Broad Institute

MARCH 12, 2025

By Leah Eisenstadt March 12, 2025 Credit: Broad Communications Scientists in the Spatial Technology Platform at the Broad develop, use, and share spatial -omics approaches, such as Perturb-FISH, that can reveal the roles of genes in various cell types and the impact of intercellular interactions on health and disease.

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RNA Science Research Disease 99

Broad Institute

JUNE 27, 2024

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain.

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Disease Therapies Engineering Clinical Trials 142

Science Daily: Pharmacology News

JULY 27, 2023

In a step forward in the development of genetic medicines, researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.

RNA 177

RNA Research Therapies Disease 177

Drug Target Review

AUGUST 28, 2024

What are the main challenges currently faced in the treatment of chronic liver diseases, and how does Resolution Therapeutics aim to address these challenges? Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years.

Therapies 116

Therapies Disease Engineering Medical Schools 116

Elrig

MARCH 25, 2025

This second ELRIG meeting on Therapeutic Oligonucleotides brings together esteemed scientists from academia, industry, and other members of the drug discovery community to explore the discovery, validation, and targeting of oligonucleotide-based drug candidates, including antisense oligonucleotides (ASOs) and small interfering RNA (siRNA).

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RNA Bioinformatics Science Pharmacokinetics 59

Drug Target Review

DECEMBER 6, 2024

This includes verifying the experimental design and understanding how the data was generated whether it was from RNA sequencing, mass spectrometry, or other biological assays. About the authors Dr Raminderpal Singh Dr Raminderpal Singh is a recognised visionary in the implementation of AI across technology and science-focused industries.

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RNA Drugs Research Science 52

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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RNA DNA Vaccine Drug Development 52

Broad Institute

JANUARY 8, 2024

Now the researchers describe how they re-engineered both eVLPs and parts of the prime editing protein and RNA machinery to boost editing efficiency up to 170 times in human cells compared to the previous eVLPs that deliver base editors. By Sarah C.P.

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Engineering Research Virus DNA 144

Broad Institute

DECEMBER 6, 2023

Cytokines are also targeted by drugs for many diseases such as rheumatoid arthritis, COVID-19, and cancer, but until now, scientists haven’t had a comprehensive view of how different immune cells respond to different cytokines because the immune system is so complex. For many immune-mediated diseases, there's no cure or treatment.

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Immune Response RNA Disease Medical Schools 138

Broad Institute

NOVEMBER 23, 2023

The work appears today in Science. 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. They studied several of the new systems in greater detail in the lab.

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RNA DNA Engineering Science 135

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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RNA Small Molecule DNA Licensing 52

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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DNA RNA Disease Hospitals 128

Broad Institute

MAY 16, 2024

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.

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Therapies FDA Approval Engineering Disease 137

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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Virus DNA RNA Science 98

PLOS: DNA Science

JULY 27, 2023

That information led, thanks to vaccine shelved from the first SARS circa 2003, to the rapid development and deployment of mRNA vaccines against the new infectious disease. Seventeen of the entire group (10.7%) had “unanticipated monogenic disease risks (uMDRs).”

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Disease DNA Virus RNA 95

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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RNA Medical Schools Engineering Disease 98

Broad Institute

JUNE 29, 2023

The method, described today in Science , provides a way for scientists to learn new details on how translation is regulated within individual cell types and how that changes in disease. By Sarah C.P. Williams June 29, 2023 Credit: Zeng H, Huang J, Ren J, et al. Paper cited Zeng, H. Online June 29, 2023. DOI: 10.1126/science.add3067

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Protein Production RNA Regulations Science 105

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. Biotechnology program at Columbia University.

Science 116

Science Medical Schools Research RNA 116

Drug Target Review

JULY 24, 2023

The results were published in Proceedings of National Academy of Sciences of United States of America (PNAS). Through phylogenetic analysis and microscopy techniques, they identified a nuclear-encoded apicoplast RNA polymerase σ subunit called ApSigma. The first identification was based on a bioinformatic analysis.

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RNA Regulations Bioinformatics Laboratories 98

Codon

JANUARY 24, 2024

This is especially true in medicine, where the temporal gap between the initiation of the disease (a random genetic mutation, say) and the appearance of symptoms makes finding the root cause difficult. A capillary shrinks over time – it’s a temporal process – but science is often limited to static snapshots.

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Science Disease Research RNA 72

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. and Virginia W.

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Engineering Protein Production RNA Vaccine 140

Broad Institute

AUGUST 31, 2023

Related News Next generation prime editing systems move closer to possible therapeutic applications New CRISPR genome editing system offers a wide range of versatility in human cells Prime editing technologies allow scientists to precisely edit the genome in a variety of ways and could one day be used to treat genetic diseases.

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DNA RNA Engineering Laboratories 145

Broad Institute

JUNE 28, 2023

Today, scientists use these reactions to produce customizable DNA and RNA molecules that enable genetic sequencing, drug and vaccine development, pathogen tests, cancer diagnostics, and many aspects of basic biomedical research. “I Longer synthetic DNA and RNA molecules are also critical for modern biologic drugs.

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DNA RNA Science Therapies 98

Drug Target Review

NOVEMBER 9, 2023

A team of neurobiologists at The Molecular Neuroscience Unit at the Okinawa Institute of Science and Technology (OIST) has made a significant breakthrough by connecting sensory neurons’ survival and pathology with the way messenger RNAs (mRNAs) are transported inside these cells. References 1 Alcaraz G, Campanac E, Carlier E, et al.

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RNA Disease Science Research 98

Broad Institute

AUGUST 19, 2024

I’m so proud of this team and the partnership we formed to follow the science and set up some major positive impacts on women’s health,” Blainey said. Using RNA sequencing and working with the Broad’s Metabolomics Platform and collaborators at St. Paper cited Zhu M et al.

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Hospitals Treatment Medical Schools Science 121

Broad Institute

AUGUST 31, 2023

We were surprised to see that just missing one copy of this gene can induce many changes at the RNA level, at the protein level, at the functional level, and at the behavioral level,” said Farsi. Tags: Stanley Center for Psychiatric Research Psychiatric Disease Paper(s) cited: Farsi Z, et al. Aug 31, 2023. DOI: 10.1016/j.neuron.2023.08.004.

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RNA Medical Schools Research Science 143

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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Drugs RNA DNA Disease 111

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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Small Molecule RNA DNA Pharmaceutical Companies 96

Drug Target Review

JULY 23, 2024

I studied Natural Sciences at the University of Cambridge, where I was exposed to a range of disciplines from chemistry to physiology, specialising in my last year in biochemistry. Shortly after finishing my studies, I landed my first job in industry working on cell biology research for several disease indications.

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Small Molecule Targeted Protein Degradation Science Disease 116

Broad Institute

JANUARY 30, 2024

More bottles on the medicine shelf would help, but Bhattacharyya, who’s now also a research scientist, realized that science might lead to better tools for diagnosing and treating such challenging cases. FROM STARS TO CELLS Bhattacharyya’s interest in science began not in the lab, but in the sky.

Hospitals 137

Hospitals Virus Medical Schools Science 137

Broad Institute

OCTOBER 28, 2024

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. Tags: Data Sciences Platform Machine Learning Single Cell Is CAS now available to use?

Engineering 119

Engineering Cell Biology Disease RNA 119

PLOS: DNA Science

JULY 13, 2023

Most studies on senescence are related to chronic inflammation, cancer, and age-related diseases. Analyzing RNA Revealed the Regeneration The researchers analyzed RNA to reconstruct the steps to regeneration. The researchers discovered RNA transcripts known to be involved in senescence in the disembodied heads.

RNA 98

RNA DNA Research Disease 98

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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DNA RNA Disease Research 105

The Pharma Data

SEPTEMBER 28, 2020

This included compiling RNA data, as well as other data points such as patient demographics, clinical treatment arm and patient diagnosis. For years, patients diagnosed with a disease often received the same treatment. And for some people, that treatment worked.

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Science Clinical Trials RNA Treatment 52

KIF1A

JULY 29, 2023

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. KIF1A in New York: The 2023 KAND Family & Scientific Engagement Conference We won’t be posting next Saturday, but not for a lack of science!

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RNA Clinical Trials Disease Science 98

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. Related news #WhyIScience Q&A: How a global collaboration is boosting science and public health in Senegal Why did you and your colleagues in Senegal decide to do this study?

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Research Disease Medical Schools RNA 111

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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RNA Regulations Disease DNA 98