KIF1A

APRIL 6, 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. When a mutation causes a deletion or duplication of larger areas of DNA, we call it Copy Number Variation. She graduated from UC San Diego with a B.S.

DNA 111

DNA Medical Schools Therapies Research 111

Broad Institute

FEBRUARY 16, 2024

The way that clinicians subdivide diabetes patients now is based on symptoms, but in this study, the frequency of genetic risk factors seems to vary among patients with youth-onset T2D,” said Jason Flannick , Broad associate member and assistant professor at Boston Children’s Hospital and Harvard Medical School. “We

Medical Schools 133

Medical Schools Hospitals Disease DNA 133

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 DNA 98

Broad Institute

NOVEMBER 13, 2023

Liquid biopsies enable clinicians to find and analyze tumor DNA in a patient’s blood sample to detect cancer early, monitor cancer recurrence, assess the patient’s response to treatment, and measure other clinically important features in real time, without invasive procedures.

DNA 116

DNA Medical Schools Treatment Therapies 116

PLOS: DNA Science

SEPTEMBER 5, 2024

Researchers from Harvard Medical School describe a new ChatGPT-like model that can guide clinical decision-making to diagnose, treat, and predict survival for several types of cancer. Analyzing DNA patterns using AI may replace time-consuming process DNA and genomic sequencing tests, Yu said.

Treatment 52

Treatment FDA Approval DNA Small Molecule 52

Drug Target Review

DECEMBER 28, 2023

During the process of transformation from a normal cell into a cancer cell, a cell acquires a series of changes, or mutations, in its DNA. But DNA mutations can also result in changes to the proteins that are displayed on the surface of the cancer cell.

Bioinformatics 105

Bioinformatics Immune Response DNA Vaccine 105

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

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

Drug Target Review

JANUARY 15, 2024

Unlike almost every other cell type (except B cells), T cells do not have the exact same chromosomal DNA sequences as other cells in the body. This produces a great advantage in infectious disease, as it becomes difficult for a virus to adapt and spread from person to person, because each person’s HLA array is genetically distinct.

Engineering 110

Engineering DNA Virus Immune Response 110

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

PLOS: DNA Science

AUGUST 22, 2024

Crichton had an MD from Harvard Medical School, but he never practiced medicine. Unforgettably, my daughter Heather threw up on the volcano, an early sign of gallbladder disease and to this day, her claim to fame. A plant genome consists of DNA, and NOT pieces of 2,4-D, which is a completely unrelated organic acid.

DNA 52

DNA Virus Organic Chemistry Science 52

Broad Institute

AUGUST 14, 2023

More recently, they are focusing on proteins to understand the biological events in cancer cells that lead to disease. With this approach, the team uncovered patterns in protein modifications linked to cancer-related processes such as DNA repair. Zamecnik Chair in Oncology at the Massachusetts General Hospital Cancer Center.

DNA 98

DNA RNA Regulations Treatment 98

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

Broad Institute

JULY 20, 2023

It's clear that new technologies, analytic techniques and models of disease are needed that can accommodate the polygenic background influences on even penetrant disease alleles before we can hope to gain greater understanding of pathological mechanisms. Please register at broad.io/stanleysymposium Please register at broad.io/stanleysymposium

Medical Schools 52

Medical Schools Hospitals DNA Disease 52

Broad Institute

JULY 10, 2023

Credit: Allison Colorado, Broad Communications Arriving in the Bay Area after a childhood in increasingly diverse surroundings, Martin became acutely aware of health disparities and the profound differences in disease prevalence among different populations. I wanted to know how I could have any impact on studying genetic diversity.”

Bioinformatics 98

Bioinformatics Disease Medical Schools Research 98

Broad Institute

AUGUST 7, 2023

STING is primarily on the lookout for DNA, which can indicate either a foreign invader such as a virus or damage to the host tissue or cell. Now, a team of MIT, Massachusetts General Hospital (MGH), Broad Institute of MIT and Harvard, and Harvard Medical School (HMS) researchers has discovered how STING activates those two pathways.

Regulations 91

Regulations Clinical Trials Engineering DNA 91

PLOS: DNA Science

SEPTEMBER 21, 2023

In addition to rare genetic diseases, talks at the Festival will address Crohn’s disease, rheumatoid arthritis, polycystic ovarian syndrome, and neurological disease and cancers. What steps should researchers take when attempting to acquire DNA samples from modern descendants? From people who have died?”

DNA 52

DNA Science Medical Schools Government 52

KIF1A

NOVEMBER 1, 2024

We know that living with a rare disease can be an overwhelming and isolating journey. The earlier a disease is identified the earlier a family can find appropriate treatment, which can save lives and have profound impacts on quality of life. of participants, making a case that newborn sequencing is feasible and useful.

Treatment 52

Treatment Medical Schools Research Disease 52

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

Broad Institute

FEBRUARY 29, 2024

In the new microbes’ DNA, the researchers found hundreds of novel genes that may offer clues to how enterococci are able to resist antibiotic treatment and thrive in the hospital environment. The findings could one day help monitor the emergence of new drug-resistant threats or highlight possible ways to prevent or treat those infections.

Hospitals 116

Hospitals Medical Schools International Research 116

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

Broad Institute

AUGUST 4, 2022

By Tracy Hampton August 4, 2022 Credit: National Institutes of Allergy and Infectious Diseases, National Institutes of Health Colorized scanning electron micrograph of a B cell lymphocyte from a human donor.

Bioinformatics 52

Bioinformatics Medical Schools Hospitals Treatment 52

KIF1A

MAY 31, 2024

Chung serves as the Mary Ellen Avery Professor at Harvard Medical School (HMS), and President of the Children’s Hospital Pediatric Associates. ASCEND Online and KOALA In-Person Natural History Study Updates Understanding KAND and the progression of the disease informs our path to treatment.

Treatment 52

Treatment Small Molecule International Research 52

Codon

JUNE 26, 2023

DNA sequences are designed on a computer, and it takes a dozen or more clicks to change a single nucleotide. DNA sequences are also checked by hand, so it’s easy to make a mistake. The tool outputs a DNA sequence that encodes all the required enzymes. Anyone who has tried to engineer a cell knows how tedious it can be.

DNA 52

DNA Therapies Engineering Clinical Trials 52

Codon

JUNE 26, 2023

DNA sequences are designed on a computer, and it takes a dozen or more clicks to change a single nucleotide. DNA sequences are also checked by hand, so it’s easy to make a mistake. The tool outputs a DNA sequence that encodes all the required enzymes. Anyone who has tried to engineer a cell knows how tedious it can be.

DNA 52

DNA Therapies Engineering Clinical Trials 52

Codon

OCTOBER 6, 2024

However, in a recent study using sheep , a team at the University of Michigan Medical School found that a nitric-oxide-rich ECMO circuit allowed them to operate an artificial womb without blood thinners. Finally, transferring the baby to the artificial womb poses several challenges.

FDA 92

FDA Hospitals Engineering Research 92