Gene Therapy News and Research

RSS

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.

Detailed 3D structure provides a roadmap for tweaking CRISPR-Cas9–guided base editors

Within a mere eight years, CRISPR-Cas9 has become the go-to genome editor for both basic research and gene therapy.

31 Jul 2020

Boosting metabolic resources in the brain could be a way to slow neurodegeneration

Researchers at Vanderbilt University Medical Center have shown for the first time that when one optic nerve in the eye is damaged, as in glaucoma, the opposite optic nerve comes to the rescue by sharing its metabolic energy.

From Vanderbilt University 22 Jul 2020

New, promising CAR T cell therapy results in exceptional patient recovery

In a clinical trial evaluating a novel immunotherapy option for cancer treatment, a child with rhabdomyosarcoma, a form of muscle cancer, that had spread to the bone marrow, showed no detectable cancer following treatment with chimeric antigen receptor (CAR) T cells that were engineered to target the HER2 protein on the surface of cancerthe cells.

16 Jul 2020

Stem cell treatment could improve the quality of donor kidneys

New research has demonstrated that kidneys can be revived prior to transplantation by delivering a cell therapy directly to the organ.

From Newcastle University 15 Jul 2020

Nano-radiomics uses imaging data to assess changes in the tumor microenvironment

Research has shown that the tumor microenvironment (TME) can help cancers grow and evade the immune response.The TME has even been shown to inhibit cellular immunotherapy, a novel form of treatment in which the cells of a patient's immune system are re-engineered in the lab to attack cancer cells.

14 Jul 2020

Study may provide a therapeutically safer strategy for gene editing

The CRISPR gene editing system is a robust tool specifically developed for the targeted editing of genomes. While this tool has considerable therapeutic potential, it is associated with improper editing of “off-target” sites.

13 Jul 2020

Developments in Engineered T Cells for Type 1 Diabetes

AZoLifeSciences speaks to Professor David J. Rawlings from the Seattle children's hospital about recent developments in engineered T cells for type 1 diabetes.

23 Jun 2020

Researchers unlock a weakness in common type of cancer cells

Like any cells in the body, cancer cells need sugar - namely glucose - to fuel cell proliferation and growth. Cancer cells in particular metabolize glucose at a much higher rate than normal cells.

19 Jun 2020

Human stem cell models reveal neurodegeneration in glaucoma

Glaucoma, a serious eye condition responsible for causing vision loss, affects over three million Americans.

12 Jun 2020

New technique restores hearing in mice with a recessive genetic mutation

Using a new genetic engineering technique, known as base editing, researchers from Boston Children's Hospital and the Broad Institute of MIT and Harvard, have restored hearing in mice with a known recessive genetic mutation.

5 Jun 2020

Scientists study how brain cancer cells acquire radiation resistance

In a vertical climb to avoid collision with a towering mountain, a plane ejects cargo to gain altitude. Investigators at the University of Minnesota showed that cancer cells perform similar feats in escaping the killing effects of radiation.

18 May 2020

Gene therapy builds muscle mass, reduces fat in mice

Exercise and physical therapy often are recommended to help people who have arthritis. Both can strengthen muscle - a benefit that also can reduce joint pain.

12 May 2020

Foxg1 gene works like a molecular knob to modulate electrical activity of the neurons

It works like a very fine "molecular knob" able to modulate the electrical activity of the neurons of our cerebral cortex, crucial to the functioning of our brain.

8 May 2020

Entos and Precision NanoSystems to develop vaccines and therapeutics using NanoAssemblr GMP System

Entos Pharmaceuticals, a healthcare biotechnology company developing next generation nucleic acid medicines with their Fusogenix delivery platform announced today a partnership with Precision NanoSystems, Inc., (PNI), a global leader in enabling transformative nanomedicines, to produce clinical grade vaccines and therapeutics using PNI's NanoAssemblr GMP System.

7 May 2020

Advanced simulations show how to improve efficiency of gene therapy

Diseases with a genetic cause could, in theory, be treated by supplying a correct version of the faulty gene.

20 Apr 2020