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.

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

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

McGill University researchers identify proteins that drive cancer stem cells. Targeting and supressing a particular protein called galectin1 could provide a more effective treatment for glioblastoma, in combination with radiation therapy.

31 Aug 2021

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

Aptamer Group Ltd and Bio-Works Technologies AB today announce that they have entered into an agreement to co-develop a new affinity resin.

From Aptamer Group 26 Aug 2021

Mission and money clash in nonprofit hospitals’ venture capital ambitions

Cone Health, a small not-for-profit health care network in North Carolina, spent several years developing a smartphone-based system called Wellsmith to help people manage their diabetes.

24 Aug 2021

Scientists develop a new way to deliver molecular therapies to cells

Researchers from MIT, the McGovern Institute for Brain Research at MIT, the Howard Hughes Medical Institute, and the Broad Institute of MIT and Harvard have developed a new way to deliver molecular therapies to cells.

19 Aug 2021

ERS Genomics and Cellular Engineering Technologies Enter CRISPR/Cas9 License Agreement

ERS Genomics Limited and Cellular Engineering Technologies today announced a non-exclusive license agreement.

17 Aug 2021

Study identifies mechanism of resistance and presents a much-needed target for advanced leukemias

Too many "exhausted" T cells left in the wake of aggressive chemotherapy regimens for patients with advanced chronic lymphocytic leukemia (CLL) make it more challenging for chimeric antigen receptor (CAR) T cell therapy to do its job.

16 Aug 2021

Newborn screening and early treatment for spinal muscular atrophy can save both lives and money

Gene therapy for spinal muscular atrophy might have a high up-front price tag. But by screening and treating infants early, the therapy can save both lives and money in the long term.

4 Aug 2021

Biolabs LA at The Lundquist Institute reaches highly significant capacity milestone

BioLabs LA at The Lundquist Institute, a co-working and incubator facility for high-potential bioscience start-ups, has reached 100% capacity ahead of schedule.

3 Aug 2021

Start codon disruption with CRISPR-Cas9 gene editing prevents Fuchs’ corneal dystrophy in mice

In a new study, researchers at the University of Oregon's Phil and Penny Knight Campus for Accelerating Scientific Impact used start codon disruption with CRISPR-Cas9 gene editing to prevent Fuchs' corneal dystrophy in mice.

3 Aug 2021

Gene therapy protects optic nerve cells and preserves vision in glaucoma mouse models

A form of gene therapy protects optic nerve cells and preserves vision in mouse models of glaucoma, according to research supported by NIH's National Eye Institute.

23 Jul 2021

Investigational Oncomagnetic Device shrinks end-stage recurrent glioblastoma

Houston Methodist Neurological Institute researchers from the department of neurosurgery shrunk a deadly glioblastoma tumor by more than a third using a helmet generating a noninvasive oscillating magnetic field that the patient wore on his head while administering the therapy in his own home.

22 Jul 2021