Covalent Modifiers

FEBRUARY 25, 2024

2024 , DOI: 10.1039/D3CB00230F N6-Methyladenosine (m6A) is the most prevalent mRNA modification and is required for gene regulation in eukaryotes. TD19 irreversibly modifies the residues C100 and C267, preventing ALKBH5 from binding to m6A-containing RNA.

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RNA Cell Based Assays Regulations Research 81

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

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. Researchers have used a variety of methods to deliver these molecular machines to cells, including lipid nanoparticles and viruses.

Engineering 144

Engineering Research Virus DNA 144

Broad Institute

OCTOBER 16, 2024

William Studier receives the 2024 Merkin Prize in ceremony at the Broad Institute for developing technology used to produce millions of doses of COVID-19 vaccines By Corie Lok October 16, 2024 Breadcrumb Home F. By Broad Communications October 16, 2024 Credit: Erik Jacobs, Anthem Multimedia Richard N. Merkin (left) and F.

Vaccine 64

Vaccine RNA Laboratories Molecular Biology 64

Drugs.com

JANUARY 3, 2024

3, 2024 -- A regularly prescribed class of heart medications might be capable of treating one of the most common forms of muscular dystrophy, a new study in mice suggests.Myotonic dystrophy type 1 (DM1) is caused by abnormal RNA. WEDNESDAY, Jan.

RNA 97

RNA Research Drugs 97

Broad Institute

SEPTEMBER 23, 2024

New approach may yield modified messenger RNAs for treating a wide range of conditions By Ari Navetta September 23, 2024 Breadcrumb Home New approach may yield modified messenger RNAs for treating a wide range of conditions The LEGO framework could help scientists create mRNA therapies that last longer and require smaller doses.

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RNA Vaccine Small Molecule Therapies 133

LifeSciVC

JANUARY 16, 2024

Tirzepatide is also trialing in a Phase 3 obese HFpEF trial, and top-line readout in 2H 2024 will suggest whether the increased weight loss achieved with dual GLP1/GIPR modulation further improves QoL and functional endpoints. ALT) continue to build momentum in the indication. Will incretins “solve” MASH in obese patients?

Small Molecule 139

Small Molecule Trials Therapies Disease 139

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

FEBRUARY 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 Japan performed whole genome sequencing on 315 patients who were enrolled in the HAM patient registry.

RNA 52

RNA DNA Disease Trials 52

Broad Institute

JUNE 18, 2024

Simple test for flu could improve diagnosis and surveillance By Allessandra DiCorato June 18, 2024 Breadcrumb Home Simple test for flu could improve diagnosis and surveillance A low-cost CRISPR-based paper strip test distinguishes between influenza types and can be reprogrammed to recognize different viruses including the H5N1 bird flu virus.

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. One of the reasons that I've traveled so much there over the years is that this is a close research partnership. Online January 25, 2024. Nature Communications. DOI: 10.1038/s41467-024-44800-7.

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.

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DNA RNA Disease Hospitals 128

Broad Institute

OCTOBER 17, 2024

By Leah Eisenstadt October 17, 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 (link).

RNA 69

RNA Hospitals Laboratories Medical Schools 69

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.

Engineering 140

Engineering Protein Production RNA Vaccine 140

Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

Drug Development 52

Drug Development Treatment FDA Approval Therapies 52

Broad Institute

JULY 19, 2024

By Boston Children's Hospital July 19, 2024 Credit: Scott-Hewitt N, et al. By Boston Children's Hospital July 19, 2024 Credit: Scott-Hewitt N, et al. Online June 27, 2024. DOI: 10.1016/j.cell.2024.05.058. 2024.05.058. C1q, produced by microglia (green and blue), interacts with neurons (purple and blue). DOI: 10.1016/j.cell.2024.05.058.

Protein Production 111

Protein Production Hospitals RNA Production 111

Broad Institute

NOVEMBER 6, 2024

By Leah Eisenstadt November 6, 2024 Credit: Ricardo Job-Reese, Broad Communications Despite being one of the rarer complications from immune checkpoint inhibitors, myocarditis is the most deadly. This work provides a biological foundation for testing more targeted therapies for myocarditis due to an immune checkpoint inhibitor.

Immune Response 118

Immune Response Medical Schools Clinical Trials Treatment 118

Broad Institute

SEPTEMBER 11, 2024

Azeet Narayan By Maria Nemchuk September 11, 2024 Goldberg SB, Narayan A, Kole AJ, et al. High-throughput RNA profiling via up-front sample parallelization. Senior Assay Research Scientist Azeet Narayan is a senior assay research scientist at the Broad Institute of MIT and Harvard. Clin Cancer Res. 2018;24(8):1872-1880.

DNA 52

DNA RNA Research Production 52

Broad Institute

AUGUST 29, 2024

Georgia By amatosni@broadinstitute.org August 29, 2024 Georgia Georgia St. The lab's research focuses on how cells respond to maintain essential functions in changing environments. Their research identified six genes that were significantly alternatively spliced in response to temperature stress.

RNA 52

RNA Research 52

Broad Institute

AUGUST 29, 2024

The lab's research centers on how cells respond to maintain essential functions in changing environments. They analyzed RNA sequencing data from the cells of eight organisms, which were cultured at both normal and elevated temperatures, using the rMATS program to compare isoform abundance under these conditions.

RNA 52

RNA Research 52

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.

RNA 84

RNA Virus DNA Vaccine 84

Broad Institute

OCTOBER 27, 2024

Our proposed method will be evaluated on synthetic zero-inflated count datasets, simulated single-cell RNA sequencing (scRNA-seq) data, and real-world multi-sample multi-condition scRNA-seq datasets.

RNA 52

RNA Research 52

Broad Institute

JUNE 14, 2024

Some CRISPR screens may be missing cancer drug targets By Allessandra DiCorato June 14, 2024 Breadcrumb Home Some CRISPR screens may be missing cancer drug targets Current CRISPR guides don’t work equally well in cells from people of all ancestries, which could lead to false negative results.

Drugs 145

Drugs RNA DNA Research 145

Broad Institute

FEBRUARY 27, 2024

Broad-MIT Seminars in Chemical Biology By Rose Circeo February 27, 2024 Breadcrumb Home Research Broad-MIT Seminars in Chemical Biology MIT Chemistry and the Broad Institute host monthly seminars in chemical biology with the intent to hear from the thought-leaders and bring together chemical biologists from both academia and industry in the Boston/Cambridge (..)

RNA 52

RNA DNA Regulations Disease 52

Broad Institute

AUGUST 19, 2024

A common fatty acid may help restore healthy vaginal bacteria after infection By Ari Navetta August 19, 2024 Breadcrumb Home A common fatty acid may help restore healthy vaginal bacteria after infection Treatments using oleic acid, a naturally occurring oil used in cells, could improve treatment of recurrent bacterial vaginosis infections.

Hospitals 121

Hospitals Treatment Medical Schools Science 121

Drug Target Review

MAY 24, 2024

2 AlphaFold 3 has been built to model DNA, RNA and smaller molecules (ligands). ChatGPT [Internet] 2024 [updated 2024 May 12; cited 2024 May] Available from: [link] 2 Howe NP, Thompson B. Nature [Internet] 2024 [updated 2024 May 8; cited 2024 May]. References 1 Wikipedia. Alphafold 3.0:

RNA 59

RNA DNA Bioinformatics Science 59

Broad Institute

MARCH 6, 2024

Schizophrenia and aging may share a common biological basis By Allessandra DiCorato March 6, 2024 Breadcrumb Home Schizophrenia and aging may share a common biological basis Tightly synchronized genetic changes in two types of brain cells may underlie cognitive impairment in both conditions, offering potential therapeutic clues.

Medical Schools 134

Medical Schools RNA Science Research 134

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. Several of these therapies have shown evidence of reaching the brain in humans.

Therapies 137

Therapies FDA Approval Engineering Disease 137

Broad Institute

SEPTEMBER 26, 2024

Luiz Mata Lopez By Rose Circeo September 26, 2024 Related Broad Summer Research Program Luiz Mata Lopez Luiz Mata Lopez is a junior studying computer science and mathematics at the University of Maryland, College Park. Morphological profiling of cells is commonly employed in drug discovery across many disease areas.

RNA 52

RNA Science Disease Research 52

Alta Sciences

APRIL 16, 2024

Inside The Altascientist: The Benefits of PCR for Your Gene Therapy Programs pmjackson Tue, 04/16/2024 - 19:04 The gene therapy landscape continues to accelerate in preclinical and clinical research, with programs constantly in development for targeted, personalized medicines. But how are PCR analysis techniques applied?

Therapies 52

Therapies DNA RNA Laboratories 52

Broad Institute

SEPTEMBER 26, 2024

Liya Mooradian By Rose Circeo September 26, 2024 Related Broad Summer Research Program Liya Mooradian Liya Mooradian is a senior studying Genetics and Science Communication at Iowa State University of Science and Technology. Non-coding genomic regions harbor 90% of disease-associated genetic variants.

RNA 52

RNA Science Disease Research 52

Drug Target Review

AUGUST 28, 2024

We then incubate the cells to mature into ‘pro-regenerative’ macrophages and insert two RNA messages to produce two human proteins that will be expressed at the site of injury when these ‘engineered’ macrophages are given back to the patient. J Hep, 2024 (EASL ILC 2024 Abstract #LBP-007). Reference 1 Brennan P et al.,

Therapies 114

Therapies Disease Engineering Medical Schools 114

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.

DNA 98

DNA RNA Science Therapies 98

Broad Institute

JUNE 27, 2024

By Greta Friar, Whitehead Institute June 27, 2024 Images of a mouse brain show the effect of a technology called CHARM in turning off the expression of a gene in the brain. Previous research has shown that as little as 21 percent elimination of the protein can improve symptoms. Credit: Neumann EN, Bertozzi TM, et al.

Disease 142

Disease Therapies Engineering Clinical Trials 142

Drug Target Review

SEPTEMBER 25, 2024

2022 Dec 12 [cited 2024 Sep 4];22(3):213–34. 2019 Dec 13 [cited 2024 Sep 4];122(4):465–72. 2020 Dec 1 [cited 2024 Sep 4];20(12):743–56. 2021 Oct 11 [cited 2024 Sep 4];13(20):5081. 2021 Apr [cited 2024 Sep 4];2(4):377–91. Cancer research [Internet]. 2024 Mar 22 [cited 2024 Jul 8];84(6_Supplement):5891–1.

Small Molecule 105

Small Molecule Therapies Regulations Treatment 105

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?

Engineering 98

Engineering Cell Biology Disease RNA 98

Broad Institute

JULY 11, 2024

Targeted drug treatment leads tumor cells to imitate viral infection By Ari Navetta July 11, 2024 Breadcrumb Home Targeted drug treatment leads tumor cells to imitate viral infection Exploiting "viral mimicry," mIDH1 inhibitors trick tumors into thinking they are infected with a virus. Online July 11, 2024.

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Treatment Immune Response Virus Drugs 72