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.

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

Novel Therapeutic Strategy for Healthy Aging and Longevity

An international study led by the Institut de Neurociències at the UAB (INc-UAB) found that raising levels of the Klotho protein in mice extends lifespan and improves both physical and cognitive health as they age.

8 May 2025

AI-Powered Protein Engineering for Precise and Scalable Gene Therapy

Genome editing has progressed rapidly, showing promising outcomes for the treatment of genetic disorders, yet there remains potential for enhancement.

23 Apr 2025

Researchers Develop STITCHR to Insert Whole Genes Without Unwanted Mutations

Investigators from Mass General Brigham and Beth Israel Deaconess Medical Center have developed STITCHR, a new gene editing tool that can insert therapeutic genes into specific locations without causing unwanted mutations.

12 Apr 2025

4basebio Receives MHRA GMP Licence for Manufacture and Supply of Synthetic DNA

4basebio PLC (“4basebio”), a company which develops and commercializes the large-scale manufacture of synthetic DNA as well as nanoparticle delivery solutions, today announced that it has received Good Manufacturing Practice (GMP) certification from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).

3 Apr 2025

Breakthrough Gene Therapy Shows Promise for Treating Hereditary Hearing Loss

Congenital hearing loss refers to impaired auditory function that occurs due to genetic causes. GJB2 is the gene responsible for approximately half of all cases of hereditary hearing loss.

28 Mar 2025

New Insights into RNA Editing Mechanisms for Autoimmune and Cancer Therapies

A research team led by Yang Gao from Rice University has made significant strides in understanding the molecular mechanisms of ADAR1, a protein that modulates ribonucleic acid (RNA) induced immune responses.

18 Mar 2025

Protective ‘Slime’ In The Brain May Help Prevent Aging And Neurodegeneration

The study shows that boosting mucin-type O-glycosylation in aged mice reinforces the blood-brain barrier and improves memory, paving the way for new treatments.

10 Mar 2025

Limula Unveils LimONE: A Breakthrough in End-to-End Cell Therapy Manufacturing

Limula has unveiled LimONE, a disruptive cell therapy manufacturing solution that automates complex and fragmented processes into a seamless workflow.

From Limula SA 4 Mar 2025

New Approach Aims to Make CRISPR Therapy Safer and More Effective

CRISPR is a powerful gene-editing tool that holds enormous potential for treating genetic diseases by allowing scientists to cut, replace, or delete mutations in DNA.

16 Jan 2025

Researchers Develop Method to Visualize Nanocarriers at Single-Cell Level

How can we ensure that life-saving drugs or genetic therapies reach their intended target cells without causing harmful side effects? Researchers at Helmholtz Munich, Ludwig-Maximilians-Universität (LMU) and Technical University Munich (TUM) have taken an important step to answer this question.

16 Jan 2025

New Gene Editing Therapy Shows Promise for Treating Stargardt Disease

Researchers have developed a highly efficient gene editing therapy that could potentially treat Stargardt disease, the most common form of inherited macular degeneration.

10 Jan 2025

AI-Designed Nanocages Mimic Intricate Structures of Viruses

Professor Sangmin Lee from POSTECH's Department of Chemical Engineering, in collaboration with 2024 Nobel Chemistry Laureate Professor David Baker from the University of Washington, has developed an innovative therapeutic platform by mimicking the intricate structures of viruses using artificial intelligence (AI).

26 Dec 2024

Targeting Cancer with Nanoparticle-Delivered Gene Therapy

Scientists are literally shining a laser on the energy centers of cancer cells in an attempt to harm these sources of power and cause widespread cancer cell death.

16 Dec 2024