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On the last day of February every year is Rare Disease Day , a dedicated day to celebrate the rare disease community, including patients, families, caregivers, clinicians, researchers, regulators, and more. For any collaboration inquiries or questions about the 2025 Rally for Rare event, reach out to rare@worldwide.com.
Scientists create next generation of tools in battle against brain disease By Corie Lok May 21, 2025 Breadcrumb Home Scientists create next generation of tools in battle against brain disease The findings contained in eight studies could lead to targeted gene therapies for brain disorders.
The tools developed function entirely post-translationally, enabling synergistic strategies with engineerable regulation across multiple levels of the central dogma. The tools developed function entirely post-translationally, enabling synergistic strategies with engineerable regulation across multiple levels of the central dogma.
Regulatory Guidance for Oligonucleotide Bioanalysis in Drug Development pmjackson Wed, 02/19/2025 - 21:30 The unique physicochemical properties of oligonucleotides require the use of specialized bioanalytical approaches, with key considerations including selectivity and specificity, sensitivity, stability, and matrix effects.
On April 10, 2025, the US FDA announced that it has a long-term plan to eliminate conventional animal testing in drug development, starting with monoclonal antibodies (mAbs).[ We remain ingrained in the regulations to ensure sponsors can optimize their programs and leverage the benefits that might be available to them via new initiatives.
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. Ten were capable of editing DNA in human cells.
Building bridges between Denmark-based and Broad researchers By Corie Lok March 27, 2025 Breadcrumb Home Building bridges between Denmark-based and Broad researchers The Novo Nordisk Foundation Center for Genomic Mechanisms of Disease is forging connections between Danish and Broad scientists. Here are two of their stories.
Plastic leaves do not emit organic signals and contain no DNA, so any mimicry could imply some kind of primitive vision. Such vascular differences are governed by auxin, a plant hormone that regulates leaf venation and shape. ” Asimov Press (2025). He is from Palo Alto and is currently based in Los Angeles.
By Broad Communications June 24, 2025 Credit: Casey Atkins David Liu is a core member at the Broad Institute where he is the Richard Merkin Professor and director of the Merkin Institute for Transformative Technologies in Healthcare. Some of these treatments, like K.J.’s,
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.” If the same virus invades the cell a second time, the gRNA’s spacer sequence will bind to the matching viral DNA sequence, then be cut by the Cas protein.
Since this discovery, it’s been unclear how regulators or sponsors might assess or address these potential risks. Clearly, the scope of the issue has grown substantially compared to what was covered in the initial guidances and AI recommendations provided by regulators in the last several years. “To ensure the safety of the U.S.
New technique expands cells to sequence DNA and capture fine structural details By Allessandra DiCorato May 29, 2025 Breadcrumb Home New technique expands cells to sequence DNA and capture fine structural details Using this method, scientists discovered key protein and gene activity changes in aging and in a rare disease called progeria.
If so, then here's a question: How much information is in your DNA? When I first looked into this question , I thought it was simple: Human DNA has about 3.1 Each base pair can take one of four values (A, T, C, or G) It takes 2 bits to encode one of four possible values (00, 01, 10, or 11) Thus, human DNA contains 6.2
By Allessandra DiCorato May 12, 2025 Credit: Allison Colorado, Broad Communications A fellow in Broad's Gene Regulation Observatory, Bo Xia studies how the structure of the genome determines cell fate in both health and disease. In school, he loved imagining how molecules made up materials and whole organisms.
The same model can also generate brand-new DNA sequences at the scale of yeast chromosomes or small bacterial genomes. AI Architectures Before explaining how Evo 2 could make bioengineering more predictable, it helps to first understand how AI models actually “learn” the language of DNA.
A Novo Nordisk - Broad collaboration is connecting Denmark-based and Broad researchers By Corie Lok March 27, 2025 Breadcrumb Home A Novo Nordisk - Broad collaboration is connecting Denmark-based and Broad researchers The Novo Nordisk Foundation Center for Genomic Mechanisms of Disease has built a bridge between Danish and Broad scientists.
We’re close to selling out of our second book —written in both text and DNA—and we recently launched a merchandise store. The reason for this shift is relatively simple: Chinese regulators have passed reforms that lower barriers to market entry and streamline approvals. ” Asimov Press (2025).
That’s astonishingly small, roughly equal to the weight of the DNA inside a single hummingbird cell. The other 30 percent — the so-called “dry mass” — is composed of everything else: DNA, RNA, proteins, lipids, and ions. Similar considerations apply for DNA, RNA, and all the other molecules, too.
What we expect the EPA to do in April 2024 The top line: In this returning feature, AgencyIQ looks at public data to determine what the EPA is planning to do in the month ahead, including deadlines, meetings, events, planned regulations, comment periods, and more. PFAS regulations for drinking water.
AI tool predicts potential drug targets by analyzing cell images By Corie Lok May 13, 2025 Breadcrumb Home AI tool predicts potential drug targets by analyzing cell images Researchers have developed a machine learning model that connects images of DNA structure to gene regulation. Online May 12, 2025. Cell Systems.
But with the rise of Chinas innovative sector, the proliferation of biologic drugs coded by DNA, and more than dozen years of abundant capital, to name just a few things – the challenge has only gotten worse. As illustrated in LEKs May 2025 report Is Biopharma Doing Enough to Advance Novel Targets ?
While reducing mutant huntingtin is desirable, the wild-type huntingtin protein plays crucial roles in normal neuronal function, including vesicular transport, synaptic function and transcriptional regulation. Accessed March 2025. Accessed March 2025. Huntington’s Disease Society of America. ” HDSA.org.
Random mutations in DNA bring about this genetic variation, and mating between unrelated individuals mixes the mutations amongst the population. Most vertebrates in the study, including the lab mouse, Mus musculus , acquire one mutation for every 100 million DNA bases per generation. percent of the mouse’s DNA.
Originally designed to regulate blood sugar in type 2 diabetes, GLP-1 receptor agonists have evolved into groundbreaking treatments for obesity, cardiovascular disease and even neurodegenerative conditions. By Ben Fidler • March 4, 2025 Permission granted by Daiichi Sankyo ASCO25 Daiichi Sankyo struck gold with ‘ADC’ cancer drugs.
Published June 5, 2025 Amy Baxter Staff Reporter post share post print email license FDA Commissioner Martin Makary attends a Senate Appropriations Committee hearing May 22, 2025. While it offered the first look at a possible framework for how pharmas using AI will be evaluated by regulators, many AI experts want more details.
The insulin released into the water is a near-perfect molecular match of the insulin produced by the fish to regulate their blood sugar, an example of convergent evolution, or the re-evolution of the same trait in different species. After entering the cell, CdtB acts like a DNA scissors and cuts the molecular threads.
Many students are taught that the Central Dogma is simply “DNA → RNA → protein.” In 1956, Crick was working on a lecture that would bring together what was then known about the “flow of information” between DNA, RNA, and protein in cells.
The connection between the destroyed lobe and Gage’s reduced capacity for emotional regulation was stark. Metagenomics, for example, allows scientists to build extensive reference libraries of DNA for use in everything from the creation of vital medicines to CRISPR tools. ” Asimov Press (2025). Cite: Balwit X.
Proteins and DNA usually unravel in such scalding environments, yet researchers who descended in submarines in 1982 found entire colonies thriving at these extreme temperatures. They also shield their genomes using an enzyme called reverse gyrase , which burns ATP energy molecules to wrap DNA in a heat-protective supercoil.
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