CURE Epilepsy的动态

An excellent gene therapy paper by Qiu et al. wherein the authors develop a new way of treating epilepsy. They use adeno-associated viruses (AAVs) to deliver a gene encoding an ion channel which dampens neuronal activity. This gene was placed under control of a promoter that turns on only when neuronal activity is excessive. So, when epileptic neuronal activity begins, the gene turns on and the ion channel suppresses the problem. A clever and exciting new approach for combatting brain disease! #epilepsy #brain #genetherapy #neurotech https://lnkd.in/gC7F7WGt

new gene therapy has restored vision in patients with Leber congenital amaurosis type I (LCA1)

Amid the business headlines and regulatory milestones, it’s easy to overlook the amazing creativity of the science at the heart of biotech. This recent study highlights the power of gene therapy to reduce seizures and improve cognitive and motor function in mice with severe epilepsy. It’s a reminder of the groundbreaking research happening every day to tackle diseases with limited treatment options. Let’s take a moment to celebrate the science that inspires us. What recent discoveries have caught your attention? #Biotech #GeneTherapy #Innovation #Science

The article discusses the advances in gene therapy for neurogenetic diseases, which are neurological conditions with a genetic cause. It highlights various gene therapy strategies, including viral vector delivery, antisense oligonucleotides, gene editing, RNA interference, and viroid delivery techniques, that have shown promising therapeutic effects in treating neurogenetic disorders. The review covers examples of gene therapy approaches used for conditions like Duchenne muscular dystrophy, spinal muscular atrophy, Huntington's disease, and amyotrophic lateral sclerosis. #clinicalgenetics #clinicalgenomics #genomicmedicine #humangenetics #medicalgenetics #neurogenetics #neurology

Recent advancements in gene therapy have shown promise in alleviating chronic pain by targeting pain receptors. Researchers have developed a chemogenetic system that reduces pain by interfering with nociceptors in the peripheral nervous system. This system, tested in mouse models, uses a modified receptor activated by an inert compound to diminish pain transmission. The potential application of this technology through gene therapy could offer a novel approach to managing chronic pain. While still in early stages, this innovation represents a significant step forward in pain management and the broader understanding of the peripheral nervous system.

#Neurogeneticdiseases are genetic #neurologicalconditions. #Genetherapy advancements, including viral vectors, gene editing, and RNA interference, show promise in treating these incurable disorders. A review of gene therapy strategies used in the treatment of neurogenetic diseases can be found in this article, which highlights a sampling of advances in #genetherapies for #neurogeneticdisorders, including ASO therapy, gene editing, gene augmentation, and RNA interference. #molecularmedicine #neurogenetics #neurology #precisionmedicine

Unlocking the power of dual therapy! A groundbreaking study sheds light on using gene therapy in combination with #risdiplam to manage #spinal #muscular #atrophy in children. Learn more on this here: https://bit.ly/3L7ddE1 #Ultrasound

Many questions to ponder in reading this news... New Gene Delivery Method Paves the Way for Advanced Brain Therapies A new study, funded by the National Institute of Mental Health and the National Institute of Neurological Disorders and Stroke, describes a promising way to carry genetic material into the brain to reach cellular targets. The researchers developed a delivery vehicle that can effectively transport genes across the blood-brain barrier by latching onto a human protein and distributing its genetic cargo to cells in the brain. Although the researchers have not yet tested the approach in people, this study brings us closer to making many life-saving treatments a reality. https://lnkd.in/gb9uEEAX

Great news for those with Retinitis Pigmentosa: A new gene therapy offers hope for all types of retinal degeneration. This innovative treatment uses a harmless virus to implant light-sensitive molecules into healthy retinal cells. https://lnkd.in/gPpCpZEM #retinaldegeneration, #genetherapy #RP

This review by Dr. Rrita Daci and myself lays out a framework to think about the efficacy, safety, and challenges with using AAV as a gene therapy vector for neurological disorders.