A 2026 twitcher-mouse study found that adenine base editing corrected only about 0.5% of genomic DNA and 5% of mRNA in a Krabbe disease model, yet that low editing signal restored GALC enzyme activity to about 5% of wild-type levels and reduced psychosine by about 47%.1 This mouse experiment shows why even small correction fractions …
A 2026 Nature Communications study found that Mecp2 overexpression deregulated approximately 5,000 genes in mouse neural progenitor cells but only approximately 500 mostly small-change genes in mature neurons, with the same progenitor-vs-neuron split reproduced in human iPSC-derived cells.1 For Rett syndrome gene therapy, that is the useful calibration: extra MeCP2 is most dangerous when it …
Parkinson’s disease (PD), a degenerative disorder that predominantly affects the motor system, has long been a challenging condition to manage due to its complex pathology and the limited efficacy of available treatments. However, recent research presents a beacon of hope through a novel combined therapy targeting the core of Parkinson’s disease’s neuronal degradation. This innovative …
Epilepsy affects over 50 million people worldwide, making it one of the most common neurological disorders globally. For many living with epilepsy, achieving reliable seizure control remains a significant challenge, underscoring the need for continued innovation in treatment approaches. Exciting new developments in the field are providing clinicians with an expanding array of therapeutic options …
Despite the fact that pharmaceutical companies continue to promote the concept of “chemical imbalances” as the root cause of depression, this is a vast oversimplification of an infinitely more complex condition. While a chemical imbalance may be one of the primary contributing factors to a person’s depression, what causes the chemicals (neurotransmitters) to become imbalanced …
When it comes to depression, most people are looking for a treatment that allows them to feel happier, while minimizing potential side effects. There are many antidepressants on the market, and while some may work slightly better than others, most research indicates that the efficacy among approved drugs is very similar. If you are looking …