Assessment of Cannabidiolic Acid’s Neuroprotective Potential in a TDP-43 Transgenic Mouse Model of Amyotrophic Lateral Sclerosis

Cannabinoids originating from plants, such as Δ9-tetrahydrocannabinol (Δ9-THC), cannabinol, and formulations resembling Sativex, have demonstrated neuroprotective properties in various preclinical models of amyotrophic lateral sclerosis (ALS). Nevertheless, the therapeutic potential of lesser-known phytocannabinoids, including cannabidiolic acid (CBDA), has not been extensively investigated. The present investigation examined the neuroprotective capabilities of CBDA, alongside cannabidivarin, cannabidiol (CBD),…

Byneuroots.coDecember 9, 20250Comments

Clinical Trials Neuroscience Science & Research

Regulatory Developments in Gene Therapy for Huntington’s Disease: Perspectives from uniQure’s Engagement with the U.S. Food and Drug Administration

uniQure N.V., a prominent entity in the gene therapy domain focused on developing innovative interventions for individuals with profound medical conditions, recently disclosed outcomes from a pre-Biologics License Application (BLA) interaction with the U.S. Food and Drug Administration (FDA) concerning AMT-130, an experimental adeno-associated virus-based gene therapy targeting Huntington’s disease (HD). This neurodegenerative disorder, characterized…

Byneuroots.coNovember 28, 20250Comments

Neuroscience

Glial Heterogeneity in Ischemic Stroke: Insights from Single-Nucleus RNA Sequencing

In recognition of outstanding contributions to neuroscience, the “Researcher of the Month” jury at the Medical University of Vienna has awarded the November 2025 distinction to Dr. Daniel Bormann for his publication in the high-impact journal Nature Communications (impact factor 15.7), entitled “Single-nucleus RNA sequencing reveals glial cell type-specific responses to ischemic stroke in male…

Byneuroots.coNovember 28, 20250Comments

Neuroscience

NF-κB Orchestrates a Dual Transcriptional Program in Hypoxia with Predominant Control over Gene Repression

Hypoxia triggers conserved adaptive cellular programmes that restore oxygen homeostasis or mitigate its deficit. Central to the oxygen-sensing machinery are the hypoxia-inducible factors (HIFs), whose stability and activity are governed by prolyl hydroxylase domain enzymes (PHDs) and the von Hippel–Lindau ubiquitin ligase complex (Kaelin and Ratcliffe, 2008; Kenneth and Rocha, 2008). In parallel, hypoxia elicits…

Byneuroots.coNovember 27, 20250Comments

BNM MND Neuroscience

Bypassing Pluripotency: Scalable Direct Reprogramming of Dermal Fibroblasts into Functional Motor Neurons for Regenerative Therapy

Researchers at the Massachusetts Institute of Technology have developed a highly efficient protocol for the direct conversion of dermal fibroblasts into mature motor neurons, circumventing the intermediate pluripotent stem cell stage traditionally required for neuronal differentiation. This lineage reprogramming approach yields more than ten motor neurons per starting fibroblast in murine cells and achieves successful…

Byneuroots.coNovember 27, 20250Comments

Neuroscience

Nanostructured Particles Facilitate Mitochondrial Augmentation and Intercellular Transfer to Restore Cellular Energetics in Senescent and Pathological States

In a seminal investigation conducted by researchers in the Department of Biomedical Engineering at Texas A&M University, a novel biomaterial-based strategy has been elucidated to counteract the progressive diminution of mitochondrial density and consequent bioenergetic deficits in human cells. Led by Akhilesh K. Gaharwar and doctoral candidate John Soukar, this approach leverages engineered nanoflowers—microscale, petal-like…

Byneuroots.coNovember 26, 20250Comments

Neuroscience

Chimeric Antigen Receptor T-Cell Interventions Achieve Sustained Remission in Refractory Autoimmune Conditions

Chimeric antigen receptor (CAR) T-cell therapies, initially developed for oncological applications, have demonstrated substantial therapeutic potential in modulating aberrant immune responses underlying autoimmune pathologies, including ulcerative colitis, rheumatoid arthritis, and systemic lupus erythematosus. In a recent investigation, Bing Du, an immunologist affiliated with East China Normal University in Shanghai, alongside collaborators, reported outcomes from a…

Byneuroots.coNovember 26, 20250Comments

Neuroscience Science & Research

FDA Approves Itvisma®: A Landmark Gene Therapy for SMA

In a pivotal advancement for rare disease treatment, the U.S. Food and Drug Administration (FDA) has approved Itvisma® (onasemnogene abeparvovec-brve), developed by Novartis, as the first and only gene replacement therapy for children aged 2 years and older, teens, and adults with spinal muscular atrophy (SMA). Announced on November 24, 2025, this approval expands access…

Byneuroots.coNovember 26, 20250Comments

Neuroscience

From Lab to Brain: Why CRISPR Works Differently in Postmitotic Neurons

Ever wondered why CRISPR gene editing works great in lab cells but faces hurdles in the brain? A groundbreaking study from Nature Communications reveals key insights into DNA repair in postmitotic neurons—non-dividing cells like those in our brains that must endure lifelong damage. 🔬 Key Findings: Using virus-like particles (VLPs) to deliver Cas9, researchers compared…

Byneuroots.coNovember 26, 20250Comments

Epigenetics Neuroscience Science & Research

A Historic Milestone in Personalized Genetic Medicine: Israel’s First Individualized RNA Therapy for a Rare Neurodevelopmental Disorder

In a groundbreaking achievement for precision medicine in Israel — and one of the very few such cases worldwide — a multidisciplinary team at Sheba Medical Center has developed and administered a fully personalized RNA-based therapy for an 8-year-old girl named Ayla, who suffers from a rare and devastating GNAO1-related neurodevelopmental disorder. The treatment,…

Byneuroots.coNovember 26, 20250Comments