Broad Institute

OCTOBER 23, 2024

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.

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Broad Institute

NOVEMBER 23, 2023

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.

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Drug Target Review

DECEMBER 6, 2023

Dear readers, RNA, or ribonucleic acid, plays a pivotal role in the intricate dance of cellular processes. We are committed to providing you with the highest quality content, and we believe that this report will deliver a different perspective on the topics covered.

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Drug Target Review

JUNE 28, 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.

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KIF1A

JANUARY 20, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. 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.

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PLOS: DNA Science

MAY 16, 2024

These factors are converging to enable both identification of novel infectious diseases as well as microbial resistance, before these threats can impact public health, write a team from the European Society for Clinical Microbiology and Infectious Diseases in Frontiers in Science. COVID clearly caught us off guard.

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Drug Target Review

APRIL 26, 2023

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.

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The Pharma Data

JANUARY 5, 2021

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.

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Drug Target Review

JULY 7, 2023

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?

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PLOS: DNA Science

JULY 13, 2023

Most studies on senescence are related to chronic inflammation, cancer, and age-related diseases. 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. Do humans have similar genes?

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Broad Institute

MARCH 22, 2024

Messenger RNAs with multiple “tails” could lead to more effective therapeutics By Corie Lok March 22, 2024 Breadcrumb Home Messenger RNAs with multiple “tails” could lead to more effective therapeutics Scientists have engineered long lasting mRNAs that increased therapeutic protein production in cells and animals.

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PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. million DNA bases. The exons are nestled amongst the much longer introns, which are DNA sequences that are not represented in the final protein. The gene presents a complex landscape.

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Codon

AUGUST 26, 2024

Credit: OpenStax College The innate immune system has been evolutionarily successful for three reasons: its tools target the most fundamental properties of viruses , act as prophylactics —meaning they prevent diseases, rather than treat them—and are widespread. Existing medicines only pose a partial solution.

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Drug Target Review

SEPTEMBER 28, 2023

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. They needed reference materials for the disease in order to develop and validate diagnostic tests.

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Drug Target Review

SEPTEMBER 4, 2023

There have been several studies that have reported the ability of infectious disease agents to be drug resistant. Infectious agent includes bacteria, virus, fungi, and parasites, and they are capable of causing disease in a living organism. They could be spread by physical contact, airborne, waterborne, foodborne, or vector-borne.

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Drug Target Review

NOVEMBER 13, 2023

Regulatory T cells (Treg) cells are specialised immune cells that are essential for maintaining peripheral tolerance, preventing autoimmune diseases, and limiting chronic inflammatory diseases. Another finding was that regulatory T cells had distinct chromatin architecture features called DNA loops.

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Codon

AUGUST 27, 2024

Allison Berke makes the case for real-time DNA sequencing and AI tools to detect pathogens before they spread widely. Doctors in training are told that when they hear hoofbeats, they should think horses, not zebras; rare diseases are the exception, not the rule. After copying the DNA to form a big pool, each piece is sequenced.

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Drug Target Review

JULY 24, 2023

Through phylogenetic analysis and microscopy techniques, they identified a nuclear-encoded apicoplast RNA polymerase σ subunit called ApSigma. The study suggests that this regulatory system could be a potential target for future malaria treatments, offering hope for combatting this deadly disease that affects millions worldwide.

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Broad Institute

OCTOBER 4, 2023

Related links Merkin Prize Inaugural Merkin Prize in Biomedical Technology awarded to Dr. Marvin Caruthers for developing technology that efficiently synthesizes DNA The inaugural Richard N. Caruthers was announced as the winner in June for his development, in 1981, of an efficient, automated technology for synthesizing DNA.

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Broad Institute

DECEMBER 13, 2023

Related news Researchers map brain cell changes in Alzheimer’s disease Courtesy of the Chen and Macosko labs. The researchers also discovered clues about cellular function and the potential roles of brain structures in disease. Courtesy of the Chen and Macosko labs. Courtesy of the Chen and Macosko labs. million mapped cells.

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PLOS: DNA Science

APRIL 20, 2023

DNA is a sleek double helix, with “rungs” consisting of a purine base paired with a smaller pyrimidine base: adenine (A) with thymine (T) and guanine (G) with cytosine (C). . ” DNA encodes amino acid sequences comprising proteins, which impart traits. Watson and F.

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New Drug Approvals

SEPTEMBER 23, 2024

Target: DNA/RNA Synthesis Pathway: Cell Cycle/DNA Damage Huntington’s disease (HD) is a progressive, autosomal dominant neurodegenerative disorder of the brain, having symptoms characterized by involuntary movements, cognitive impairment, and mental deterioration. SCHEME PATENT PTC Therapeutics Inc., Audiol Res.

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PLOS: DNA Science

MARCH 14, 2024

Biotechnology has solved many problems, from recombinant DNA and monoclonal antibody-derived drugs, to gene therapy and stem cell transplants, to RNA-based vaccines and genetically modified plants that resist diseases and pesticides. Let Them Eat Snake appeared first on DNA Science. The post Cultivated Meat?

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Drug Target Review

JULY 5, 2023

Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. Please summarise Eterna Therapeutics’ ($ERNA) new research at ASGCT on a cell therapy approach that integrates multiple cell types to emulate the natural immune system’s fight against diseases?

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Codon

DECEMBER 12, 2024

DNA and RNA molecules are also built from exclusively right-handed nucleic acids. Across the tree of life, organisms strictly require exactly one of the two chiral forms of their molecular building blocks — amino acids, nucleotides of RNA and DNA. 4 As far as we know, right-handed proteins never occur naturally.

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Reprocell

AUGUST 8, 2024

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. As we delve deeper into the molecular mechanisms of diseases, the quality of nucleic acid extraction plays a critical role in unlocking new therapeutic avenues.

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Drug Target Review

FEBRUARY 6, 2024

Prostate cancers detected at the local stages, when the disease is limited to the prostate and its immediately surrounding regions, have a nearly 100 percent five-year survival rate. Advances in DNA and RNA-focused molecular diagnostic methods have made blood-based multi-cancer early detection (MCED) tests a reality, but not for all patients.

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Broad Institute

AUGUST 14, 2023

More recently, they are focusing on proteins to understand the biological events in cancer cells that lead to disease. With this approach, the team uncovered patterns in protein modifications linked to cancer-related processes such as DNA repair. They then characterized 33 molecular signatures to group biologically similar tumors.

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Broad Institute

MARCH 8, 2024

By Claire Hendershot March 8, 2024 Credit: National Institute of Allergy and Infectious Diseases, National Institutes of Health via U.S. There are a lot of patients that they couldn’t treat unless it was malaria or a small host of other diseases. The bacteria causes infections with malaria-like symptoms.

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Drug Target Review

AUGUST 9, 2023

Published on bit.bio’s website, this article authored by Dr Tony Oosterveen dives into the close relationship between FTD and ALS with a focus on mechanisms underlying the neuropathology of both diseases. ALS is the most common motor neuron disease and is estimated to affect more than 80,000 people worldwide.

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KIF1A

MAY 4, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. By talking to groups with these mutations, we may identify other diseases whose patients have KIF1A variants. She graduated from UC San Diego with a B.S.

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DrugBank

MAY 22, 2024

These tough nuts to crack in medical science—biological targets known to play roles in diseases but resistant to traditional drug design—are now seeing new strategies that shift the paradigm from "undruggable" to "druggable." Beyond Proteins: DNA and RNA Frontier The story doesn’t end with proteins.

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Codon

JUNE 11, 2024

Biologists use nanopores for everything from diagnosing diseases and monitoring changes within rainforest ecosystems to discovering proteins from microbes frozen in Icelandic glaciers. A scientist can isolate DNA and load up a flow cell in fifteen minutes. Nanopore devices work incredibly fast. An Oxford Nanopore device.

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Broad Institute

FEBRUARY 11, 2025

Mapping mRNA through its life cycle within a cell By Corie Lok February 11, 2025 Breadcrumb Home Mapping mRNA through its life cycle within a cell Xiao Wangs studies of how and where RNA is translated could lead to the development of better RNA therapeutics and vaccines. I wasnt trained at MIT, and I had never lived in Boston before.

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PLOS: DNA Science

MAY 25, 2023

Myotonic dystrophy type 1 (DM1), an inherited disease affecting muscles, was one of the first described “expanding repeat” disorders. In these 50 or so conditions, symptoms may appear earlier and worsen from generation to generation, as the mutant gene grows, adding copies of a 3- or 4-base DNA sequence.

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Drug Target Review

OCTOBER 2, 2024

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. This approach can be highly targeted, with the potential to correct mutations that lead to specific diseases or deliver therapeutic genes to improve heart function.

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The Pharma Data

NOVEMBER 17, 2021

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.

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