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 use aerosolized molecules to shut down proteins generated by coronavirus

Researchers at Georgetown University Medical Center have successfully used molecules comprised of small strands of RNA to shut down the production of destructive proteins generated by the COVID-19 virus.

16 Nov 2020

New data highlights safety concerns on rAAV vectors used in gene therapy

New data highlight safety concerns for the replication of recombinant adeno-associated viral (rAAV) vectors commonly used in gene therapy.

16 Nov 2020

New method using light-activated liposomes is safer, more direct to 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.

12 Nov 2020

Pitt neuroscientists uncover additional complexities behind sensation of pain from light touch

Researchers from the Pittsburgh Center for Pain Research at the University of Pittsburgh School of Medicine announced today in the journal Neuron that they've uncovered additional complexities behind mechanical allodynia--the sensation of pain from innocuous stimuli, such as light touch.

11 Nov 2020

Dietary induction of hepatocyte regeneration may be a viable strategy to enhance gene repair

Use of thyroid hormone to boost hepatocyte proliferation enhanced the efficiency of CRISPR/Cas9-mediated gene correction in the mouse liver.

11 Nov 2020

Semi-automated approach accelerates artificial organoid growth and selection

The method currently used to produce stem cell-derived tissues has a very limited throughput. By semi-automating tissue differentiation, researchers from MIPT and Harvard have made the process nearly four times faster, without compromising on quality.

10 Nov 2020

Repairing brain damage caused by MS

Neurological symptoms as a result of Multiple Sclerosis (MS), such as loss of cognitive or motor function, lead to a great many problems for over 2.5 million people worldwide.

10 Nov 2020

PhoreMost and Oxford Biomedica collaborate to develop next-generation CAR-T cell therapies

PhoreMost Limited, the UK-based biopharmaceutical company dedicated to developing drugs against intractable disease targets, and Oxford Biomedica plc, a leading gene and cell therapy group, today announced that they have entered into a discovery collaboration to develop next-generation CAR-T cell therapies.

From PhoreMost 10 Nov 2020

Researchers to develop new optogenetic tools for biology and medicine

The European Research Council (ERC) is providing 10 million euros in funding for an interdisciplinary, collaborative project in the structural and biophysical analysis of selected photoreceptors and their development into "OptoGPCRs", light-controlled molecular switches with a wide range of applications in biology and medicine.

8 Nov 2020

Scientists use gene therapy to regenerate damaged optic nerve fibers

Scientists have used gene therapy to regenerate damaged nerve fibers in the eye, in a discovery that could aid the development of new treatments for glaucoma, one of the leading causes of blindness worldwide.

5 Nov 2020

Research projects receive BTCA grants to develop novel drugs for various diseases

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.

30 Oct 2020

Newly developed light-sensing protein restores vision in blind mice

A newly developed light-sensing protein called the MCO1 opsin restores vision in blind mice when attached to retina bipolar cells using gene therapy.

22 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

Study helps restore retinal and visual functions of mice models of inherited retinal disease

A breakthrough study, led by researchers from the University of California, Irvine, results in the restoration of retinal and visual functions of mice models suffering from inherited retinal disease.

20 Oct 2020

Tracing lineage to demonstrate direct astrocyte-to-neuron conversion in mouse brain

Regeneration of functional new neurons to repair injured human brain is a long-term unsolved problem up till today.

16 Oct 2020

CAR NKT cells offer a promising approach to combat hard-to-treat 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.

13 Oct 2020

Using mRNA therapies or CRISPR gene editing to treat cystic fibrosis

The potential for treating cystic fibrosis (CF) using mRNA therapies or CRISPR gene editing is possible regardless of the causative mutation. CF clinical trials showing that a genotype-agnostic gene therapy for CF is possible are reviewed in the peer-reviewed journal Human Gene Therapy.

8 Oct 2020

Biodegradable microneedle patch for local therapeutic gene delivery

There is great potential in gene therapy for treating certain types of cancer and genetic defects, immunological diseases, wounds and infections. The therapies work by delivering genes into the patients' cells, which then produce therapeutic proteins to treat the affliction.

7 Oct 2020

ACGT awards $500,000 grant to advance strategies for treating solid tumors

With a $500,000 Research Grant from Alliance for Cancer Gene Therapy, Sidi Chen, PhD, assistant professor in the Department of Genetics and Systems Biology Institute at Yale School of Medicine and member of Yale Cancer Center, will advance a versatile and highly scalable strategy he's been developing and calling MAEGI -- Multiplexed Activation of Endogenous Genes as an Immunotherapy.

6 Oct 2020

Researchers use single-cell sequencing to identify cell types that occur in pancreatic tumors

Led by the Translational Genomics Research Institute (TGen), an affiliate of City of Hope, and by HonorHealth Research and Innovation Institute, an international team of researchers have described in detail the individual cells that comprise the pancreatic cancer microenvironment, a critical step in devising new treatment options for patients with this aggressive and difficult-to-treat disease.

1 Oct 2020