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.

RNA 83

RNA Disease Research 83

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.

RNA 99

RNA Science Research Disease 99

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.

Engineering 144

Engineering Research Virus DNA 144

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.

RNA 52

RNA Drugs Research Science 52

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).

RNA 59

RNA Bioinformatics Science Pharmacokinetics 59

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.

RNA 128

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.

Virus 144

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.

Research 111

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.

DNA 128

DNA RNA Disease Hospitals 128

Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. These fields explore highly precise biological processes related to RNA, specific proteins and gene expression mechanisms.

Clinical Development 59

Clinical Development Clinical Trials Clinical Pharmacology Trials 59

SCIENMAG: Medicine & Health

JULY 27, 2023

If translated […]

RNA 69

RNA Research Hospitals Disease 69

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.

RNA 135

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.

DNA 145

DNA Engineering RNA 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.

RNA 115

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.

RNA 143

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

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?

Bioinformatics 76

Bioinformatics Immune Response RNA Packaging 76

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.

RNA 52

RNA Bioethics Disease Research 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.

RNA 97

RNA Engineering Disease Drugs 97

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.

DNA 105

DNA RNA Disease Research 105

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.

Protein Production 105

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.

Treatment 96

Treatment RNA Hospitals Disease 96

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.

RNA 98

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).

RNA 100

RNA Virus Disease Drugs 100

Broad Institute

JULY 19, 2024

These new findings — published in Cell by researchers at Boston Children's Hospital and the Broad Institute of MIT and Harvard — suggest C1q may be involved in age-related cognitive changes and neurodegenerative conditions.

Protein Production 111

Protein Production Hospitals RNA Production 111

Broad Institute

DECEMBER 13, 2023

By Leah Eisenstadt December 13, 2023 Two teams of Broad researchers produced detailed maps of the mouse nervous system. Related news Researchers map brain cell changes in Alzheimer’s disease Courtesy of the Chen and Macosko labs. Courtesy of the Chen and Macosko labs. Courtesy of the Chen and Macosko labs.

DNA 140

DNA Virus Research Engineering 140

Broad Institute

MARCH 27, 2025

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.

Research 52

Research DNA Regulations Disease 52

KIF1A

SEPTEMBER 16, 2023

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 pre-print*, researchers at the Tata Institute of Fundamental Research in Mumbai investigated how a modification to UNC-104, the C.

Regulations 105

Regulations RNA Disease Research 105

DrugBank

MARCH 12, 2025

Introduction Messenger RNA (mRNA) technology has emerged as one of the most significant medical breakthroughs. However, mRNA technology is not limited to infectious diseases. This approach can be personalized for each patient by sequencing the tumor's DNA and RNA to identify unique mutations.

Vaccine 97

Vaccine Treatment Clinical Trials Therapies 97

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. Next week we’re overjoyed to convene families, clinicians, and researchers to discuss progress and goals in our mission to find treatments and cures of KAND.

RNA 98

RNA Clinical Trials Disease Science 98

Broad Institute

AUGUST 19, 2024

A team of researchers at the Broad Institute of MIT and Harvard; the Ragon Institute of Mass General Brigham, MIT, and Harvard; and St. Jude Children’s Research Hospital have discovered that oleic acid, one of the most abundant fatty acids in the body, restores a healthy balance of vaginal microbes in a laboratory model of BV.

Hospitals 121

Hospitals Treatment Medical Schools Science 121

DrugBank

MAY 15, 2024

Researchers would sift through natural extracts , hoping to stumble upon a compound that exhibited beneficial effects. Today, we're able to identify and target specific molecules involved in disease processes—a method that's much more like using a sniper rifle than throwing darts blindfolded.

Small Molecule 98

Small Molecule Molecular Biology Drugs Disease 98

Drug Target Review

JULY 23, 2024

Shortly after finishing my studies, I landed my first job in industry working on cell biology research for several disease indications. At the time, research and leadership roles in biotech/pharma industry were predominately held by men and there were not as many opportunities for career development and mentorship from women leaders.

Small Molecule 116

Small Molecule Targeted Protein Degradation Science Disease 116

DrugBank

MAY 22, 2024

In the dynamic world of drug discovery, the notion of "undruggable" targets presents both a significant challenge and an intriguing frontier for researchers and pharmaceutical companies. Here, researchers are looking at allosteric modulation—hitting different parts of the molecule to increase selectivity.

Small Molecule 96

Small Molecule RNA DNA Pharmaceutical Companies 96

Broad Institute

OCTOBER 28, 2024

By Corie Lok October 28, 2024 Credit: Ricardo Job-Reese, Broad Communications One of the first steps for researchers in studying and analyzing single cells is to determine the cells’ identity: what type and subtype of cells are these, and how similar or different are they to previously analyzed cells? Is CAS now available to use?

Engineering 119

Engineering Cell Biology Disease RNA 119

Broad Institute

JUNE 30, 2023

Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize. Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize.

Small Molecule 105

Small Molecule Cell Based Assays Disease Assay Development 105