Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

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

GEMORNA Platform Generates Novel mRNA Sequences With Superior Drug Properties

Raina Biosciences Inc., ("Raina"), an mRNA technology and therapeutics company, today announced the publication of data from its generative AI platform in Science.

28 Aug 2025

New Tool Offers Insight Into When Genes Turn On and Off in the Brain

A Yale research team has created a new computer tool that can pinpoint when exactly genes turn on and off over time during brain development - a finding that may one day help doctors identify the optimal window to deploy gene therapy treatments.

20 Aug 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

AI-Powered Gene Editing Method Enables Unprecedented Precision

A research team headed by the University of Zurich has developed a powerful new method to precisely edit DNA by combining cutting-edge genetic engineering with artificial intelligence.

13 Aug 2025

Prime Editing Reverses Symptoms of Severe Childhood Neurological Disease

Scientists use a precise form of gene editing called prime editing to correct the most common genetic mutations that cause alternating hemiplegia of childhood, a rare and severe neurological disorder that begins in infancy.

From The Jackson Labs 6 Aug 2025

UCLA Researchers Reprogram Stem Cells to Produce Continuous Cancer-Fighting T Cells

In a first-of-its-kind clinical trial, UCLA scientists have shown it's possible to reprogram a patient's blood-forming stem cells to generate a continuous supply of functional T cells, the immune system's most powerful cancer-killing agents.

5 Aug 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

New Software Boosts Genome Editing Accuracy for Genetic Therapies

This innovative software, CRISPRware, optimizes guide RNA design for genome editing, supporting research on genetic disorders and uncharacterized peptides.

9 Jul 2025

Engineered Macrophages Enable CAR T Cells to Overcome Glioblastoma Barriers

A team of researchers from the San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET, Milan), led by Nadia Coltella and Luigi Naldini, has unveiled a powerful strategy to rejuvenate the effectiveness of chimeric antigen receptor (CAR) T cell therapy against glioblastoma, one of the most lethal and treatment-resistant brain tumors.

4 Jul 2025

Scientists Grow Liver Organoids with Functional Blood Vessels

Scientists from Cincinnati Children's and colleagues based in Japan report achieving a major step forward in organoid technology--producing liver tissue that grows its own internal blood vessels.

26 Jun 2025

Gene Editing Halts Symptoms of Rare Smooth Muscle Disease in Preclinical Study

Using gene editing in a preclinical model, researchers at UT Southwestern Medical Center blocked the symptoms of a rare smooth muscle disease before they developed.

25 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

Blocking Viral Enzyme Boosts Brain Immunity Against HSV-1

Infections caused by herpes simplex virus type 1 (HSV-1) can lead to HSV-1 encephalitis-a rare but deadly condition that inflames the brain.

4 Jun 2025

Study Identifies SMARCB1 and MEOX2 Genes as Growth Regulators of Lung Cancers

A Cancer Gene Therapy study reveals that MEOX2 promotes lung cancer progression while SMARCB1 acts as a tumor suppressor, influencing EGFR expression and response to tyrosine kinase inhibitors.

30 May 2025

Autophagy: A Double-Edged Sword in Cancer and Protein Conformational Disorders

A new FEBS Letters review explores the dual role of autophagy in cancer and protein misfolding diseases—highlighting its potential as a therapeutic target and the challenges of tipping the balance between protection and harm.

20 May 2025

Scientists Develop EvoCAST for Efficient and Accurate Gene Insertion

Ask scientists what gene editing tool is most needed to advance gene therapy, and they'd probably describe a system that's now close to realization in the labs of Samuel Sternberg at Columbia University Vagelos College of Physicians and Surgeons and David Liu at the Broad Institute of MIT and Harvard.

16 May 2025