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.

Scientists use gold nanoparticles to edit genes tied to HIV, inherited blood disorders

Scientists at Fred Hutchinson Cancer Research Center took a step toward making gene therapy more practical by simplifying the way gene-editing instructions are delivered to cells.

28 May 2019

Study demonstrates the concept of systemic gene therapy treatment for gamma-sarcoglycanopathy

Gamma-sarcoglycanopathy is one of the most common limb-girdle myopathies.

25 May 2019

Newly approved gene therapy for SMA is based on delivery vehicle discovered by Penn expert

James Wilson, MD, PhD, recalls being struck by the devastating toll of rare diseases as a young physician in the 1980s. He set out on a path to correct the genes that cause these conditions, including spinal muscle atrophy (SMA), the most common inherited fatal disease in infants.

25 May 2019

Children's Tumor Foundation establishes significant research initiative to combat NF2

On the occasion of World NF2 Awareness Day, the Children's Tumor Foundation has announced the establishment of a significant research initiative dedicated to finding effective treatments for NF2, along with a substantial investment of $2.3 million in this bold new effort.

23 May 2019

Newly discovered molecules safely halt the gene editing process

Scientists have discovered two non-toxic molecules that safely halt the CRISPR-Cas9 gene editing process.

22 May 2019

WVU researcher studies stubborn blood disease that strikes children

Treating a stubborn blood disease that strikes children may come down to tweaking energy production in stem cells, suggests research out of West Virginia University.

14 May 2019

Researchers develop stem cell-based model to study neuron resilience and vulnerability in ALS

Researchers at Karolinska Institutet in Sweden have developed a stem cell-based model in order to study the resilience and vulnerability of neurons in the neurodegenerative disease ALS.

9 May 2019

CU Anschutz researchers identify new immunodeficiency disease

A new immunodeficiency disease caused by a novel genetic mutation has been identified by researchers at the University of Colorado Anschutz Medical Campus providing unique insights into cell biology.

6 May 2019

Discovery of small-molecule inhibitors could enable precise control over CRISPR-Cas9 genome editing

The discovery of the first small-molecule inhibitors of the Streptococcus pyogenes Cas9 (SpCas9) protein could enable more precise control over CRISPR-Cas9-based genome editing, researchers report May 2 in the journal Cell.

3 May 2019

Sartorius Stedim Biotech introduces new ambr 15 cell culture microbioreactor system

Sartorius Stedim Biotech, a leading international partner of the biopharmaceutical industry, is introducing its new generation of ambr 15 cell culture automated microbioreactor system.

From Sartorius 3 May 2019

Potential stem cell therapies moving from lab bench to clinical application

Stem cell research is leading to potential new therapies to treat disease, with several applications in clinical trials or expected to enter trials in the coming months.

3 May 2019

Will gene-editing of human embryos ever be justifiable?

World-leading medical ethicist Professor Arthur Caplan has published a series of recommendations with the aim of addressing worldwide concerns over gene editing of human embryos.

30 Apr 2019

Gene repair improves seizure and memory in adult mouse models of autism

A new study challenges the presumption that people born with developmental brain disorders such as severe autism will benefit from medical interventions only if treated during a narrow window in infancy or early childhood.

27 Apr 2019

UT Southwestern researchers determine how circulating 'bad cholesterol' enters artery walls

UT Southwestern researchers have determined how circulating "bad cholesterol" enters artery walls to cause the plaque that narrows the blood vessels and leads to heart attacks and strokes.

25 Apr 2019

Yposkesi chairman to speak on ‘Manufacturing and the CDMO Perspective’ at Cell and Gene Meeting

Yposkesi, a leading CDMO for gene therapy viral vector manufacturing, today announces that its chairman Frederic Revah Ph.D. will participate as a panelist on the topic of ‘Manufacturing and the CDMO Perspective’.

23 Apr 2019

Reviving the heart's regenerative capacities using microRNAs

Once the heart is fully formed, the cells that make up heart muscle, known as cardiomyocytes, have very limited ability to reproduce themselves. After a heart attack, cardiomyocytes die off; unable to make new ones, the heart instead forms scar tissue. Over time, this can set people up for heart failure.

23 Apr 2019

Study compares effectiveness and cost of gene therapy and HSCT in major beta-thalassemia

Gene therapy offers the promise of a cure for beta-thalassemia and a new study has shown that it is associated with fewer complications and hospital admissions over 2 years than treatment by allogeneic hematopoietic stem cell transplantation

18 Apr 2019

New gene therapy cures babies with fatal 'Bubble Boy' disease

A new gene therapy created by US researchers appears to have cured eight infant boys born with a rare genetic disorder referred to as “Bubble Boy” disease.

18 Apr 2019

Researchers identify cause of inherited metabolic disorder

A new study from BC Children's Hospital, the University of British Columbia (UBC) and an international team of researchers published in the New England Journal of Medicine is the first to identify a rarely-seen type of DNA mutation as the cause of an inherited metabolic disorder.

16 Apr 2019

Key gene could decrease detrimental brain swelling after stroke

Could a medication someday help the brain heal itself after a stroke, or even prevent damage following a blow to the head? A new USC study lends support to the idea.

15 Apr 2019