit of multiple complementary partners. We all share a common understanding of the benefits and challenges of small molecule therapeutics.
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.
In the paper, researchers from Johns Hopkins University and elsewhere found a natural long-form transactivating CRISPR RNA (tracr-L) in Streptococcus pyogenes that functions to downregulate its endogenous CRISPR-Cas9 system. But altering the tracr-L with genetic engineering to make it function more like a guide RNA increased CRISPR-Cas9 cuts.
Their solution was to fuse hairpins, little loops made from RNA or DNA, at various positions along the DNA strand that was being sequenced. 3 Nanopore engineers decided to cleverly exploit the natural speed limit of DNA polymerase to slow down DNA translocation through the pores. 3 This speed varies depending on the organism.
For example, innate immune peptides being imitated by SARS-CoV-2 fragments have a special ability to organise double stranded RNA into structures that strongly amplify activation of the human innate immune system in autoimmune diseases. He currently serves on the Editorial Board of Physical Review E.
This laid the groundwork for my career in drug discovery using novel modalities like small molecule inhibitors, antibodies, RNA interference (RNAi) and, currently, small molecule protein degraders – which are opportunities to change how we think about medicines.
The company’s product candidates consist of novel human cells engineered to produce the crucial proteins, enzymes or factors needed by patients living with chronic diseases such as hemophilia, diabetes and lysosomal storage disorders. Laying down a new track for RNA processing, Remix launched with $81 million in financing.
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.
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. We have built a global, full-service CRO powered by intuitive end-to-end technology.
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.
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.
The CureVac-GSK COVID-19 collaboration announced in February 2021 extends the existing strategic mRNA technology partnership both companies started in July 2020, which focuses on the development of new products based on CureVac’s second-generation RNA-technology for different targets in the field of infectious diseases.
Because I was put into the highest tier, I was automatically mapped to the engineering track. Researchers were able to use our synthetic RNA controls as a reference to verify and validate assays. I was very good at math, which at the time in France directed me towards STEM studies.
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.
In the top row, human lung cancer cells were engineered to over-express GRP78 in the nucleus. Researchers from the Keck School of Medicine of USC used imaging techniques to study how the protein GRP78 controls cancer cell behavior. In the bottom row, cells lacked GRP78 in the nucleus.
Anderson Professor of Chemical and Biomolecular Engineering, and his colleagues, are reporting in iScience the event of an intranasal subunit vaccine that gives durable local immunity against inhaled pathogens. But now, we’re one step closer. Navin Varadarajan, University of Houston M.D.
As research developments into RNA vaccines help scientists accelerate drug candidates to arm the immune system against coronavirus, Pharma IQ ’s Keeping tabs on Covid-19 update returns with news from some of the biotechnology innovators leading the fight against the global pandemic.
She is currently the CEO of Twentyeight-Seven, a biotech company focused on novel RNA biology that she co-founded alongside prominent Harvard investigators. Enthera’s discovery engine and assets are protected by a broad portfolio of patents. The Company is backed by Sofinnova Partners and JDRF T1D fund. Connect with us on LinkedIn.
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