ASPIRE Precision Medicine Virtual Research Institute Abu Dhabi’s Post

Source: Molecular neurobiology This study investigates the metabolic dysregulations caused by blast-induced trauma in a preclinical animal model. Using advanced metabolomic and statistical bioinformatic platforms, the study identifies significant changes in metabolic markers at different time points post-injury. The study also explores pathway dysregulations and correlation networking, shedding light on the biological mechanisms of blast-induced brain or systemic trauma.

Modeling Lewy body disease with SNCA triplication iPSC-derived cortical organoids and identifying therapeutic drugs Researchers modeled Lewy body disease (LBD) using human-induced pluripotent stem cell-derived cortical organoids from patients with SNCA gene triplication. They identified synaptic and mitochondrial dysfunction in excitatory neurons with high SNCA expression. A drug screen revealed that entacapone, tolcapone, phenazopyridine hydrochloride, and zalcitabine, all FDA-approved compounds, inhibited α-SYN aggregation and alleviated mitochondrial dysfunction, suggesting potential therapeutic targets for LBD. The article was published in Science Advances: https://lnkd.in/eRSY2d9V. #genetics #genomics #precisionmedicine #genomicmedicine #brain #neurology #neuroscience #neurodegeneration #parkinson #lbd #metabolism #physiology #cardiovasculardisease #mitochondia #drugrepurposing #neurons #therapeutics #biotechnology #innovation #research #science #sciencecommunication

We are excited to share an article from Science Magazine confirming the power of Sangamo’s zinc fingers for editing in the brain, click here to view it, bit.ly/3W2Apd9. The authors demonstrate that Sangamo's previously published prion zinc fingers were superior to other platforms and enabled Neumann et al to test their new epigenetic editing tool. We are proud to have pioneered the zinc finger technology that continues to lead the field of epigenetic editing. These compact, flexible tools can precisely target DNA, enhancing our potential therapies for neurological diseases including prion disease. Our zinc finger-based prion program, ST-504, is advancing towards the clinic enabled by our proprietary STAC-BBB capsid. Learn more about our whole brain delivery solution by clicking on the links below. Whole brain delivery solution, https://bit.ly/3tH18AH STAC-504, https://bit.ly/3RLFpjI STAC-BBB, https://bit.ly/3xKCWiz #GenomicMedicine #neurology #zincfinger #prion

The sortilin-related receptor, SORL1, might play a pivotal role in fueling tau aggregation in glial cells. In a groundbreaking study led by Bradley Hyman and Dudley Strickland, scientists uncovered that SORL1 binds tau and co-localizes with it in neuroglioma cells. 💡 Moreover, manipulating SORL1 levels impacted tau seeding, shedding light on its potential involvement in Alzheimer's pathology. 🔍 Read the full article now on Alzforum and stay ahead of the curve in Alzheimer's research. - https://bit.ly/4ahHGtf #ADPD2025 #ADPD2024 #alzheimers #parkinsons #dementia #endalz #neuroscience #neuroscientist #researcher

Glad to share that our article "The memory ameliorating effects of novel N-benzyl pyridine-2-one derivatives on scopolamine-induced cognitive deficits in mice" is now available in the public domain. This study showcase the potentials of small heterocyclic moieties for the management of Alzheimer's disease. #research #neuroscience #alzheimersdisease #drugdevelopment Here is the free access link for the complete article https://lnkd.in/g5Batrsz

🔆 Five Biological Variants of Alzheimer’s Discovered 🧠 Researchers from Amsterdam UMC have identified five biological variants of #Alzheimer’s disease through cerebrospinal fluid analysis, shedding new light on the complexity of the condition. These variants differ in amyloid production, blood-brain barrier integrity, nerve cell growth, protein synthesis, and immune system functioning. 1. Alzheimer’s disease has five distinct biological variants, affecting #amyloid production, #blood-#brain barrier integrity, and less #cell #growth and #protein #synthesis. 2. Each subtype was associated with distinct AD #genetic risk factors, further supporting that each CSF AD subtype reflects specific underlying #molecular #mechanisms. 3. Tailored treatments based on these variants may enhance the effectiveness of Alzheimer’s drugs. 4. #Personalized #medicine could lead to improved treatment outcomes and reduced risk of side effects for Alzheimer’s patients. Nature Aging: Cerebrospinal fluid proteomics in patients with Alzheimer’s disease reveals five molecular subtypes with distinct genetic risk profiles

Scientists at LSU Health New Orleans’ Neuroscience Center of Excellence, led by Nicolas Bazan, MD, PhD, Boyd Professor and Director, have identified a new mechanism that regulates a protein key for cell survival. Researchers made a pivotal discovery in neuroprotection. They identified how Elovanoid-34, a molecule in the brain, modulates the protein TXNRD1 to combat oxidative stress, a precursor to neurodegenerative diseases. This breakthrough reveals that Elovanoid-34 can prevent cell death in conditions like Age-Related Macular Degeneration by regulating oxidative stress. The study emphasizes the potential of this discovery for developing new therapies for age-related diseases and promoting successful nervous system aging. Elovanoid-34, found in the brain, plays a crucial role in controlling the protein TXNRD1, which is essential in managing oxidative stress. This discovery offers potential new treatments for neurodegenerative diseases and conditions like Age-Related Macular Degeneration. The research, supported by the National Eye Institute and the Eye, Ear, Nose & Throat Foundation, opens new avenues for understanding aging and developing targeted therapeutics for various age-related diseases. https://lnkd.in/g7gYbzAP

A forward-genetics screen for GCase activity modulators identified Current therapeutic strategies targeting increased β-glucocerebrosidase (GCase) activity hold promise for Gaucher disease (GD) and Parkinson's disease (PD). Previously, we explored genetic modifiers of GCase activity https://lnkd.in/eeX9t-Uz. This study employs a complementary approach, utilizing a CRISPR activation library to identify modifying transcription factors (TFs) of GCase in the human glioblastoma cell line LN-229. These findings reveal 11 TFs as regulators of GCase activity. Ablation of USF2 resulted in increased GBA mRNA expression, leading to enhanced GCase protein levels and activity. This suggests USF2's role in repressing GBA transcription. Additionally, ONECUT2 was identified to control GCase trafficking through modulation of the guanine exchange factors PLEKHG4 and PLEKHG4B. This work significantly expands the understanding of the transcriptional landscape governing GBA regulation. By uncovering these novel regulatory factors, the study paves the way for potential therapeutic targets in GD and PD. The study is available as a Preprint: https://lnkd.in/eZpcwgJJ #genetics #genomics #precisionmedicine #genomicmedicine #brain #neurology #neuroscience #neurodegeneration #raredisease #gaucher #parkinson #biochemistry #therapeutics #biotechnology #innovation #research #science #sciencecommunication

Shares of UniQure rose more than 60% Tuesday morning after their AMT-130 treatment for Huntington’s disease showed significant dose-dependent deceleration of disease progression. AMT-130, a modified AAV5 viral vector containing an HTT exon1 targeting miRNA, slowed progression by 80% over two years with high doses, maintaining motor and cognitive function. Lower doses achieved a 30% slowdown. Additionally, neurofilament light chain levels, a biomarker for neurodegeneration, dropped by 11%. This progress underscores AMT-130's potential in combating Huntington’s by targeting toxic mutant huntingtin production. https://lnkd.in/eiBugcJn #GeneTherapy #AAV #HuntingtonsDisease #Neuroscience #BiotechInnovations