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.

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

Artificial Intelligence Designs Novel DNA Elements for Precise Gene Control

Using artificial intelligence, scientists at Yale University, the Broad Institute of MIT and Harvard, and The Jackson Laboratory have created thousands of novel DNA switches that can precisely regulate a gene's expression in many cell types.

24 Oct 2024

Magnetic Control of Brain Cells: A Step Forward in Neuromodulation

The introduction of magnetogenetics provides a noninvasive technique to modulate brain circuits, paving the way for innovative therapies in neuromodulation.

18 Oct 2024

Study Expands Potential of Retrons for DNA Editing

Within bacterial cells, specialized immune systems known as retrons fend off viral attacks. But that's not all they can do.

18 Sep 2024

Exploring Retrotransposon-Derived DNA Zip Codes in Myeloma Cell Internalization

A new editorial was published in Oncoscience (Volume 11) on July 13, 2024, entitled, "Unveiling retrotransposon-derived DNA zip code for myeloma cell internalization."

12 Aug 2024

Scientists Develop AI-Powered Tool to Optimize Gene Delivery

Gene therapy could potentially cure genetic diseases but it remains a challenge to package and deliver new genes to specific cells safely and effectively. Existing methods of engineering one of the most commonly used gene-delivery vehicles, adeno-associated viruses (AAV), are often slow and inefficient.

9 Aug 2024

Noninvasive Ultrasound Method Enables Tracking of Gene Expression in Specific Brain Regions

The ability to alter or prevent the expression of faulty genes in the brain could be leveraged as a powerful therapeutic against neurodegenerative disease. However, the molecular underpinnings of the living brain are still largely inaccessible, hampering progress on such promising therapeutics.

9 Aug 2024

Innovative Nanosensing Method Promises Safer Gene Therapy with Improved AAV Vector Quality

Over the past few decades, there has been remarkable progress in genetic manipulation technologies, bringing us closer to the point where genes can be modified in vivo.

2 Aug 2024

New Gene Therapy Approach Improves Muscle Strength in DMD Mice

Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for people with Duchenne muscular dystrophy (DMD).

29 Jul 2024

Developing DNA Test to Eliminate Inherited Blindness in English Shepherd Dogs

Researchers at Cambridge have discovered the genetic mutation that causes English Shepherd Dogs to suffer from progressive retinal atrophy, an irreversible form of blindness.

22 Jul 2024

TRPC5 Gene Alterations: A Key Player in Obesity and Postpartum Depression

Researchers at Baylor College of Medicine, the University of Cambridge and collaborating institutions have discovered that alterations in the human gene TRPC5 cause obesity and postpartum depression.

3 Jul 2024