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Gene Therapy for Parkinson’s Disease Associated with GBA1 Mutations

Abeliovich et al. make a compelling case for the promise of using gene therapy to treat Parkinson’s disease (PD) patients who possess mutations in the GBA1 gene. People interested in the clinical-translational side of biomedicine should definitely check this out!


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Dyno Therapeutics Launches Two New AAV Capsids and AI Platform for Rare Disease Therapeutic Development at the 2026 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting

Dyno continues to develop impressive new AAV capsids with their AI-guided design approach!


About Dyno Therapeutics.

Dyno Therapeutics is on a mission to build high-performance genetic technologies that transform patients’ lives. Dyno applies AI to build technologies for gene delivery and sequence design that advance “Genetic Agency” — an individual’s ability to take action at the genetic level to live a healthier life — through safe, effective and widely accessible genetic treatments. With frontier AI models and high-throughput in vivo experimentation, Dyno designs optimized AAV delivery vectors that solve gene delivery challenges across a wide range of therapeutic applications including eye, muscle and CNS. Dyno partners across industries to ensure these life-transforming technologies can help as many patients as possible, including through strategic collaborations with leading gene therapy developers Astellas and Roche and with technology companies including NVIDIA. Dyno’s AI-designed capsids are available for direct licensing and through the Dyno Frontiers Network. Visit www.dynotx.com for more information.

‘Dyno Therapeutics’, ‘dyno’, the Dyno logo, and mountain logo are registered trademarks of Dyno Therapeutics, Inc. All rights reserved.

A fresh approach to peppermint: 250 new variants could boost flavor and fight disease

The genomics of peppermint are not as fresh as their flavor but scientists from the University of California, Davis, have found a way to breathe new genetic variation into the species. The findings, published in the Proceedings of the National Academy of Sciences, could help the mint industry develop new varieties of peppermint and provide a roadmap for improving clonal crops more generally.

Similar to strawberries, potatoes and many fruit trees, peppermint plants (Mentha × piperita) are reproduced asexually by a process called clonal propagation. In the case of peppermint, this means that their genomes have remained unaltered for more than 200 years. This lack of genetic variation leaves them susceptible to disease and means that properties such as yield and flavor have remained stagnant.

UC Davis plant biologists used radiation to induce mutations in the leading peppermint clone grown in the United States, resulting in more than 250 new and genetically distinct variants. Altogether, they introduced 1,406 large genetic mutations, which can now be used to identify key genes for breeding or selecting new and superior peppermint varieties.

Image-based, pooled phenotyping reveals multidimensional, disease-specific variant effects

Variant in situ sequencing (VIS-seq) links genetic variants to cell images, revealing how variants affect molecules, subcellular structures, and cells at scale. Applied to thousands of LMNA and PTEN variants, VIS-seq illuminated how variants impacted a multidimensional phenotypic continuum that is not recapitulated by any single functional readout.

Brain histamine map connects genes to brain function and mental health

New research from King’s College London and the University of Porto has mapped the histamine system in the brain. Histamine, a molecule more commonly associated with allergies, plays a separate but poorly understood role in brain function. This study addresses this gap, building the first multiscale map of the histamine system that spans from genetics to behavior and related mental health conditions.

The findings provide a new framework for understanding how this often-overlooked chemical system contributes to brain function and could point toward new treatment strategies for histamine-related conditions such as depression, ADHD, and schizophrenia. The study is published in Nature Mental Health.

Histamine is a neurotransmitter, a molecule crucial for neurons to communicate with one another. Neuroscience research has classically focused on understanding other neurotransmitter systems such as dopamine and serotonin.

Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study

Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of childhood epilepsy. After the edit, the mice have far fewer seizures and live much longer. As published in Science Translational Medicine, the results suggest that a one-time genetic correction could someday treat the root cause of the disease rather than just managing its symptoms. The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.

The study also reflects growing momentum behind gene editing as a therapeutic platform for rare diseases. In February 2026, the Food and Drug Administration issued its Plausible Mechanism Framework guidance, outlining a regulatory pathway for individualized therapies targeting specific genetic conditions. It recognizes that for rare genetic diseases, a well-characterized biological mechanism can serve as the foundation for approval where large clinical trials are not feasible.

“For families affected by Dravet syndrome, our study provides proof of concept that a genetic correction approach could have real impact, a future with treatments that don’t just manage the disease but actually address its cause,” said Matthew Simon, a senior study director at The Jackson Laboratory (JAX) Rare Disease Translational Center (RDTC) who co-led the study. “We’re at an inflection point in genetic medicine, where we can now actually repair the DNA itself.”

Scientists successfully transfer longevity gene and extend lifespan

Researchers at the University of Rochester showed that one of those biological advantages can be moved into another mammal. By transferring a gene linked to the naked mole rat’s unusually high levels of high molecular weight hyaluronic acid (HMW-HA), the team improved health and modestly extended lifespan in mice.

The work, published in Nature in 2023, suggested that at least some longevity traits that evolved in long-lived animals may be adaptable beyond the species that developed them. The genetically modified mice lived healthier lives and had an approximate 4.4 percent increase in median lifespan compared with ordinary mice.

“Our study provides a proof of principle that unique longevity mechanisms that evolved in long-lived mammalian species can be exported to improve the lifespans of other mammals,” says Vera Gorbunova, the Doris Johns Cherry Professor of biology and medicine at Rochester.

Optogenetic mediated contractility enables reversible control of microglial morphology and migration in vivo

Biermeier et al. use live imaging in zebrafish to show that microglia alternate between distinct morphological states that support brain surveillance and phagocytosis. By optogenetically controlling cytoskeletal contractility, they demonstrate programmable, reversible control of microglial behavior in the living brain.

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CRISPR safeguard changes how engineered microbes can be controlled

Engineered microorganisms are widely used in industrial biotechnology and biopharmaceutical applications, including the production of biofuels, sustainable chemicals, and therapeutic compounds. However, concerns remain regarding the unintended environmental release and uncontrolled proliferation of genetically engineered microbes. For this reason, biocontainment technologies, which are designed to prevent microorganisms from surviving outside controlled environments, have become increasingly important in both academia and industry.

Conventional biocontainment strategies have relied on auxotrophy-based approaches, toxin–antitoxin systems, or DNA cleavage-based technologies such as CRISPR-Cas9. However, these methods often suffer from environmental dependency, genetic instability, and the risk of unintended mutations and cellular stress caused by DNA double-strand breaks.

In particular, DNA cleavage-based systems may compromise genomic stability and allow certain mutant cells to escape survival control. In addition, CRISPR interference (CRISPRi)-based systems are inherently reversible, posing challenges for achieving complete and permanent control of cell viability.

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