Broad Institute

OCTOBER 30, 2024

Researchers map communities of single cells in metastatic breast cancers By Corie Lok October 30, 2024 Breadcrumb Home Researchers map communities of single cells in metastatic breast cancers The team compared different single-cell and spatial profiling methods to explore the diversity of cells in metastatic breast cancer biopsies.

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Research RNA Medical Schools Treatment 127

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

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

BioPharma Drive: Drug Pricing

APRIL 22, 2024

billion, is the latest research collaboration involving small molecule drugs that modify RNA. The deal, which could be worth up to $1.8

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RNA Small Molecule Disease Research 69

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

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 RNA Treatment 98

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 114

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

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

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.

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RNA Research Therapies Disease 72

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

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

SCIENMAG: Medicine & Health

JULY 27, 2023

If translated […]

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RNA Research Hospitals Disease 69

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

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

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

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

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

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

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

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.

Clinical Research 52

Clinical Research Research RNA DNA 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 98

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

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

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

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.

Drugs 111

Drugs RNA DNA Disease 111

Broad Institute

JUNE 29, 2023

Now, researchers at MIT and the Broad Institute of MIT and Harvard have developed RIBOmap, a technique that lets them pinpoint and visualize the precise locations of thousands of proteins being produced within an intact tissue and even individual cells. By Sarah C.P. Williams June 29, 2023 Credit: Zeng H, Huang J, Ren J, et al.

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Protein Production RNA Regulations Science 105

Broad Institute

NOVEMBER 7, 2023

While RA therapies targeted to specific inflammatory pathways have emerged, only some patients’ symptoms improve with treatment, emphasizing the need for multiple treatment approaches tailored to different disease subtypes. They also found that patients’ CTAPs were dynamic and could change over time in response to treatment.

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Treatment RNA Hospitals Disease 96

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. In this week’s article, researchers in Brazil identified a KIF1A mutation in a cohort of patients with congenital ataxia.

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Science Medical Schools RNA Research 116

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 86

PLOS: DNA Science

JULY 13, 2023

Most studies on senescence are related to chronic inflammation, cancer, and age-related diseases. A Surprise Finding In a series of astounding experiments, researchers from the NHGRI and the University of Galway found that just a head amputated from a Hydractinia body can regenerate a new body! Within 3 days, a new head appears.

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RNA DNA Research Disease 98

Chemical Biology and Drug Design

JULY 27, 2023

cells induced by poly (I:C) were treated with or without PA, and then RNA-sequencing, GO and KEGG analysis were carried out to obtain the five differentially expressed genes (IL-1β, HMOX1, PTGS2, Abca1, and P65), which were verified by QRT-PCR and western blot. RNA-sequencing was applied to analyze the differentially expressed genes (DEGs).

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