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 CRISPR-based tool can snip out faulty genes and replace them with new ones

Building on the CRISPR gene-editing system, MIT researchers have designed a new tool that can snip out faulty genes and replace them with new ones, in a safer and more efficient way.

24 Nov 2022

New family of adeno-associated viral vectors found to be better at delivering genetic cargo

Gene therapies can treat, even potentially cure, certain genetic diseases, but it is challenging to deliver the treatments to the parts of the body where they are needed.

22 Nov 2022

Rice bioengineer receives support for brain cancer research

Kevin McHugh, an assistant professor of bioengineering at Rice University's George R. Brown School of Engineering, has earned a Distinguished Scientist Award for 2022 from The Sontag Foundation, which helps launch the careers of scientists expected to make a significant impact in the fight against brain cancer.

21 Nov 2022

Scientists explore links between survival motor neuron protein and sarcopenia

Adopting some of the strategies behind successfully treating the childhood disease spinal muscular atrophy may enable development of therapies to curb the muscle decline that accompanies aging, new research suggests.

15 Nov 2022

IVI and Lemonex sign MOU to join forces on vaccine development

The International Vaccine Institute announced on November 10 it signed a memorandum of understanding (MOU) with Lemonex, a company specializing in RNA gene therapy development, and agreed to seek cooperation in research and development.

10 Nov 2022

Tissue nanotransfection shows promise as a treatment for traumatic muscle loss

Technology developed by researchers at the Indiana University School of Medicine that can change skin tissue into blood vessels and nerve cells has also shown promise as a treatment for traumatic muscle loss.

9 Nov 2022

What is the role of adenoviruses in human health?

With advances in molecular biology informing recent developments in vaccinology, gene therapy, and other fields of medicine, human adenoviruses (HAdVs) have been of primary importance in medical research. A recent review looks at the promises of HAdVs, their potential dangers, and future challenges.

9 Nov 2022

Charles River Laboratories: New Center Expands Cell and Gene Therapy Capabilities

In this interview, Niall Dinwoodie, Executive Director for Gene Therapy for Charles River in Europe discusses the opening of their new HQ Plasmid Center of Excellence at Alderley Park.

From Charles River Laboratories, Inc. 4 Nov 2022

New closed-loop gene therapy approach to treat brain circuit disorders

On-demand expression of a gene that inhibits neuronal activity provides a way to reduce spontaneous seizures in mice, researchers report.

3 Nov 2022

Brain Canada Foundation funds future leaders in Canadian brain research

Twenty early-career researchers from across Canada will receive $100,000 each in funding for innovative projects to better understand the brain, through one of Brain Canada's flagship programs Future Leaders in Canadian Brain Research.

28 Oct 2022

Researchers use CRISPR-edited T cells to treat seriously ill children with resistant leukaemia

Researchers at Great Ormond Street Hospital for Children (GOSH) and UCL Great Ormond Street Institute of Child Health (UCL GOS ICH) have used CRISPR/Cas9 technology to engineer donor T cells to try to treat seriously ill children with resistant leukaemia, who had otherwise exhausted all available therapies.

27 Oct 2022

Discovery points to a new understanding of Stargardt disease progression

Using a new stem-cell-based model made from skin cells, scientists found the first direct evidence that Stargardt-related ABCA4 gene mutations affect a layer of cells in the eye called the retinal pigment epithelium (RPE).

27 Oct 2022

Pioneering gene editing technique can repair faulty T cells in patients with CTLA-4 insufficiency

A fault in cells that form a key part of the immune system can be repaired with a pioneering gene editing technique, finds new research demonstrated in human cells and mice, led by UCL scientists.

27 Oct 2022

New studies point to a broadly applicable treatment for neurodegenerative diseases

Two new studies from Washington University School of Medicine in St. Louis support development of a broadly applicable treatment for neurodegenerative diseases that targets a molecule that serves as the central executioner in the death of axons, the wiring of the nervous system.

26 Oct 2022

UArizona researchers working on a less toxic treatment for deadly breast cancer

After decades of research and two previous drug development attempts, things are looking promising for a team of University of Arizona researchers working on a less toxic treatment for a specific type of breast cancer.

25 Oct 2022

New approach to immunotherapy provokes a robust anti-tumor immune response in preclinical models

A new approach to cancer immunotherapy that uses one type of immune cell to kill another-;rather than directly attacking the cancer-;provokes a robust anti-tumor immune response that shrinks ovarian, lung, and pancreatic tumors in preclinical disease models, according to researchers at the Icahn School of Medicine at Mount Sinai in New York.

25 Oct 2022

Celebrating the Alderley Park Grand Opening

HQ Plasmid Center of Excellence based at UK life science hotspot now open for business.

From Charles River Laboratories, Inc. 24 Oct 2022

New research could have major implications for minimally invasive, robotically delivered brain surgery

The early-stage research tested the delivery and safety of the new implantable catheter design in two sheep to determine its potential for use in diagnosing and treating diseases in the brain.

21 Oct 2022

Study shows how brain's sensitivity to regret may be altered in mood disorders

Researchers from the Icahn School of Medicine at Mount Sinai have learned that the way the brain processes the complex emotion of regret may be linked to an individual's ability to cope with stress, and altered in psychiatric disorders like depression.

19 Oct 2022

The Emerging Field of Gene Therapy with Adeno-Associated Viruses

In this interview, Arnaud Delobel, the R&D and Innovation Director at Quality Assistance talks to NewsMed-LS about the emerging field of gene therapy with adeno-associated viruses (AAV).

From Waters | Wyatt Technology 18 Oct 2022