Gene Therapy News -- ScienceDaily

Research teams have created a versatile set of gene delivery systems that can reach different neural cell types in the human brain and spinal cord with exceptional accuracy. These delivery systems are a significant step toward future precise gene therapy to the brain that could safely control errant brain activity with high precision. In contrast, current therapies for brain disorders mostly trea…

Cancer treatment with a cell-based immunotherapy causes mild cognitive impairment, a Stanford Medicine team found. They also identified compounds that could treat it.

Modern HIV medicine is based on a common genetic mutation. Now, researchers have traced where and when the mutation arose -- and how it protected our ancestors from ancient diseases.

Many mitochondrial diseases have been difficult to study and treat due to the inherent challenges in accessing mitochondrial DNA (mtDNA). Now, researchers have optimized mitochondrial-targeted compounds that can selectively modify the ratio of normal versus mutant mtDNA in patient-derived stem cells. This technology enables the creation of research models with varying mutation loads and demonstra…

Historically, small molecule drugs have been precisely designed down to the atomic scale. Considering their relatively large complex structures, nanomedicines have lagged behind. Researchers argue this precise control should be applied to optimize new nanomedicines.

New research suggests that decreased activity of ELOVL2 -- a key enzyme in lipid metabolism -- accelerates white blood cell aging and alters genes associated with the onset of blood cancers.

Scientists have developed a new type of handheld multi-purpose radiation detector that comprehensively detects all types of ionizing radiation. The device can be used by industrial and medical radiation users, regulatory authorities, the nuclear energy industry, first responders and military users. The technology has been patented and is currently being explored for commercialization.

An international team of researchers has discovered a natural mechanism that protects the heart from heart failure with preserved ejection fraction (HFpEF), a serious condition in need of effective treatment. The team reports that when the cardioprotective mechanism fails, it promotes the development of HFpEF. Importantly, restoring the mechanism prevents the progression of the condition. The fin…

A research team has developed an advanced delivery system that transports gene-editing tools based on the CRISPR/Cas9 gene-editing system into living cells with significantly greater efficiency than before. Their technology, ENVLPE, uses engineered non-infectious virus-like particles to precisely correct defective genes -- demonstrated successfully in living mouse models that are blind due to a m…

Scientists have shown that a sequence of just three amino acids may reduce the severity of psoriasis, when applied topically in an emollient cream.

The new study reveals a remarkable way that cells keep us safe from transposable elements (TEs) gone wild. The researchers found that cells have taken advantage of an entire protein network to repress TE activity and keep themselves healthy.

A research team has developed a new system to estimate a person's biological age -- a measure of how well their body has aged, rather than just counting the years since birth. Using just five drops of blood, this new method analyzes 22 key steroids and their interactions to provide a more precise health assessment. The team's breakthrough study offers a potential step forward in personalized heal…

Investigators have found that the gene Asah1 plays a crucial protective role in preventing the progression of nonalcoholic fatty liver diseases (NAFLD) into more severe forms of liver disease by regulating hepatic lipid homeostasis and cellular maintenance processes. The findings have the potential to inform new therapeutic strategies and improve outcomes for NAFLD patients.