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.

A Gene Mutation That Protects Against Disease

Researchers think they have found a fountain of youth, and it is unique to a few French Canadian families.

3 Dec 2020

New gene therapy can protect visual function in patients with dominant optic atrophy

Researchers have created a novel gene therapy approach that shows promise for someday treating an eye disease that results in progressive vision loss.

27 Nov 2020

Researchers use metal-organic frameworks to target prostate cancer cells

Scientists have used metal-organic frameworks to effectively deliver the CRISPR/Cas9 genetic snipping tool into human cancer cells.

23 Nov 2020

Researchers identify rare genetic marker that protects against cardiovascular disease

According to researchers from McMaster University and the Montreal Clinical Research Institute, they have identified a “fountain of youth” in a rare genetic marker that is unique to a few French-Canadian families.

20 Nov 2020

Somatic mutation acts as natural gene therapy in patients with GATA2 deficiency

Researchers affiliated with the Center for Cell-Based Therapy (CTC) in Ribeirão Preto, Brazil, have identified for the first time a non-hereditary mutation in blood cells from a patient with GATA2 deficiency, a rare autosomal disease caused by inherited mutations in the gene that encodes GATA-binding protein 2 (GATA2).

18 Nov 2020

Light-activated liposomes could help deliver CRISPR gene therapy

Light-activated liposomes could help to deliver CRISPR gene therapy - and the method could prove safer and more direct than current methods.

From The University of New South Wales (UNSW) 16 Nov 2020

New approach can prevent toxicity linked to neurological gene therapy

Scientists have deigned a new method to inhibit a toxicity observed in the sensory neurons of DRG following gene therapy to treat neurological diseases.

12 Nov 2020

Dietary induction of hepatocyte regeneration could help enhance gene repair in the liver

The thyroid hormone used to promote the proliferation of hepatocytes improved the efficiency of CRISPR/Cas9-mediated gene repair in the mouse liver.

11 Nov 2020

Research projects receive BTCA awards to develop new drugs

Five research projects with exceptional promise to deliver new life-changing and health-altering therapies have received the inaugural Blavatnik Therapeutics Challenge Awards (BTCA) at Harvard Medical School.

From Harvard Medical School 30 Oct 2020

New DNA-cutting tool could advance gene editing for novel treatments

Gene editing for the development of new treatments, and for studying disease as well as normal function in humans and other organisms, may advance more quickly with a new tool for cutting larger pieces of DNA out of a cell's genome, according to a new study by UC San Francisco scientists.

21 Oct 2020

Immunotherapy using CAR NKT cells shows promise for treating solid tumors

Natural killer T (NKT) cells, a type of immune cells known for their potent anti-cancer properties in murine tumor models, have been developed into a novel form of immunotherapy to treat patients with cancer.

14 Oct 2020

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