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Gene Therapy News

May 27, 2024

Top Headlines


In an effort to improve delivery of costly medical treatments, a team of researchers in electrical engineering has developed a stimulating method that could make the human body more receptive to ...
In a proof-of-concept study, researchers demonstrated the effectiveness of a potential new therapy for Timothy syndrome, an often life-threatening and rare genetic disorder that affects a wide range ...
A recent breakthrough study has shown potential to improve therapeutic outcomes for patients suffering from lung ...
New research sheds light on the significance of the glucocorticoid receptor in drug-resistant prostate cancer, showing that the development of drug resistance could be prevented by limiting the activity of coregulator ...

Latest Headlines

updated 9:18am EDT

Earlier Headlines


A previously mysterious small RNA molecule in mice is found to play a crucial role in gene expression, and may be the first identified member of a new class of regulatory ...

Researchers have laid the foundation for the development of a gene therapy for the genetic heart disease arrhythmogenic cardiomyopathy (ACM). Their approach, based on replacement of the PKP2 gene, ...

Leukemia is the most common type of cancer in children. Treatment involves intensive chemotherapy, which has severe side effects due to its non-specific mode of action. A team has now discovered a ...

Researchers have discovered that 'cell competition' following gene therapy results in the accumulation of stem cells with genetic mutations which make them grow faster. Some of these ...

Many hidden genetic variations can be detected with Chameleolyser, a new method. The information is already yielding new patient diagnoses and may also lead to the discovery of as yet unknown disease ...

Individuals living with severe sickle cell disease (SCD) are highly interested in new, potentially curative gene therapy treatments and are willing to accept associated risks for a chance at a cure, ...

One problem in gene therapy is that not all genes transfer equally well into the target cells. Researchers have now developed a flexible method to transfer large genes efficiently and without ...

A clinical trial has tested a potentially curative stem cell gene therapy for sickle cell disease. The results were ...

Researchers have developed a new form of gene therapy that can stop cell division in fusion-driven ...

A change in just one letter in the code that makes up a cancer-causing gene can significantly affect how aggressive a tumor is or how well a patient with cancer responds to a particular therapy. A ...

Researchers have made a remarkable step forward in finding a potential cure for a type of childhood kidney ...

New research has successfully reversed hearing loss in mice. Scientists used a genetic approach to fix deafness in mice, restoring their hearing abilities in low and middle frequency ...

A study shows new steps toward more patients getting gene ...

In a step forward in the development of genetic medicines, researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the ...

Cancer therapies that target specific genetic abnormalities in tumors have revolutionized treatment possibilities over the past two decades. While quality of life and survival are improved with ...

A scientific team has designed a strategy to fight obesity and diabetes in mice through ex vivo gene therapy which consists of implanting cells that have been manipulated and transformed in order to ...

Researchers uncover a mechanism in a retinal transporter protein that causes blindness, could lead to targeted ...

Researchers used the drug fasudil to restore neurons and improve methamphetamine-induced cognitive dysfunction in a mouse model of ...

Researchers have uncovered a gene that is overexpressed in mutated IDH1. Studies in human cells and a novel mouse model both show that this gene, called ZMYND8, plays a critical role in the radiation ...

Scientists have demonstrated in animal models the possibility of using lipid nanoparticles and messenger RNA, the technology underpinning COVID-19 vaccines, to treat blindness associated with a rare ...

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