Gene Editing News and Research

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Nature-inspired vesicles offer new path for precision medicine therapies

A quiet revolution is underway in modern medicine: Drug development is aiming to move from managing disease to correcting it through RNA and gene-editing therapies.

17 Apr 2026

Stanford researchers create noninvasive way to deliver light deep inside the body

Light has an increasing number of applications in biology and medicine – it can be used to stimulate cell growth, manipulate neural signals, and treat some cancers – but it doesn't easily pass through tissue.

13 Apr 2026

Nanotube membrane injector enables precise transfer of cytoplasmic contents between cells

Cells are not isolated units; they continuously exchange proteins, genetic material, and even entire organelles with their neighbors.

3 Apr 2026

Gene-edited treatment shows promising outcomes for severe sickle cell disease

New results from a clinical trial show promising outcomes for a gene-edited treatment for severe sickle cell disease, a genetic blood disorder with few curative options.

1 Apr 2026

Tiny gene mutations shape cancer behavior and immune response

Cancer is often thought of as a single disease. Yet even tumors that arise in the same organ can follow very different genetic paths.

19 Mar 2026

SonoPIN technology boosts intracellular delivery of large cancer drugs

Engineers at Duke University have demonstrated a technique that uses microbubbles and ultrasound to help relatively large cancer drugs enter tumor cells and cause them to self-destruct.

13 Mar 2026

DNA-based barcoding reveals which nanoparticles successfully deliver genetic therapies

Drug delivery researchers have vastly improved the potential of genetic therapies by overcoming the challenge of consistently getting genes and gene-editing tools where they need to be within cells.

11 Mar 2026

New CRISPR technique boosts mitochondrial function to treat heart failure

After a heart attack, the heart struggles to recoup and maintain energy. One third of patients develop heart failure as a result ⎯ a condition that impacts 6.8 million Americans and carries a high lifetime risk with 1 in 4 adults in the U.S. expected to develop the condition during their lifetime.

9 Mar 2026

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