National Institute of Mental Health (NIMH)’s Post

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

Exciting news in the world of gene therapy! A new study by the Broad Institute has revealed a promising gene delivery vehicle that could revolutionize treatment for genetic brain disorders. The innovative adeno-associated virus, or AAV, has been engineered to efficiently cross the blood-brain barrier and target the human transferrin receptor. This approach has shown significantly higher brain penetration levels compared to FDA-approved AAVs, reaching crucial brain cells like neurons and astrocytes. Demonstrated in humanized mice, this groundbreaking development offers new hope for diseases such as Parkinson’s and Huntington’s. Imagine a future where genetic brain disorders are no longer life sentences! #GeneTherapy #BrainHealth #Innovation 🧬💡🧠🚀

🧬 A fascinating read on genetic research! If you’re interested in understanding how mutations in the IRF2BPL gene lead to a neurodevelopmental disorder called NEDAMSS, and how gene therapy can address this issue, this review article is a must-read. Dive into the intricate mechanisms and the latest findings in this area of study. Max Delbrück Center #Genetics #Neurodevelopment #NEDAMSS #IRF2BPL #Research

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

𝐄𝐧𝐠𝐢𝐧𝐞𝐞𝐫𝐞𝐝 𝐕𝐢𝐫𝐮𝐬 𝐂𝐫𝐨𝐬𝐬𝐞𝐬 𝐁𝐥𝐨𝐨𝐝-𝐁𝐫𝐚𝐢𝐧 𝐁𝐚𝐫𝐫𝐢𝐞𝐫 𝐭𝐨 𝐃𝐞𝐥𝐢𝐯𝐞𝐫 𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐲! 💉🧫Researchers have developed a gene therapy delivery system capable of effectively crossing the blood-brain barrier in mice. 🧪🧬This innovative approach utilizes a human protein to transport therapeutic genes into the brain, offering the potential for safer and more effective treatments for a range of brain conditions, including neurodevelopmental and neurodegenerative disorders. Learn more: https://lnkd.in/gSCu3deN

Delivering drugs to the brain has always been difficult because of the blood-brain barrier which is there to protect the brain from toxins and pathogens etc. This also makes drug delivery to the brain to treat brain diseases especially hard. Day 17 of celebrating good news in cell and gene therapies: Promising research by researchers from Broad Institute of MIT and Harvard that managed to engineer adeno-associated virus (AAV) that efficiently crosses the blood-brain barrier and delivers genes throughout the brain in humanized mice. If successful in human trials, this mechanism may benefit patients with genetic brain diseases (e.g. Huntington's, Alzheimer's, Tay-Sachs), all serious unmet medical needs. Congratulations on this important discovery!! Also - if anyone is going to be at ISCT, International Society for Cell & Gene Therapy Vancouver, please do reach out! Paolo Siciliano and I would love to catch up in person :) https://lnkd.in/dsdHQ4Un #CGT #genetherapies #drugdelivery #celebrate #monthofCGTcelebrations

Amazing results from a gene therapy study in children with Giant Axonal Neuropathy (GAN). Findings hint at possible regeneration of sensory neurons affected in a neurodegenerative disease from a treatment that appears to be safe and well tolerated. Kudos to not only the #NIHintramuralresearchers who participated in this work but to #HannahsHopeFund, the driving force that made this study possible. https://lnkd.in/eXWfAk2n #RareDiseaseResearch #genetherapy #CarstenBonnemann #PatientDrivenDrugDevelopment

Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. But FDA-approved forms of the most commonly used vehicle for packaging and delivering these therapies to target cells, adeno-associated viruses (AAVs), aren’t able to efficiently cross the blood-brain barrier and deliver cargo. Now, from the Ben Deverman Lab comes an important step toward gene therapy for brain diseases: the first published AAV that targets a human protein and reaches the brain in humanized mice. Because the vehicle binds to a well-studied human target, the scientists say it has a good chance at working in patients. #BroadInstitute #Science #ScienceNews #Research #ScientificResearch #AAV #BrainResearch

Angelman syndrome is a rare genetic disorder caused by mutations in the maternally-inherited UBE3A gene and characterized by poor muscle control, limited speech, epilepsy, and intellectual disabilities. Researchers have identified a small molecule that could be safe, non-invasively delivered, and capable of "turning on" the dormant paternally-inherited UBE3A gene copy brain-wide, which would lead to proper protein and cell function, amounting to a kind of gene therapy for individuals with Angelman syndrome. https://lnkd.in/gNa_Vth4.

I'm delighted to announce the publication of our latest work in the Genome Research journal. In this study, we achieved three significant milestones: 1) Engineered a groundbreaking endogenous Drosophila tauopathy model through CRISPR-Cas9 gene editing. 2) Utilized cutting-edge scRNA-seq analysis to demonstrate the conservation of tau function across evolutionary time (~600 million years onwards). 3) Uncovered novel mechanisms and potential therapeutic targets for Alzheimer's disease and related dementias. This research substantially advances our understanding of neurodegenerative disorders and offers promising avenues for future therapeutic development. "https://lnkd.in/e6jJi2rs