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.

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

New Class of CRISPR-Cas Enzymes for Precise Genome Editing

Recent research published in the journal “Science Bulletin” detailed the creation of a tool called ChCas12b that can accurately edit genomes and identify single-base mutations.

9 Dec 2024

Discovery of START Domain Reveals Secrets of Plant Development

Within all complex, multicellular living systems such as plants and humans, there exists a set of genetic elements that can be likened to the blueprints, tools, and specialized personnel at a construction site for an expanding development.

6 Dec 2024

New Light-Driven Biohybrid System Supports Precise Photoimmunotherapy

In a study published in Cell Reports Physical Science on Nov. 25, a research team led by Prof. CAI Lintao from the Shenzhen Institutes of Advanced Technology (SIAT) of the Chinese Academy of Sciences reported its development of an innovative intelligent light-guided biohybrid system, the CTPA/siCSF1R system, to target tumor-associated macrophages (TAMs), thus enabling precise spatiotemporal siRNA delivery.

28 Nov 2024

London Ranked Top City in Europe for Life Sciences

A newly released report has named London the #1 city in Europe and #3 globally for life sciences, behind Boston and New York, and ahead of Amsterdam, Munich, Paris, San Francisco, and Singapore.

18 Nov 2024

New Strategy Reinvigorates Exhausted T Cells to Fight Cancer

As cancer cells grow, they pump out metabolic byproducts such as lactic acid into the tumor microenvironment. Exhausted T cells -; which have lost their cancer-fighting oomph -; consume this lactic acid, which further saps their energy, according to new research from the University of Pittsburgh and UPMC Hillman Cancer Center.

11 Nov 2024