Gene Editing News and Research

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Engineered Base Editor Corrects ACTA2 Mutation in Rare Childhood Disease

Multisystemic smooth muscle dysfunction syndrome (MSMDS) is a rare condition associated with stroke, aortic dissection (tearing) and death in childhood.

13 Sep 2025

DNA-Wrapped Nanoparticles Enhance Efficiency of Gene Editing Tools

With the power to rewrite the genetic code underlying countless diseases, CRISPR holds immense promise to revolutionize medicine.

2 Sep 2025

New Gene Silencers Offer Hope for Hereditary Blood Disorders

According to a recent study, chemical tags that were previously thought of as genetic clutter are really potent gene silencers; eliminating them could lead to safer treatments for hereditary blood disorders.

18 Aug 2025

Proteins as a Natural Weapon Against Predatory Bacteria

Amyloids are perhaps best recognized as a major cause of Alzheimer’s disease.

30 Jul 2025

Gene Editing Offers New Hope to Stop Malaria Transmission

Mosquitoes kill more people each year than any other animal. In 2023, the blood-sucking insects infected a reported 263 million people with malaria, leading to nearly 600,000 deaths, 80% of which were children.

24 Jul 2025

Gene Editing Reverses Symptoms of Rare Brain Disorder in Mice

Scientists have corrected gene mutations in mice causing an ultra-rare disease by editing DNA directly in the brain with a single injection, a feat with profound implications for patients with neurological diseases.

22 Jul 2025

Advancing Gene Editing Through Values-Based Regulation

The review from the University of Adelaide argues for balanced regulations on gene editing to support innovation while ensuring societal safety and benefits.

24 Jun 2025

UT Southwestern Researchers Develop Multi-Organ Gene Editing Strategy

A gene-editing delivery system developed by UT Southwestern Medical Center researchers simultaneously targeted the liver and lungs of a preclinical model of a rare genetic disease known as alpha-1 antitrypsin deficiency (AATD), significantly improving symptoms for months after a single treatment, a new study shows.

23 Jun 2025

Targeting the Chagas Parasite by Interrupting Its Lifecycle

Chagas disease is often called a silent killer because many people don't realize they have it until complications from the infection kill them.

9 Jun 2025

Breakthrough Gene Discovery Uncovers a Master Switch for Food Intake

The Gpr45 gene is linked to food intake regulation, offering insights for obesity therapies. Study utilizes Automated Meiotic Mapping for gene identification.

6 Jun 2025

Base Editing Breakthrough Brings Hope for Genetic Disease Treatment

You may have seen it in the news recently: a baby in Pennsylvania with a rare genetic disorder was healed with a personalized treatment that repaired his specific genetic mutation.

5 Jun 2025

Gene Editing Using CRISPR and AAV6 Impacts Long-Term Regeneration of Blood Stem Cells

Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, have found that gene editing using CRISPR-Cas9 in combination with AAV6 vectors can trigger inflammatory and senescence-like responses in blood stem cells, compromising their long-term ability to regenerate the blood system.

4 Jun 2025

New CRISPR Technology for Precise RNA Delivery in Neurons

Proteins produced by RNA segments can aid in the repair of damaged neurons in human bodies.

22 May 2025

Precision Mitochondrial DNA Editing Emerges as a Breakthrough in Neurological Care

Mitochondrial DNA (mtDNA) editing has emerged as a revolutionary approach in the fight against neurodegenerative diseases (NDDs).

13 May 2025

Unraveling How E. histolytica Kills Cells and Evades the Immune System

Understanding how E. histolytica attacks human cells and evades immunity is crucial for developing effective treatments and vaccines against this parasite.

13 May 2025

Depleting TM6SF2 in the Gut Worsens Liver Fat Buildup, Study Finds

A new study finds that gut-specific TM6SF2 depletion exacerbates liver fat accumulation and dysbiosis in mice on high-fat diets, highlighting its importance in gut-liver metabolic regulation.

12 May 2025

ORNL Scientists Develop Fluorescent Biosensor to Track RNA in Plants

Scientists at Oak Ridge National Laboratory have developed a first-ever method of detecting ribonucleic acid, or RNA, inside plant cells using a technique that results in a visible fluorescent signal.

16 Apr 2025

Breakthrough Delivery System Advances CRISPR-Based Treatments

A research team from Helmholtz Munich and the Technical University of Munich has developed an advanced delivery system that transports gene-editing tools based on the CRISPR/Cas9 gene-editing system into living cells with significantly greater efficiency than before.

10 Apr 2025

A Next-Gen CRISPR Tool Just Dropped

Over the past 15 years, developments in the gene-editing technique known as CRISPR-Cas9 have produced significant new understandings of the functions that particular genes play in a variety of disorders.

21 Mar 2025

Study Shows How Transposons Exploit Telomeres in Bacteria

Transposons, or "jumping genes" – DNA segments that can move from one part of the genome to another – are key to bacterial evolution and the development of antibiotic resistance.

8 Mar 2025