Gene Editing News and Research

RSS

New strategy targets Porphyromonas gingivalis without harming healthy microbes

For years, treating gum disease has meant scraping away plaque, cutting out damaged tissue or turning to antibiotics that kill bacteria indiscriminately.

3 Mar 2026

Identifying the core catalyst for muscle energy production

Researchers have investigated the role of a certain enzyme in regulating energy in muscle and exercise performance for decades, but a new study by Virginia Tech scientists has identified more precisely than ever how this mechanism works.

26 Feb 2026

Breakthrough gene therapy for sickle cell disease remains out of reach in Africa

As Uganda rolls out mandatory nationwide screening of newborns for sickle cell disease this month, a gene therapy celebrated in the United States and Europe remains financially out of reach for most patients in Africa.

25 Feb 2026

Engineers develop highly precise gene editor for safer cystic fibrosis treatments

Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic "base pairs" to a new level of precision, opening the door to safer, more reliable therapies for a wide range of genetic diseases, and to potential treatments for some cystic fibrosis patients that may yield better outcomes than existing therapies.

23 Feb 2026

New gene editing approach offers hope for cystic fibrosis patients

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene into human airway cells, restoring key biological function in a laboratory model of cystic fibrosis and establishing a potential new path toward mutation-agnostic gene therapy for inherited lung diseases.

17 Feb 2026

Cellares to expand automated manufacturing to gene-edited stem cell therapies

Cellares, the first Integrated Development and Manufacturing Organization (IDMO), today announced a collaboration with the Stanford Center for Definitive and Curative Medicine (CDCM) and Stanford Innovative Medicines Accelerator (IMA) to automate manufacturing and release testing for gene-edited hematopoietic stem cell (HSC) therapies, expanding the application of Cellares' automation platforms beyond T cell therapies into a new cell modality.

From Cellares 8 Feb 2026

Research uncovers why certain brain cells are more resilient to tau protein buildup

New research by UCLA Health and UC San Francisco has uncovered why certain brain cells are more resilient than others to the buildup of a toxic protein that is a hallmark of Alzheimer's disease and related dementias, potentially leading to new targets for therapies or treatments.

30 Jan 2026

First topical CRISPR gene therapy corrects disease-causing mutations in human skin

Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the University of British Columbia are extending those possibilities to the skin for the first time.

27 Jan 2026

Purdue researchers discover new therapeutic target for triple-negative breast cancer

A Purdue University team led by Kyle Cottrell has discovered a new therapeutic target for triple-negative breast cancer.

27 Jan 2026

CRISPR-Cas3: A safer gene-editing tool shows promise for transthyretin amyloidosis treatment

Genetic disorders occur due to alterations in the primary genetic material, deoxyribonucleic acid (DNA), of an organism. Transthyretin amyloidosis (ATTR) is a progressive disorder involving amyloid deposits of misfolded transthyretin (TTR) proteins.

14 Jan 2026

APOE gene: A major driver in Alzheimer's disease

Potentially more than 90% of Alzheimer's disease cases would not occur without the contribution of a single gene (APOE), according to a new analysis led by UCL researchers.

9 Jan 2026

CIRM awards $7.4 million to advance stem cell-based gene therapy for Friedreich's ataxia

The California Institute for Regenerative Medicine (CIRM) has awarded $7.4 million to support a University of California San Diego team developing a first-of-its-kind stem cell-based gene therapy for Friedreich's ataxia, a rare inherited neurodegenerative disease that causes progressive loss of coordination, muscle strength, heart function and overall mobility.

8 Jan 2026

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