For a rare inherited disease, researchers are pursuing three gene therapies.

Published Date: 25 Oct 2023

David Corey, a neurobiologist, had no idea he would face a race against time to create a cure when he attended a conference on a rare disease in 2017.

Read Full News

Latest News

Molecule that could cause COVID clotting key to new treatments

In a surprising discovery, a "sticky molecule" that occurs naturally in our blood vessels could be both a culprit behind blood clots and organ failure during COVID and long COVID and the key to new treatments to counter COVID-related ...

New potential gene-based treatment for sepsis and lung injury

A new discovery from the lab of YouYang Zhao, Ph.D., from Stanley Manne Children's Research Institute at Ann & Robert H. Lurie Children's Hospital of Chicago, opens promising new directions for treatment of life-threatening ...

DNA methylation patterns in newborns point to susceptibility to schizophrenia

Genetic susceptibility to neurodevelopmental conditions such as schizophrenia, autism, and ADHD can be linked, in part, to distinct epigenetic signatures in newborn cord blood. An analysis of nearly 6,000 newborns published ...

CRISPR breakthrough reverses chemotherapy resistance in lung cancer

In a major step forward for cancer care, researchers at ChristianaCare's Gene Editing Institute have shown that disabling the NRF2 gene with CRISPR technology can reverse chemotherapy resistance in lung cancer. The approach ...

Fertility treatments linked to higher mutations than natural conception in mice

Mice pups conceived with in vitro fertilization (IVF) in the lab have slightly increased rates of DNA errors, or mutations, compared to pups conceived naturally, a new study on artificial reproductive technologies suggests.

A time-sensitive genetic switch for sex-specific features of developing neurons

Researchers have identified that the precisely timed transcription of two genes named grim and reaper is responsible for the targeted death of neurons within the developing nervous system of female flies. This group of neurons ...

Todays Updates

Belgian Algorithm Reshapes Endometrial Cancer Care

A new Belgian algorithm may help clinicians choose adjuvant therapy more precisely in early endometrial cancer, moving beyond TNM staging alone.

Todays Updates

Belgian Algorithm Reshapes Endometrial Cancer Care

A new Belgian algorithm may help clinicians choose adjuvant therapy more precisely in early endometrial cancer, moving beyond TNM staging alone.

Latest News

In a surprising discovery, a "sticky molecule" that occurs naturally in our blood vessels could be both a culprit behind blood clots and organ failure during COVID and long COVID and the key to new treatments to counter COVID-related ...

A new discovery from the lab of YouYang Zhao, Ph.D., from Stanley Manne Children's Research Institute at Ann & Robert H. Lurie Children's Hospital of Chicago, opens promising new directions for treatment of life-threatening ...

Genetic susceptibility to neurodevelopmental conditions such as schizophrenia, autism, and ADHD can be linked, in part, to distinct epigenetic signatures in newborn cord blood. An analysis of nearly 6,000 newborns published ...

In a major step forward for cancer care, researchers at ChristianaCare's Gene Editing Institute have shown that disabling the NRF2 gene with CRISPR technology can reverse chemotherapy resistance in lung cancer. The approach ...

Mice pups conceived with in vitro fertilization (IVF) in the lab have slightly increased rates of DNA errors, or mutations, compared to pups conceived naturally, a new study on artificial reproductive technologies suggests.

Researchers have identified that the precisely timed transcription of two genes named grim and reaper is responsible for the targeted death of neurons within the developing nervous system of female flies. This group of neurons ...

News All Time

Surprising blood stem cell diversification revealed in humans

Every second, millions of blood cells are produced in the human body, and about 90% of the cells replaced daily are blood cells. These include red blood cells essential for oxygen transport, platelets for blood clotting, ...

Q&A: Research offers hope for new autoimmune treatments

Alec Whited ('25 MS, Biology) is helping uncover how cells remove waste, a process that could lead to better treatments for autoimmune diseases and improved wound healing.

Tracking RNA levels reveals new clues in triple-negative breast cancer progression

When researchers look for potential cancer therapy targets, they typically go after protein-coding genes that participate in disease progression. Disrupt them and negate whatever role they play in cancer's development or ...

New clinical practice resource on managing RAD51C, RAD51D, and BRIP1 variants released

The American College of Medical Genetics and Genomics (ACMG) has published a new clinical practice resource, "Management of Individuals with Heterozygous Germline Pathogenic Variants in RAD51C, RAD51D, and BRIP1: A clinical ...

Should kids be screened for high cholesterol genes? Study weighs costs and benefits

In the United States, 1 in every 250 people has inherited a genetic variant that leads to dangerously high cholesterol levels from birth.

James Watson helped crack DNA's code, sparking medical advances and ethical debates

On a foggy Saturday morning in 1953, a tall, skinny 24-year-old man fiddled with shapes he had cut out of cardboard. They represented fragments of a DNA molecule, and young James Watson was trying to figure how they fit together ...

Breast cancer relapse: Key cellular resistance mechanism discovered

Triple-negative breast cancer is one of the most aggressive and difficult cancers to treat. It can initially respond well to therapeutic combinations of chemotherapy, but a tiny minority of tumor cells may manage to tolerate ...

Genetically altered hair cells show how inner ear structures are organized

Northwestern Medicine scientists have uncovered how a specific type of cell in the inner ear plays a commanding role in shaping the cellular landscape of the organ responsible for hearing, according to a study published in ...

Key histone variant may explain some forms of infertility

An epigenetic factor that is essential for producing mature egg cells in mice has been identified by RIKEN researchers for the first time. This discovery could aid research into cases of infertility caused by immature eggs.

Hundreds of genes act differently in the brains of men and women

Differences between men and women in intelligence and behavior have been proposed and disputed for decades.

CRISPR screen identifies new regulator of androgen receptor in prostate cancer

A poorly characterized protein, historically thought to be a chaperon or enzyme, may actually be a key player in prostate cancer. In a systematic CRISPR screen, scientists from Arc Institute, UCSF, and the Fred Hutchinson ...

Study identifies key genes linked to aggressive prostate cancer in people of African descent

New prostate cancer research from an international team led by the Center for Genetic Epidemiology at the Keck School of Medicine of USC has yielded discoveries that could improve screening and treatment for patients of African ...

Experimental drug blocks key protein to trigger cancer cell self-destruction in lung tumors

NYU Langone Health researchers found that a type of cell death caused by a buildup of highly reactive molecules suppresses lung tumor growth.

Bowel cancer's 'Big bang': How a crucial moment determines future growth

Like the astronomical explosion that kickstarted the universe, bowel cancer has a "Big Bang" moment which determines how it will grow, according to new research.

DNA discovery could help identify mothers at risk of pre-eclampsia

The human genome is riddled with relics of viral infections—bits of DNA from viruses that have been inserted in human DNA over millions of years and never left. Most are silent but some have taken on functional roles, particularly ...

Hidden nuclear droplets link multiple leukemias and reveal a new therapeutic target

A hidden structure inside the cell is rewriting how scientists understand leukemia. Beneath the microscope, what looked like disorder turned out to follow a simple physical rule—one that connects several major mutations ...