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Gene Therapy News

December 1, 2023

Top Headlines

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 site in the DNA of cancer cells that is essential ...
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 genetic mutations have been previously seen at higher levels in ...
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 ...
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 significant side effects. The approach has strong ...
Latest Headlines
updated 11:01pm EST

Earlier Headlines

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 ...

Research has found that misfolded proteins in liver cells contribute to the development of liver cancer, shedding new light on the mysterious origins of one of the world's deadliest diseases. ...

Technology that can change skin tissue into blood vessels and nerve cells also shows promise as a treatment for traumatic muscle ...

A new treatment for neurological and psychiatric diseases, that works by reducing the excitability of overactive brain cells, has been ...

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 ...

A new study identifies common and rare gene mutations that impact radiation resistance and sensitivity, an important step toward providing more individualized and effective radiotherapy for patients ...

Research led by doctors and scientists have identified a gene that may provide a therapeutic target for the deadly, treatment-resistant brain cancer glioblastoma multiforme ...

Adults with a genetic form of childhood-onset blindness experienced striking recoveries of night vision within days of receiving an experimental gene ...

The blood-brain barrier (BBB) is an imposing foe for gene therapy. Formed of cells wedged tightly together, the BBB keeps toxins and pathogens that may be present in the blood from entering brain ...

Researchers explain how precision genome editing agents have enabled precise gene correction and disease rescue in inherited retinal diseases ...

Researchers have developed a new gene therapy approach that shows promise for treating the dry form of Age Related Macular Degeneration (AMD) -- a progressive eye disease that affects up to 10% of ...

A new study in mice finds that a gene therapy appears to correct a rare creatine deficiency disorder that commonly results in intellectual disabilities, problems with speech, involuntary movements ...

Scientists reported on an experimental gene-therapy-like technique to restore the normal activity of the TCCF4 gene deficient in individuals with Pitt-Hopkins ...

Pancreatic cancer has a significantly poor prognosis; therefore, the development of effective treatments is an unmet clinical need. The major drawback in this field was the lack of useful model ...

Researchers have revealed how an 'accordion effect' is critical to switching off genes, in a study that transforms the fundamentals of what we know about gene silencing. The finding expands ...

A novel angiogenic microRNA drug can be a new option for the treatment of ischemic cardiovascular disease, according to a new study. In the study, the researchers describe a novel nuclear acting ...

The diagnosis is rare, but devastating -- children with congenital muscle disorders often never learn to walk. Until now, there was no chance of recovery, but researchers are now presenting a ...

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