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 RNA Research 116

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. First author Zoë Levine is an MD-PhD candidate at Harvard Medical School and graduate student in the lab of Pardis Sabeti at the Broad.

Research 111

Research Disease Medical Schools RNA 111

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

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 Hospitals RNA 40

Drug Discovery World

OCTOBER 25, 2023

DDW Editor Reece Armstrong speaks to Dr Darrell Green , Lecturer in RNA Biology Biomedical Research Centre Norwich Medical School University of East Anglia, about his work using next generation sequencing (NGS) and the areas the technology is impacting within drug discovery and development.

Bioinformatics 287

Bioinformatics Pharmacogenetics RNA Medical Schools 287

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 Hospitals Research 144

Drug Discovery World

DECEMBER 26, 2023

Representing a dominant field of drug discovery, it isn’t surprising that breakthroughs in cancer research were popular with DDW readers in 2023, particularly strategies to overcome treatment resistance and prevent the spread of disease. The drug works by targeting proprotein convertase subtilisin/kexin type 9 serine protease (PCSK9).

Clinical Trials 162

Clinical Trials Vaccine RNA Trials 162

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 Medical Schools Virus Science 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.

Immune Response 138

Immune Response RNA Medical Schools Disease 138

Broad Institute

AUGUST 31, 2023

We were surprised to see that just missing one copy of this gene can induce many changes at the RNA level, at the protein level, at the functional level, and at the behavioral level,” said Farsi. Tags: Stanley Center for Psychiatric Research Psychiatric Disease Aug 31, 2023. DOI: 10.1016/j.neuron.2023.08.004. 2023.08.004.

RNA 143

RNA Medical Schools Research Science 143

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 Medical Schools 98

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

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

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 Molecular Biology Therapies 59

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 Hospitals Disease 124

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 Medical Schools Disease Research 52

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 RNA Science 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

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