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 ...
Tag: gene therapy
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.
Stem Cell Transplant Cured a Girl’s Leukemia but Left Her Friedreich Ataxia Unchanged
A decade-long case report shows that allogeneic stem cell transplantation cured a Friedreich's ataxia patient's leukemia and normalized her blood ...
Protein Axis That Shields the Cornea Offers New Hope Against Dry Eye Pain
A newly identified PPP4C-CALML5 protein axis restores corneal nerves, tear secretion and pain relief in a mouse model of dry ...
Six Experts Map the Future of Rare Metabolic Disease Research
Six leading researchers outline the opportunities, challenges and emerging priorities for advancing research and therapy development in rare metabolic diseases.
CRISPR’s Journey From Molecular Scissors to Precision Genome Editors
A new review traces how CRISPR genome editing has evolved from blunt molecular scissors into increasingly precise therapeutic tools for ...
Base Editors Correct Artemis Mutations, Offering New Hope for SCID Gene Therapy
Researchers have shown that CRISPR base editors can correct three pathogenic Artemis mutations ex vivo with up to 50 percent ...
Copper Overload Pushes Cells Toward Death in Wilson Disease
A new review in Cell Death & Discovery links copper overload, mitochondrial failure and regulated cell death pathways including cuproptosis ...
Nonprofit Accelerator Commits $1.7 Million to Five Projects Targeting Blindness, Sepsis and Rare Disease
Critical Path Institute's Translational Therapeutics Accelerator has awarded nearly $1.7 million across five BRIDGe grants to advance therapies for neuropathic ...
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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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