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

SCIENMAG: Medicine & Health

AUGUST 14, 2023

A technique that enables scientists to record gene mutations and patterns of gene activity in individual cells has been extended to cover RNA splicing as well, in a study led by researchers at Weill Cornell Medicine, the New York Genome Center and the Princess Margaret Cancer Centre in Toronto.

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RNA Disease Research 83

Broad Institute

JANUARY 8, 2024

Williams January 8, 2024 Credit: Susanna Hamilton, Broad Communications Researchers have developed virus-like particles that can deliver gene-editing cargo to cells, including those in the mouse brain. Delivery dilemma Gene editing approaches promise to treat a range of diseases by precisely correcting genetic mutations that cause disease.

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Engineering Research Virus DNA 144

Broad Institute

SEPTEMBER 28, 2023

By Allessandra DiCorato September 28, 2023 Credit: Strittmatter Laboratory, Yale University Amyloid beta clusters (red) builds up among neurons (green) in a mouse model of Alzheimer’s disease. Neurons are thought to produce the plaque-forming protein called amyloid beta, and the researchers found evidence for this in their data.

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Disease Medical Schools Research Hospitals 144

Drug Target Review

JANUARY 24, 2024

Scientists from Northwestern Medicine have demonstrated that RNA interference could have a crucial role in the onset and development of Alzheimer’s disease (AD). Alzheimer’s disease Alzheimer’s is the most common cause of dementia. 4 Short RNAs (sRNAs) do not code for proteins.

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RNA Disease DNA Engineering 64

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.

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Therapies Disease Engineering Medical Schools 116

Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. Using humans as the model, we use an approach called deep phenotyping to explore the relationships between cells, genes, biological pathways and patterns of disease. Why are you focusing on liver disease?

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Therapies Disease RNA Engineering 102

Drug Target Review

AUGUST 22, 2023

This Q&A explores how pre-clinical research is being used to identify potential therapies for Huntington’s disease, a devastating condition that currently lacks disease- modifying treatments. What makes the potential therapeutic for Huntington’s disease unique compared to other treatments?

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Therapies Disease Treatment RNA 98

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 Pharmacokinetics Science 59

Drug Target Review

DECEMBER 6, 2024

In drug discovery and biological research, the scientists workflow often follows a structured and iterative approach to ensure accuracy, reproducibility and scientific integrity. At the heart of any research is the scientific question. This acts as a safeguard against false positives and misinterpretation of the data.

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RNA Drugs Research Science 52

Drug Target Review

JANUARY 2, 2024

In research published in Scientific Reports , 1 investigators focused on mesenchymal stem cells (MSCs), known for their potential in treating cell defects and regulating immune responses. We are studying the placement of organelles within cells and how they communicate to help better treat disease,” said Coskun.

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RNA Immune Response Therapies Disease 128

Broad Institute

JUNE 18, 2024

By Allessandra DiCorato June 18, 2024 Credit: Jon Arizti-Sanz SHINE, a rapid diagnostic test developed by Pardis Sabeti's lab in 2020, uses paper strips and CRISPR enzymes to identify specific sequences of viral RNA in samples. The researchers also adapted SHINE to distinguish between different flu strains.

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Virus RNA Laboratories Medical Schools 144

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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Research Disease Medical Schools RNA 111

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

OCTOBER 11, 2023

Manguso, who’d recently graduated from college and was conducting research at the University of Copenhagen as a Fulbright scholar, moved back to the Boston area to be with his mother as she underwent treatment. His mother had a presentation of the disease that suggested her immune system was already on the job.

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Research Immune Response Clinical Trials Therapies 137

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

SCIENMAG: Medicine & Health

JULY 27, 2023

If translated […]

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RNA Research Hospitals Disease 69

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. The study appears in Nature Biotechnology.

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RNA Medical Schools Engineering DNA 98

Broad Institute

NOVEMBER 23, 2023

By Allessandra DiCorato November 23, 2023 Credit: Jessica Hernandez, Broad Communications Related news Researchers uncover new CRISPR-like system in animals that can edit the human genome Microbial sequence databases contain a wealth of information about enzymes and other molecules that could be adapted for biotechnology.

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RNA DNA Engineering Science 135

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.

Therapies 137

Therapies FDA Approval Engineering Disease 137

Broad Institute

AUGUST 31, 2023

Evolved prime editors are smaller and more efficient for therapeutic applications By Corie Lok August 31, 2023 Breadcrumb Home Evolved prime editors are smaller and more efficient for therapeutic applications Researchers have evolved the editing machine at the heart of the prime editing system, which can make a wide range of changes to the genome.

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DNA RNA Engineering Laboratories 145

Broad Institute

OCTOBER 21, 2024

By Leah Eisenstadt October 21, 2024 Credit: Kevin Middleton, Broad Communications In the Bhattacharyya lab, researchers develop methods to quickly identify the most appropriate treatments for bacterial infections. The findings appear in the Journal of Clinical Microbiology.

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RNA Hospitals Laboratories Medical Schools 115

Broad Institute

AUGUST 31, 2023

By Leah Eisenstadt August 31, 2023 Credit: Susanna Hamilton, Broad Communications Researchers studied the effects of a schizophrenia gene mutation in different regions of the mouse brain. One of these is the GRIN2A gene, which encodes a subunit of a protein complex called the NMDA receptor.

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RNA Medical Schools Research Science 143

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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Clinical Research Research RNA DNA 52

The Open Targets Blog

JANUARY 30, 2025

gget consists of a collection of modules, each enabling researchers to perform common tasks in genomics, transcriptomics, and proteomics data analysis in just one line of code without exceeding the computational capabilities of a laptop.  Was there anything particularly difficult or unexpected in the process of creating gget?

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Bioinformatics Immune Response RNA Packaging 76

Codon

NOVEMBER 3, 2024

Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. Even after microbiologists discovered the bacterium that causes the illness in 1882, it wasn’t until the 1920s that researchers were able to develop a vaccine for TB.

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Vaccine Trials Therapies Treatment 96

Broad Institute

APRIL 4, 2024

A Practical guide to bulk RNA-seq, including guidelines for eQTL analysis By Rose Circeo April 4, 2024 Breadcrumb Home A Practical guide to bulk RNA-seq, including guidelines for eQTL analysis The Primer on Medical and Population Genetics is a series of weekly lectures on genetics topics related to human populations and disease.

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RNA Bioethics Disease Research 52

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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Engineering Protein Production RNA Vaccine 140

Dark Matter Blog

AUGUST 27, 2020

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.

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Small Molecule RNA Clinical Trials Drugs 52

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. I tracked the numbers of sequenced SARS-CoV-2 genomes posted at GISAID every few days during the pandemic, contributed by researchers everywhere.

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Disease DNA Virus RNA 95

Drug Target Review

JULY 5, 2023

In nature, proteins take millions and trillions of years to evolve, 1 but scientists do not have this time when designing drugs for life-threatening diseases. Therefore, researchers use directed protein evolution technologies , to evolve and design proteins with specific functions under well-defined conditions and a practical time frame.

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RNA Engineering Disease Research 97

Drug Target Review

JULY 11, 2023

In the quest to combat and eradicate diseases, scientists and researchers are increasingly turning to the immense potential of artificial intelligence (AI) as a tool. It was here that the Impact Medicine Fund was raised as a concept around wellness for those with disease, with the ambition of unlocking some of this vast potential.

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Disease RNA Small Molecule Clinical Development 75

Drug Target Review

JULY 24, 2023

Researchers from Tokyo Tech’s World Research Hub Initiative conducted a study to investigate the regulation of gene expression in the apicoplast of the malaria parasite Plasmodium falciparum. He has shared his excitement about the study’s implications and how it opens up new avenues in malaria research.

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RNA Regulations Bioinformatics Laboratories 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. In this week’s article, researchers in Shanghai, China identified 6 children with KIF1A mutations and compared their symptoms to cases in the literature.

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DNA RNA Disease Research 105

Codon

AUGUST 26, 2024

The Coalition for Epidemic Preparedness Innovations (CEPI)—a nonprofit organization that funds vaccine research and development—thinks so. Many research groups are now making broad-spectrum antivirals inspired by the innate immune system, leveraging recent advances in chemistry and biology. This work is already underway.

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RNA Virus DNA Vaccine 97

Drug Target Review

NOVEMBER 9, 2023

For example, neurons that innervate a person’s leg can be more than a metre long: they may have their nucleus near the spinal cord but sense a tickling sensation in the feet or pain in the big toe.” Cellular network Neurons have long protrusions, named axons, within which molecules like proteins, RNA, and organelles travel.

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RNA Disease Science Research 98