Gene Therapy Restores Hearing in Congenital Deafness Trial Milestone
A new review charts the rapid rise of AAV-mediated OTOF gene replacement therapy, from the first human trials to the ...
Tag: gene therapy
Hidden Androgen Receptor Isoform Shows Promise Against Kennedy’s Disease
A naturally occurring short isoform of the androgen receptor, AR-A, dramatically reduces aggregation and toxicity of the mutant protein causing ...
Base Editing Rewrites PCSK9 in Human Embryos Without DNA Breaks, Study Finds
Researchers delivered an adenine base editor as a protein into human embryos, achieving efficient PCSK9 editing without double-strand breaks, though ...
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 ...
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 ...
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Mothers who receive childcare support from maternal grandparents show more parental warmth, finds NTU Singapore study
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University of Seville Breaks 120-Year-Old Mystery, Revises a Key Einstein Concept
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Bee body mass, pathogens and local climate influence heat tolerance
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Researchers record first-ever images and data of a shark experiencing a boat strike
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Groundbreaking Clinical Trial Reveals Lubiprostone Enhances Kidney Function
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