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.

Newly developed biodegradable microneedles deliver genes into the skin

Gene therapy offers an excellent potential for treating specific types of genetic defects and cancer, immunological diseases, infections, and wounds.

9 Oct 2020

Microneedles For Therapeutic Gene Delivery

There is great potential in gene therapy for treating certain types of cancer and genetic defects, immunological diseases, wounds and infections.

7 Oct 2020

Researchers discover novel genes for human deafness in Israeli families

Until now, seven genes were known to be involved in hearing loss in Israel's Jewish population. A new study led by Zippora Brownstein, PhD, and Prof. Karen Avraham from the Sackler Faculty of Medicine at Tel Aviv University has found that 32 genes are responsible for inherited hearing loss in Israeli Jewish families.

24 Sep 2020

Gene therapy overcomes cardiac effects in mouse model of Freidreich’s ataxia

Gene therapy was successfully used to overcome the cardiac effects of Freidreich’s ataxia (FA) in a mouse model of the disease, as reported in the peer-reviewed journal Human Gene Therapy. Click here to read the full-text article free online through October 18, 2020.

21 Sep 2020

Researchers compare developed carriers for delivery of genome editing tools

Scientists made a comparison of their developed carriers for delivery of genome editing (GE) tools with other available analogs.

18 Sep 2020

CRISPR in a Box: New educational gene editing tool kit

Introducing CRISPR in a Box™ - it's not a new crunchy cereal or the latest snack - it's an educational tool kit consisting of state-of-the-science gene editing technology that could be coming to a school near you.

10 Sep 2020

Researchers develop new gene therapy for congenital blindness

Retinitis pigmentosa is the most prevalent form of congenital blindness. Using a retinitis pigmentosa mouse model, researchers from Ludwig-Maximilians Universitaet (LMU) in Munich have now shown that targeted activation of genes of similar function can compensate for the primary defect.

3 Sep 2020

AF4-MALS: A powerful tool to separate viruses and aggregates

Postnova Analytics has published an applications report that presents data from a collaboration with the Analytical Development group at Biogen Inc. on separation of a virus mixture using Asymmetrical Flow Field-Flow Fractionation and measurement of their radius of gyration.

From Postnova Analytics 27 Aug 2020

Study looks into safety and early outcomes of gene therapy for spinal muscular atrophy

In May 2019, the U.S. Food and Drug Administration (FDA) approved a gene replacement therapy for the inherited, progressive neuromuscular disease 5q-linked spinal muscular atrophy (SMA).

26 Aug 2020

Eliminating tumors with engineered ‘off-the-shelf’ therapeutic T cells

Personalized cancer treatments are no longer just options of the future. In the past few years, researchers have made significant progress in 'teaching' the body's immune T cells to recognize and kill specific cancer cells, and human clinical trials have shown that this approach can successfully eliminate tumors

21 Aug 2020

Gene therapy to the inner retina prevents blindness in Cln3-deficient mice

Gene therapy to the inner retina prevented blindness in a mouse model of the neuro-degenerative disorder CLN3 Batten disease.

11 Aug 2020

West Virginia University performs revolutionary Alzheimer's treatment

A hospital nurse for 33 years, Nanette Miller would call her husband Frank at the end of each shift to let him know she was coming home.

10 Aug 2020

Identifying a new genetic cause of inherited neuropathy

Inherited mutations in a gene that keeps nerve cells intact were shown, for the first time, to be a driver of neuropathy known as Charcot-Marie-Tooth disease.

4 Aug 2020

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