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What started 28 years ago as a meeting for academic researchers has evolved into a dynamic, cross-disciplinary hub for the entire cell and gene therapy ecosystem. Over just a few short days, I connected with researchers, sponsors, and partners to talk shop: industry trends, regulatory shifts, and scientific advances.
This blog post is part of a series that will explore applications and expansions of the Open Targets informatics ecosystem, particularly the Open Targets Platform and Open Targets Genetics through conversations with our users. The gget opentargets module plays a key role in enabling researchers to interpret the identified variants.
In the research world, there is a myriad of tools this could be applied to. However, RNA interference (RNAi) remains steadfast in the background, a handy “oldie” as you might say. An oldie but a goodie — a phrase used to refer to something that has fallen out of fashion but is still useful and most importantly, effective.
Absorbance measurements, often referred to as light absorbance, are so versatile and widespread that they are one of the first techniques many life science researchers encounter in a practical setting. In this blog, we examine the utility of absorbance and optical density measurements, including examples where microplate readers can help.
This blog provides an overview of key biomarkers for five major neurodegenerative diseases: Alzheimers disease (AD), Parkinsons disease (PD), amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS), and Huntingtons disease (HD).
There's a sharp divide between "impressionistic" research that deals with simple predictions and abstract nouns (merely documenting effects and phenomena) and “mechanical research” that attempts to model what we observe in terms of rules and entities. Drunk Cats and Second-Order Effects Most video games are abstract.
SYN [link] According to the inventor’s research and understanding, at present, the synthesis of azvudine mainly includes the following methods according to different raw materials: 1. It acts as an RNA-dependent RNA polymerase (RdRp) inhibitor, the same mechanism as the previously approved molnupiravir and remdesivir.
However, since most small molecule drugs target proteins in the cell, the vast majority of target identification methods available to research scientists are aimed at identifying interactions between small molecules and proteins. First, given an RNA target, could we identify where on the RNA an rSM bound?
We are moving rapidly toward escape velocity in our RNA expedition at Arrakis. We want to mark this progress with some updates and perspectives in the Dark Matter blog. Moreover, there is an even vaster universe of biology contained in non-coding transcripts that can only be reached by directly targeting RNA.
Moving research from concept to market is like conducting a symphony — there are numerous players and stakeholders, each bringing their unique expertise to work in harmony and advance a project. BioDAOs fund and incubate projects during the initial stages of translational research, often through IP-NFTs, even before a company is established.
As soon as I learned about DNA and RNA, I wanted to be a molecular biologist. Growing up in Shrewsbury, Massachusetts, it was natural for me to attempt to get a summer job up the road at the Worcester Foundation for Biomedical Research, where the first oral contraceptive was created. I wanted to use molecular biology to create drugs.
Imaging Modalities Help Visualize and Confirm Findings An emerging method of studying viruses is by using high-content imaging solutions to visualize the activity of small interfering RNA (siRNA), also known as short interfering RNA or “silencing” RNA.
As an important step in this direction, NIH-funded researchers at the Morgridge Institute for Research and University of Wisconsin-Madison have produced neural tissue chips with many features of a developing human brain. The sequencing data reflect the genomic activity within the organoid following exposure to each of the 60 compounds.
In this blog, we examine how quality control of nucleic acids on microplates before sequencing can facilitate next-generation sequencing and look at some of the emerging next-generation sequencing solutions available to researchers. If RNA is being sequenced it is first converted to cDNA by reverse transcription.
However, in June I happened to attend the Gordon Research Conference on Chemical Biology and High-throughput Chemistry where I saw a session on small molecules and RNA. Making small-molecule drugs against RNA structures looked like a really cool problem – I want to do THAT! This is a thing? I did NOT get the email.
Research in mice has shown that RBM3 expression is protective against misfolded protein damage, suggesting the protein as an interesting target in the development of therapies for neurodegenerative conditions such as Alzheimer’s disease. What are the next steps in this research? Where did the idea for this project come from?
In a paper published today in Nature Communications , Open Targets researchers and collaborators used single-cell RNA sequencing to create a high resolution molecular map of immune cells in NSCLC tumours, to better understand the role of these cells in disease progression. This could then pave the way for subtype-specific therapies.
So when I was asked recently to share advice to a group of undergraduate students doing research at Penn State, I was drawn to the idea of connecting scientific principles to some life lessons. The central dogma of molecular biology is that information generally (with few exceptions) flows from DNA to RNA to Protein.
citation needed ] The pharmacokinetic properties of obeldesivir and improved was first published by Chinese researchers in May 2022. 2-NTP , an analog of ATP, inhibits the SARS-CoV-2 RNA-dependent RNA polymerase replication and transcription of viral RNA. The collected fractions were concentrated to give Intermediate 2.
Learn how Strateos’ automated synthetic biology modules supported rapid, iterative screening of RNA sequences in modular pbuE expression system and E. Owing to their modular organization, riboswitches are ideal for engineering de novo sensing elements for use in synthetic biology. coli extract-based cell-free protein expression assays.
You need to return from SLAS in San Diego with new tools to raise your scientific research… your “Out of the Office ROI.” Time away from the lab is limited and valuable.
We bid our head of research and employee #005, Jim Barsoum, au revoir , as he marches bravely into a well-earned retirement. Today, we reach a bittersweet transition at Arrakis.
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.
Open Targets actually has some research projects in this area. The Open Targets Platform uses quite a lot of bulk RNA expression data, to inform on target expression profiles and disease associations, and the team is currently investigating the best way to integrate single cell expression data as well.
A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. In fact, it was these early pharmaceutical successes that gave us the confidence that we would ultimately succeed in systematically drugging a wide range of RNA structures.
More than two decades ago, though, some researchers in Tsukuba, Japan figured out a way to get around this problem and watch ATP synthase as it spins. In 2008, researchers at UC Berkeley devised an ingenious solution. The protein is too small and moves too fast. But again, how do we know? It’s an astonishing experiment.
The virus SARS-CoV-2 emerged in Wuhan, home of a premiere research institute that focuses on viruses. A year before the initial outbreak, Wuhan researchers proposed nurturing a coronavirus with enhanced ability to attach onto, and enter, human cells. Her points: 1. Shortly thereafter, some workers became sick.
Most people, and in particular most investors we spoke to in those early days, thought we were nuts – that RNA lacked the structural and molecular complexity that medicinal chemistry exploits with such great effect for proteins. One is that molecular recognition is purely a matter of physics and RNA has to play by the same rules.
This is Codon Digest, a weekly roundup of research papers, news articles, and industry highlights about engineered biology. Nucleic Acids Research. A giant dataset about the rat brain, and how neurons connect to each other, was collected and pooled together from 20 different research laboratories. Nucleic Acids Research.
For a recent study, researchers edited 21 different genes in poplar trees, a species commonly used to make paper. The researchers studied trees with 70,000 different gene-editing combinations, just 0.5% Because it turns out that at least half of them , in humans, also bind to RNA molecules. Max Berry left an excellent comment.
For a recent study, researchers edited 21 different genes in poplar trees, a species commonly used to make paper. The researchers studied trees with 70,000 different gene-editing combinations, just 0.5% Because it turns out that at least half of them , in humans, also bind to RNA molecules. Max Berry left an excellent comment.
The researchers began by taking beta cells from people with early-stage diabetes and from people without diabetes. To ensure that this problem stemmed from beta cells themselves and not the pancreas as a whole, the researchers transplanted the diabetic beta cells and the healthy beta cells into healthy mice, and – voila!
Research in gene therapies and genetically engineered drugs and vaccines are growing exponentially, and will only continue to become more popular. This was just one of many questions we explore in our comprehensive survey of research sites and their internal activation practices.
This study sheds light on how, exactly, AI-generated text differs from the real stuff, and how it could be used to (soon) write entire research papers. Read Ribosomes are able to build proteins because they contain a strand of RNA that imbues them with their catalytic power. Nucleic Acids Research. Nucleic Acids Research.
This study sheds light on how, exactly, AI-generated text differs from the real stuff, and how it could be used to (soon) write entire research papers. Read Ribosomes are able to build proteins because they contain a strand of RNA that imbues them with their catalytic power. Nucleic Acids Research. Nucleic Acids Research.
Goodsell, Scripps Research and RCSB Protein Data Bank. I pulled them from a 2011 blog post ( oH mY goD tHat’S nOt pEEr-ReVieWeD!!! ” A blog post for the American Society for Microbiology : Early studies were promising, though experiments were often improperly designed by today's standards (i.e., Source 3 of 30.
Goodsell, Scripps Research and RCSB Protein Data Bank. I pulled them from a 2011 blog post ( oH mY goD tHat’S nOt pEEr-ReVieWeD!!! ” A blog post for the American Society for Microbiology : Early studies were promising, though experiments were often improperly designed by today's standards (i.e., Source 3 of 30.
Since the World Health Organization (WHO) declared the novel coronavirus (COVID-19) outbreak a global pandemic on March 11, 2020, researchers around the world have been working tirelessly to discover effective preventive treatments and therapeutic agents. mg/mL to 1 mg/mL) compared with a positive control (antibody inhibitor AC384).
This is Codon Digest, my weekly roundup of research, news, and industry highlights about the future of biology. Small snippets of double-stranded RNA were sprayed onto hot pepper plants to control a pest, called Frankliniella occidentalis. The RNA strands shut down a specific gene in the insects, and killed half after 7 days.
Emmanouil Metzakopian is a group leader at the UK Dementia Research Institute at the University of Cambridge, as well as VP of Research and Development at bit.bio. We then designed our own CRISPR guide RNA library against the druggable genome and transduced the corresponding lentiviruses into millions of our iNeurons.
Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. Nucleic Acids Research. coli and yeast), signed an agreement with the nonprofit, ATCC , to help get their microbes to researchers. Stahl et al.
Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. Nucleic Acids Research. coli and yeast), signed an agreement with the nonprofit, ATCC , to help get their microbes to researchers. Stahl et al.
The speed at which researchers developed effective vaccines to prevent the spread of COVID-19 required unprecedented international cooperation on a scale never seen before. Meanwhile, researchers are continuing to investigate possible treatments for patients suffering with the disease. For research use only. Kirschner, L.,
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