Gene Therapy News and Research

RSS

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.

Drug developed at Duke is approved by FDA as only therapy for congenital athymia

The phone rang into the pediatric unit that Louise Markert, M.D., Ph.D., was staffing as a junior faculty member in 1991. Had she not answered, there would have been little hope for the more than one hundred patients who were subsequently treated.

11 Oct 2021

Study supports efficacy and safety of lung-selective nanotherapy against SARS-CoV-2 infection

A new preprint shows the potential role of a host protease cathepsin L inhibitor, which mediates viral entry into the host cell by its effect on the viral spike protein.

8 Oct 2021

Australian researchers hope to switch on sight with new gene therapy

Australian researchers are developing a new gene therapy which they hope will ‘switch on sight’ by regenerating light-sensing photoreceptor cells in the retina at the back of the eye.

7 Oct 2021

New understanding of cellular defects related to Cystic Fibrosis may pave the way for treatments

A team in the College of Medicine led by Drs. Juan Ianowski (PhD) and Julian Tam (MD) found that sodium transport is abnormal in lungs with CF.

6 Oct 2021

Researchers identify mechanism that can lead to deafness in rare Norrie disease

A team of Mass Eye and Ear Scientists led by Albert Edge, PhD, working with research fellow Yushi Hayashi, MD, PhD, has identified the mechanism that can lead to deafness in the rare syndrome, Norrie disease.

5 Oct 2021

Restoring visual function after stroke with gene therapy

Most strokes happen when an artery in the brain becomes blocked. Blood flow to the neural tissue stops, and those tissues typically die. Because of the locations of the major arteries in the brain, many strokes affect motor function.

3 Oct 2021

Gyros Protein Technologies introduces Gyrolab AAV9 Titer Kit to support AAV-based cell and gene therapy production

Gyros Protein Technologies AB today announced the introduction of Gyrolab® AAV9 Titer Kit for rapid determination of titer in AAV9 vector-based cell and gene therapy manufacturing.

From Gyros Protein Technologies AB 30 Sep 2021

Supercoiling and looping can transmit mechanical stress along the DNA backbone, research shows

When people think of DNA, they visualize a string-like double helix structure. In reality, the DNA double helix in cells is supercoiled and constrained into loops. This supercoiling and looping are known to influence every aspect of DNA activity, but how this happens has not been clear.

28 Sep 2021

Revival of the cell and gene therapy field opens new avenues for repairing the Parkinsonian brain

After several decades of pre-clinical development, cell- and gene-based therapies for Parkinson's disease (PD) are now actively being explored.

21 Sep 2021

Multi-million government fund given to 21 projects to propel innovation across the UK

Regulators are clearing the runway for drones to transport vaccines and other live-saving drugs, in a ground-breaking venture funded by government.

16 Sep 2021

Novel cell reprogramming offers a promising therapeutic strategy to slow down glioblastomas

Gliomas are the most common primary brain tumors in adults. Among them, high-grade glioblastomas (GBMs) are particularly known to be notoriously aggressive and invasive, which makes it challenging to treat them. The disease rapidly progresses to an advanced stage in a short span of time, and the survival of such patients is often dismal.

16 Sep 2021

Study pinpoints a potential pathway for treating epileptic seizures in patients with autism

Autism affects about 2% of children in the United States, and about 30% of these children have seizures. Recent large-scale genetic studies revealed that genetic variants in a sodium channel, called voltage-gated sodium channel Nav1.2, is a leading cause of autism.

15 Sep 2021

First rat model recapitulates all disabling alterations experienced by patients with Morquio A disease

A research team from the Universitat Autònoma de Barcelona has generated the first rat model recapitulating all disabling alterations experienced by patients with Mucopolysaccharidosis type IVA, also known as Morquio A disease.

12 Sep 2021

New gene-delivering workhorse could make gene therapy safer, more effective for muscle diseases

Genetic muscle diseases lead to progressive muscle wasting and often early death, with few treatment options and no cure.

9 Sep 2021

Researchers develop the smallest CRISPR to date for genome editing

A compact and efficient CRISPR-Cas system, named CasMINI, could be broadly useful for cell-engineering and gene-therapy applications because it is easier to deliver into cells. The findings appear in a study publishing September 3 in the journal Molecular Cell.

4 Sep 2021

Stanford researchers engineer an efficient, multi-purpose, mini CRISPR system

The common analogy for CRISPR gene editing is that it works like molecular scissors, cutting out select sections of DNA. Stanley Qi, assistant professor of bioengineering at Stanford University, likes that analogy, but he thinks it's time to reimagine CRISPR as a Swiss Army knife.

4 Sep 2021

Gene Therapy News and Research

Further Reading

Drug developed at Duke is approved by FDA as only therapy for congenital athymia

Study supports efficacy and safety of lung-selective nanotherapy against SARS-CoV-2 infection

Australian researchers hope to switch on sight with new gene therapy

New understanding of cellular defects related to Cystic Fibrosis may pave the way for treatments

Researchers identify mechanism that can lead to deafness in rare Norrie disease

Restoring visual function after stroke with gene therapy

Gyros Protein Technologies introduces Gyrolab AAV9 Titer Kit to support AAV-based cell and gene therapy production

Supercoiling and looping can transmit mechanical stress along the DNA backbone, research shows

Revival of the cell and gene therapy field opens new avenues for repairing the Parkinsonian brain

Multi-million government fund given to 21 projects to propel innovation across the UK

Novel cell reprogramming offers a promising therapeutic strategy to slow down glioblastomas

Study pinpoints a potential pathway for treating epileptic seizures in patients with autism

First rat model recapitulates all disabling alterations experienced by patients with Morquio A disease

New gene-delivering workhorse could make gene therapy safer, more effective for muscle diseases

Researchers develop the smallest CRISPR to date for genome editing

Stanford researchers engineer an efficient, multi-purpose, mini CRISPR system

Targeting and inhibiting a protein could provide more effective treatment for brain cancer

Aptamer Group and Bio-Works Technologies partner to enable improved solutions for gene therapy manufacturing

Mission and money clash in nonprofit hospitals’ venture capital ambitions

Scientists develop a new way to deliver molecular therapies to cells

Industry Focus eBook - Cell & Gene Therapy (1st Edition) eBook

The Critical Role of Sample Storage in Drug Discovery and Development

Advancing Neuroscience Research Through Collaboration and Patient-Centered Innovation

The impact of vibration on microscopy

Latest News