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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.
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.
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.
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.'
MONDAY, April 8, 2024 -- A blood test appears capable of detecting early-stage pancreatic cancers with up to 97% accuracy, a new study reports.The test looks for eight small RNA particles and eight larger DNA markers shed by pancreatic cancers,
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.
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.
3c10581 Helicases, classified into six superfamilies, are mechanoenzymes that utilize energy derived from ATP hydrolysis to remodel DNA and RNA substrates. Kapoor Journal of the American Chemical Society 2024 DOI: 10.1021/jacs.3c10581
Together, they have published dozens of papers on the mechanics of mitochondrial DNA and RNA in a single-celled, disease-causing parasite called Trypanosoma brucei. As molecular biologists at Boston University and as husband and wife, Ruslan Afasizhev and Inna Afasizheva, have worked together for decades.
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. What had not been done until now is improving the reverse transcriptase in a way that’s tailor-made for prime editing.”
We’ll cover the common terminology for the proteins, DNA, and RNA used in CRISPR. CRISPR is an elegant bacterial immune system and an efficient gene editing tool… but boy does it have a lot of parts! If you’re still a bit confused by CRISPR acronyms, this post is for you.
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.
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.
That was so in 1977, when “intervening DNA sequences” – aka “introns” – were discovered to interrupt protein-encoding genes. But some species control genetic responses another way – via RNA editing. Because the edits are in RNA, and not DNA, they are fleeting.
Tweaking of chemical compositions of natural nucleotide bases could be developed as novel building blocks of DNA/RNA. In the Hachimoji base pairing, synthetic building blocks are formed using eight modified nucleotide (DNA/RNA) letters (hence the name “Hachimoji”). The modified bases (dP, dZ, dS, and dB etc.)
Prime editing is a versatile genome editing technology that allows precise modifications of DNA (replacements, small insertions, and deletions) without introducing DNA double-strand breaks (Anzalone et al., 2019; Chen & Liu, 2023).
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.
That is in part because of the ongoing challenge of understanding the DNA switches, called cis-regulatory elements (CREs), that control the expression and repression of genes. CREs themselves are not part of genes, but are separate, regulatory DNA sequences – often located near the genes they control. In a paper published in Oct.
Plasmid DNA Design Considerations for Cell and Gene Therapy Cell and Gene therapy organizations must balance the need for rapid clinical progression and stringent quality expectations throughout development. In this whitepaper, Aldevron provides an in-depth look at the considerations of plasmid manufacturing used in commercial applications.
For both cancer and healthy genomes, we find that cysteine acquisition is a ubiquitous consequence of genetic variation that is further elevated in the context of decreased DNA repair.
Prior to 2015, I had a casual relationship, at best, with targeting RNA. Targeting RNA is a Whole New World Then in 2015, I became smitten and eloped with RNA, setting out to build a company devoted to bringing to bear industrial drug discovery concepts and methods on a new problem of drugging RNA with small molecules.
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.
Beyond Proteins: DNA and RNA Frontier The story doesn’t end with proteins. DNA and RNA are also key players, each bringing unique challenges. Many cancer treatments target DNA, but often these can cause collateral damage, leading to severe side effects.
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.
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.
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.
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?
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?
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. Researchers were able to use our synthetic RNA controls as a reference to verify and validate assays.
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.
The instructions to form a cell are encoded in DNA strands, wrapped in proteins and RNA, and wound together into a 3D structure called chromatin. Another finding was that regulatory T cells had distinct chromatin architecture features called DNA loops. This indicated other Foxp3 protein-containing complexes may be involved.
Researchers from the Laboratory of Bacteriology at The Rockefeller University have now found that bacteria sense phages by a defensive response named CBASS (cyclic oligonucleotide-based antiphage signalling system) which detects viral RNA. The novel, hairpin-shaped molecule was named cabRNA for CBASS-activating bacteriophage RNA.
In the ever-evolving landscape of drug discovery, the ability to extract and analyze DNA and RNA from clinical biospecimens has become a cornerstone of innovative research. It is critical for downstream applications such as RT-PCR, RNA sequencing, and Next Generation Sequencing (NGS).
These CRISPR guides are short sequences of RNA that steer the CRISPR Cas9 enzyme to a specific site in the genome to cut DNA and deactivate a targeted gene. The new findings, from scientists at the Broad Institute of MIT and Harvard, show about 2 percent of these guides miss their target.
An oligonucleotide is a short strand of nucleotides, the building blocks of DNA or RNA, used in genetic research and therapy. Our experts stay up to date on all new and evolving regulations to guarantee regulatory compliance in your studies.
The team analyzed the full transcriptome of cells from nearly 100 regions across the mouse brain using high-throughput single-nucleus RNA sequencing, the preferred approach for efforts to create a human brain atlas. Related news Researchers map brain cell changes in Alzheimer’s disease Courtesy of the Chen and Macosko labs.
In terms of targeted gene insertion, we presented data on a new gene-editing endonuclease called UltraSlice nickase, which is a novel protein-based gene editing system that is very different to CRISPR, as it does not involve a guide RNA. Those kinds of mutations are often associated with double-strand breaks in DNA.
These numbers reflect impressive developments in DNA sequencing technologies but in most cases represent the immediate costs of consumables on the sequencing instrument. 2 DNA sequencing is also reliant on sample processing and DNA library preparation before sequencing as well as bioinformatics and data analysis after sequencing.
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.
Gene therapy requires DNA and/or RNA delivery and analysis, and while most ongoing research involves therapies being delivered in vivo via adeno-associated viral (AAV) vectors, other in vivo delivery methods are on the rise. Multiple rounds of DNA synthesis will then take place to amplify that section.
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