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

Broad Institute

OCTOBER 17, 2024

The method, called Genotypic and Phenotypic Antibiotic Susceptibility Testing through RNA detection, or GoPhAST-R, analyzes the growth and genetic activity of the bacteria to quickly determine the pathogen’s susceptibility to various medicines.

RNA 69

RNA Hospitals Laboratories Medical Schools 69

The Pharma Data

NOVEMBER 22, 2020

At the moment, CRISPR Cas-9 is only utilized for rare diseases on cells that have been removed from the body. The product is the most advanced gene-editing approach in development for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease. Cas-9 represents the Cas9 protein, an enzyme that cuts foreign DNA.

DNA 52

DNA Medical Schools Treatment Science 52

Broad Institute

JUNE 21, 2023

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

Small Molecule 52

Small Molecule Cell Based Assays Medical Schools Disease 52

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., Dr. Zhang completed his postdoctoral fellowship at Harvard Medical School/Boston Children’s Hospital. as Head of Business Development.

Medical Schools 40

Medical Schools Clinical Development Therapies RNA 40

Drug Target Review

JULY 1, 2024

As a non-NSAID, it reduces adverse effects associated with these pain relief drugs, including kidney injury, GI gastritis/ulceration and blood pressure elevation, which can affect patients with cardiovascular disease and the elderly. We set out to improve health by developing a novel non-opioid that avoids potential abuse.

Treatment 59

Treatment Clinical Trials Therapies Molecular Biology 59

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. The scientists identified a suite of changes in cells unique to the early stages of Alzheimer’s, including some not seen before in animal studies.

Disease 144

Disease Medical Schools Research Hospitals 144

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

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.

Immune Response 138

Immune Response RNA Disease Medical Schools 138

Broad Institute

MAY 2, 2024

Today, she is a senior associate computational biologist in the Getz lab, where she applies computational techniques to DNA and RNA sequencing data to analyze rapid autopsy samples, taken from multiple sites throughout the body at the time of a cancer patient’s death. I love that in all forms of science, you solve problems.

Science 124

Science Medical Schools Disease Hospitals 124

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. She graduated from UC San Diego with a B.S.

Science 116

Science Medical Schools Research RNA 116

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.

RNA 98

RNA Medical Schools Engineering Disease 98

Broad Institute

AUGUST 14, 2023

More recently, they are focusing on proteins to understand the biological events in cancer cells that lead to disease. Ding’s team studied a range of features of more than 5,000 driver mutations such as their frequencies across different cancers and their impact on RNA, proteins, protein complexes, and PTMs.

DNA 98

DNA RNA Regulations Treatment 98

The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. She’s a pediatric hematology-oncology specialist at Cohen Children’s Medical Center in New Hyde Park, N.Y.

Therapies 52

Therapies Disease Medical Schools Virus 52

Broad Institute

NOVEMBER 20, 2024

The intestine maintains a delicate balance in the body, absorbing nutrients and water while maintaining a healthy relationship with the gut microbiome, but this equilibrium is disrupted in parts of the intestine in conditions such as celiac disease, ulcerative colitis, and Crohn’s disease.

Regulations 84

Regulations Immune Response Molecular Biology Disease 84

Broad Institute

JUNE 13, 2023

The results add to our understanding of the development of BPDCN, which is critical to devising new and better treatments for the disease," he continues. To better understand this process, researchers did a deep dive into the genetics of patients' bone marrow, blood, and skin leukemia cells – sequencing the DNA and RNA in individual cells. "We

Hospitals 52

Hospitals Disease Medical Schools Research 52

Codon

JUNE 26, 2023

A T4 phage can hold 171,000 bases of DNA or other molecules, including proteins and RNA. It uses single-molecule RNA fluorescence to measure mRNAs and fluorescent reporters to measure the proteins. The animals had a common disease variant, caused by a single amino acid substitution in the phenylalanine hydroxylase enzyme.

DNA 52

DNA Therapies Engineering Clinical Trials 52

Codon

JUNE 26, 2023

A T4 phage can hold 171,000 bases of DNA or other molecules, including proteins and RNA. It uses single-molecule RNA fluorescence to measure mRNAs and fluorescent reporters to measure the proteins. The animals had a common disease variant, caused by a single amino acid substitution in the phenylalanine hydroxylase enzyme.

DNA 52

DNA Therapies Engineering Clinical Trials 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. Harvard Medical School researchers recently described an “AI tool” that “Decodes Brain Cancer’s Genome During Surgery,” in the journal Med.

Disease 92

Disease DNA Virus RNA 92

Broad Institute

JANUARY 30, 2024

Bhattacharyya wears many coats today, splitting his time between the Broad and the infectious disease ward at MGH, where a diagnostic method he developed at Broad is now being tested to see if it helps patients quickly receive the best antibiotics for their infections.

Hospitals 137

Hospitals Virus Medical Schools Science 137

keypoint

JANUARY 8, 2025

Dr. Cheruiyot is a post-doctoral fellow at the Joslin Diabetes Center, Harvard Medical School (HMS) I n the lab of Dr. Jean Schaffer, s he studies molecular mechanisms underlying protein synthesis in pancreatic beta cells, and alterations of such mechanisms in diabetes.

Medical Schools 59

Medical Schools RNA Laboratories Doctors 59

Elrig

MARCH 10, 2025

The free-to-attend event will bring together scientists, researchers and entrepreneurs from across the drug discovery community working on proteins, RNA, cell and gene therapies, PROTACs, as well as the application of big data to support target identification, biomarkers and the development of big molecules.

Research 52

Research Laboratories Medical Schools Engineering 52

Broad Institute

JANUARY 7, 2025

A clinical genomicist harnesses team-based science to help rare-disease patients By Allessandra DiCorato January 7, 2025 Breadcrumb Home A clinical genomicist harnesses team-based science to help rare-disease patients Heidi Rehm convinced labs and scientists to work together and share data.

Disease 121

Disease Science Laboratories Medical Schools 121

Broad Institute

JANUARY 16, 2025

By Allessandra DiCorato January 16, 2025 Credit: Scott Sassone, Broad Communications Tara McDonald (left) and Steve McCarroll (right) are coauthors of a new paper describing a biological mechanism underlying Huntington's disease. The cumulative death of many such cells leads to the symptoms of Huntingtons disease.

Disease 130

Disease DNA Medical Schools RNA 130

Broad Institute

NOVEMBER 6, 2024

They also saw an increase in abundance of several immune cell subsets, as well as an increase in abundances of certain cellular groupings composed of specific cytotoxic T cells, conventional dendritic cells (cDCs), and inflammatory fibroblasts that were found together in the hearts of patients with active disease.

Immune Response 125

Immune Response Medical Schools Clinical Trials Treatment 125

The Pharma Data

DECEMBER 23, 2020

One person familiar with the list said it included the lipids that encase the RNA material in both the Moderna and Pfizer vaccines, the Times reported. ” Several infectious disease experts said Monday the variant may not have even originated in Britain, the Washington Post reported. “It may very well be here.

Vaccine 52

Vaccine Government Virus Medical Schools 52

The Pharma Data

DECEMBER 28, 2020

” With more than 19 million COVID-19 cases in the United States by Monday and more than 333,000 Americans now killed by the disease, more people than ever have now been personally affected by the new coronavirus. National Institute of Allergy and Infectious Diseases, said last week. “It may very well be here.

Vaccine 52

Vaccine Government Virus Medical Schools 52