Gene Editing News and Research

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Reprogramming immunity to protect beta cells in type 1 diabetes

In the battle against type 1 diabetes (T1D), one researcher at the Medical University of South Carolina (MUSC) is leading a bold new front. With $1 million in funding from Breakthrough T1D, the leading global T1D research and advocacy organization, Leonardo Ferreira, Ph.D., assistant professor of Pharmacology and Immunology, and his collaborators at partnering institutions will explore a new approach to treating – and potentially curing – the disease.

7 Jan 2026

Researchers identify hundreds of genes essential for the development of brain cells

An international research team identified hundreds of genes essential for the development of brain cells, including one gene linked to a severe neurodevelopmental disorder not previously described.

5 Jan 2026

New method can help better assess precision genome editing technology

Scientists and physicians can better assess precision genome editing technology using a new method made public today by St. Jude Children's Research Hospital. Significant amounts of time and resources spent improving CRISPR gene editing technology focus on identifying small off-target sites that pose a safety risk, which is also technically challenging.

2 Jan 2026

Advances in hemophilia gene therapy bring hope for pediatric patients

In the past three years, gene therapy has reshaped what's possible in hemophilia treatment for patients 18 and older. But a key question remains: How soon will these advances reach children?

18 Dec 2025

Junk' DNA may hold new clues to Alzheimer’s disease

When most of us think of DNA, we have a vague idea it's made up of genes that give us our physical features, our behavioral quirks, and keep our cells and organs running.

18 Dec 2025

Researchers uncover how a gene influences cell size across various cell types

What keeps our cells the right size? Scientists have long puzzled over this fundamental question, since cells that are too large or too small are linked to many diseases. Until now, the genetic basis behind cell size has largely been a mystery.

17 Dec 2025

Scientists discover rapid synapse adaptation in neuronal communication

Every movement you make and every memory you form depends on precise communication between neurons. When that communication is disrupted, the brain must rapidly rebalance its internal signaling to keep circuits functioning properly.

16 Dec 2025

Virus shells use asymmetry to control RNA release and infection

Viruses are typically described as tiny, perfectly geometric shells that pack genetic material with mathematical precision, but new research led by scientists at Penn State reveals a deliberate imbalance in their shape that helps them infect their hosts.

12 Dec 2025

Study shines new light on how cancer avoids the mitotic stopwatch pathway

When cell division (mitosis) takes too long, it can be a sign that something is wrong with the cells, for example DNA damage or chromosomal instability.

10 Dec 2025

AI-assisted liquid biopsies show promise for early cancer detection

The prospect of diagnosing cancer through painless methods—such as a simple AI-assisted blood or urine test that detects tiny particles known as exosomes—is becoming increasingly realistic.

2 Dec 2025

Researchers uncover GFAP’s role in mitochondrial dysfunction

Some brain disorders are straightforward, such as the direct frontal lobe assault of a concussion or traumatic brain injury. Others, like Alexander disease, are akin to guerilla warfare.

1 Dec 2025

Regenerative medicine offers a pathway toward curing type 1 diabetes

Type 1 diabetes (T1D) is an autoimmune disorder characterized by the specific destruction of insulin-producing pancreatic β-cells.

26 Nov 2025

Scientists uncover how brain enzyme OTULIN controls tau expression

Scientists have uncovered a surprising mechanism by which a brain enzyme called OTULIN controls the expression of tau, the protein that forms toxic tangles in Alzheimer's disease.

25 Nov 2025

Researchers use organoids to study the earliest moments of electrical activity in the brain

Humans have long wondered when and how we begin to form thoughts. Are we born with a pre-configured brain, or do thought patterns only begin to emerge in response to our sensory experiences of the world around us? Now, science is getting closer to answering the questions philosophers have pondered for centuries.

24 Nov 2025

ASHG to host Genetic Diagnosis & Rare Disease Virtual Symposium

The American Society of Human Genetics (ASHG) will host the Genetic Diagnosis & Rare Disease Virtual Symposium on December 2-3. This initiative ensures that groundbreaking research and clinical innovations are accessible to scientists, clinicians, and educators.

22 Nov 2025

CRISPR-edited fungus boosts protein production and cuts environmental impact

In a new study publishing November 19 in the Cell Press journal Trends in Biotechnology, researchers used a gene-editing technology called CRISPR to increase a fungus's production efficiency and cut its production-related environmental impact by as much as 61%-all without adding any foreign DNA.

21 Nov 2025

Innovative CRISPR strategy resensitizes lung cancer to treatment

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.

17 Nov 2025

Switching APOE4 to APOE2 in adult mice reduces Alzheimer’s pathology and improves memory

Replacing APOE4 with APOE2 in adult mice rapidly reshapes brain lipids and gene expression, even after development is complete. This switch, especially when targeted to astrocytes, reduces amyloid burden, dampens gliosis, and improves associative memory despite E2-like peripheral lipid risks.

12 Nov 2025

One-time CRISPR therapy safely lowers cholesterol and triglycerides in early trial

In a 15-patient, Phase 1 one, first-in-human trial, a one-time, CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol and triglycerides in people with difficult-to-treat lipid disorders, according to a preliminary late-breaking science presentation today at the American Heart Association's Scientific Sessions 2025.

10 Nov 2025

New initiative focuses on advancing human disease research through AI and frontier biology

Mark Zuckerberg and Priscilla Chan today announced the launch of a first-of-its-kind initiative combining frontier artificial intelligence and frontier biology to dramatically accelerate scientific progress toward understanding and addressing human disease.

7 Nov 2025