Engineered AAV Capsid Reaches the Outer Retina Through a Simple Eye Injection
Researchers used directed evolution to create AAV5-AlyT, an adeno-associated virus variant that delivers genes efficiently to photoreceptors after minimally invasive ...
Tag: gene therapy
Astrocyte Protein LAMC1 Emerges as a Genetic Guardian Against Deadly Brain Bleeds
A multi-omics and mouse study identifies the astrocyte-derived extracellular matrix protein LAMC1 as a genetically validated protector of the blood–brain ...
Gene Therapy Now Leads Spinal Muscular Atrophy Treatment as Real-World Sequencing Patterns Emerge
A large US claims analysis shows that after widespread newborn screening, gene replacement therapy has become the dominant first treatment ...
Diatomaceous Earth Filtration Scales Up Viral Vector Harvest for Gene Therapy
Researchers at Roche Diagnostics have shown that alluvial filtration with diatomaceous earth can clarify rAAV vector lysates at scales up ...
Choosing the Right Adenine Base Editor: New Study Maps the Rules for Precision Gene Editing
A systematic comparison of SpCas9-based adenine base editors in human cells reveals that conventional NGG-targeting editors deliver the highest activity ...
Rare but Treatable: The Brain Diseases Radiologists Cannot Afford to Miss
A new review in Pediatric Radiology maps the MRI and MR spectroscopy patterns of treatable neurogenetic diseases, urging radiologists to ...
Masked AAV Vectors Switch On Gene Delivery Only Where Disease Signals Strike
Researchers have engineered adeno-associated viral vectors whose infectivity is chemically masked and restored only by liver proteases, near-infrared light or ...
Gene Therapy Tames the Brain’s Dangerous Electrical Storms
Researchers used adeno-associated virus to express the KCNN4 gene in mouse neocortex, cutting the amplitude of spreading depolarization several-fold and ...
Epigenetic Editing Steps Closer to the Clinic as Bioengineering Tools Mature
The EpiBio-24 conference in Amsterdam showcased maturing epigenetic engineering tools, durable gene silencing in animal models, and growing clinical momentum ...
Hidden RNA Switch Protects Jaw Cartilage From Osteoarthritis Damage
A newly mapped OIP5-AS1/miR-223-3p/FoxO3 regulatory axis protects jaw cartilage from degeneration, and boosting FoxO3 in rats slowed temporomandibular joint osteoarthritis.
-
Mothers who receive childcare support from maternal grandparents show more parental warmth, finds NTU Singapore study
-
University of Seville Breaks 120-Year-Old Mystery, Revises a Key Einstein Concept
-
Bee body mass, pathogens and local climate influence heat tolerance
-
Researchers record first-ever images and data of a shark experiencing a boat strike
-
Groundbreaking Clinical Trial Reveals Lubiprostone Enhances Kidney Function
Embark on a thrilling journey of discovery with Scienmag.com—your ultimate source for cutting-edge breakthroughs. Immerse yourself in a world where curiosity knows no limits and tomorrow’s possibilities become today’s reality!