Gene Editing News and Research

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Study demonstrates successful gene therapy for maple syrup urine disease

A study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, restored coordinated expression of the affected genes and stabilized biomarkers in a calf as well as in mice.

28 Feb 2025

Researchers make breakthrough in understanding the role of tau protein in Alzheimer's

A research team at the University of Cologne has made a significant breakthrough in understanding the role of the tau protein in Alzheimer's disease.

28 Feb 2025

Cambridge launches program to tackle engineered pandemic risks

Covid-19 showed us how vulnerable the world is to pandemics – but what if the next pandemic were somehow engineered? How would the world respond – and could we stop it happening in the first place?

27 Feb 2025

Genetic mutation offers hope for better rheumatoid arthritis treatments

Research led by York University's Faculty of Health reveals how a specific mutation in a protein called TRAF1 can shut down an overactive immune response, dramatically reducing inflammation in mice.

27 Feb 2025

Gene editing shows promise for treating trisomy at cellular level

Gene editing techniques may eventually allow trisomy to be treated at the cellular level, according to an in vitro proof-of-concept study.

18 Feb 2025

Human-specific gene variant may hold key to evolution of language

The origins of human language remain mysterious. Are we the only animals truly capable of complex speech? Are Homo sapiens the only hominids who could give detailed directions to a far-off freshwater source or describe the nuanced purples and reds of a dramatic sunset?

18 Feb 2025

Stanford researchers identify key variants linked to inherited cancer risk

Thousands of single changes in the nucleotides that make up the human genome have been associated with an increased risk of developing cancer.

17 Feb 2025

Discovery of gene silencer offers hope for autosomal dominant leukodystrophy

A team led by University of Pittsburgh School of Public Health geneticists has shown, for the first time, that a gene "silencer" that resides in junk DNA is directly sparing people from a devastating and fatal progressive neurological disease.

13 Feb 2025

One shot to lower cholesterol for life? Scientists unlock a groundbreaking gene therapy

Researchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction without altering DNA, offering a potential one-time treatment for high cholesterol.

11 Feb 2025

MaxCyte® acquires SeQure Dx to broaden cell engineering offerings with on-target and off-target editing assessments

MaxCyte, Inc., a leading, cell-engineering focused company providing solutions to advance the discovery, development and commercialization of next-generation cell therapeutics announced today the acquisition of SeQure Dx, a market leader of on-target and off-target editing assessment services for cell and gene therapies.

From MaxCyte, Inc. 6 Feb 2025

Breakthrough drug improves treatment of medulloblastoma in mice

Brain cancer is the second-leading cause of death in children in the developed world. For the children who survive, standard treatments have long-term impacts on their development and quality of life, particularly in small children and infants.

4 Feb 2025

Engineered fat cells starve tumors and prevent cancer growth

Scientists transformed energy-storing white fat cells into calorie-burning 'beige' fat. Once implanted, they outcompeted tumors for resources, beating back five different types of cancer in lab experiments.

4 Feb 2025

Breakthrough in gene-editing with Cas12a for modeling human diseases

Australian cancer researchers are the first to establish a next-generation gene-editing tool for modeling and interrogating human disease.

31 Jan 2025

New findings could make off-the-shelf CAR T cell therapy a reality

CAR T cell therapy is one of the most promising new cancer treatments to emerge in recent years. It involves removing a patient's own immune T cells and engineering them to recognize specific targets on the surface of the cancer cell.

31 Jan 2025

BEND lipids revolutionize mRNA delivery and gene-editing

Every time a shuttle docks with the International Space Station (ISS), a delicate dance unfolds between the shuttle's docking system and its counterpart on the station.

28 Jan 2025

New rabbit model provides insights into CF-related pancreatic endocrine pathology

Cystic fibrosis (CF) is a life-threatening genetic disease affecting multiple organ systems, with pancreatic dysfunction representing a critical and often overlooked complication.

17 Jan 2025

Researchers launch startup to revolutionize targeted drug delivery using milk exosomes

Two Nebraska researchers have launched a startup company aimed at bringing to market an innovative method for delivering therapeutics, gene editing tools, plasmids and more to targeted locations in the human body.

17 Jan 2025

New approach shows promise in fighting multiple cancers

The tissue adjacent to a tumor behaves differently than areas farther away: The tumor's cancerous cells influence their surroundings, blocking the body's immune defenses and creating a sort of haven in which the tumor can grow. Treatments that target some of these pro-tumor actions are effective in a number of cancers, but only for some patients; in others, these treatments have little effect.

17 Jan 2025

New gene-editing treatment extends lifespan in mice with prion disease

Researchers at the Broad Institute of MIT and Harvard have developed a gene-editing treatment for prion disease that extends lifespan by about 50 percent in a mouse model of the fatal neurodegenerative condition.

15 Jan 2025

Developing and refining muscle atrophy models for drug discovery

With the intensification of global population aging, muscle atrophy, characterized by the loss of muscle mass and function, has become an important health issue affecting the elderly. Researchers have widely used various animal and cellular models to gain a deeper understanding of the pathophysiological mechanisms of muscle atrophy and develop effective treatment strategies.

9 Jan 2025