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.
Tag: gene therapy
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 ...
Prime Assembly Enables Targeted Genomic Integration and Large-Scale DNA Rearrangements
A new prime editing-based strategy called prime assembly enables precise integration of large DNA fragments and programmable genomic rearrangements without ...
Tumor Slice Cultures Reveal New Pitfalls in Testing AAV Gene Therapy for Glioblastoma
A new study shows that the choice of patient-derived culture model strongly shapes how AAV-mediated gene delivery performs in glioblastoma, ...
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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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