Scientists Discover Drug That Reverses Rare Blood Disorder by Boosting Protein Production
Researchers found that inhibiting LSD1 enzyme increases SEC23A protein levels, potentially treating congenital anemia.
20 articles
Researchers found that inhibiting LSD1 enzyme increases SEC23A protein levels, potentially treating congenital anemia.
Scientists develop first drug targeting AVIL protein, showing effectiveness against deadly glioblastoma tumors in preclinical trials.
AstraZeneca's baxdrostat wins FDA approval for hypertension, offering a novel mechanism for patients uncontrolled on existing therapies.
New research explores using Klotho protein with stem cell-derived vesicles to treat acute kidney injury, potentially offering regenerative therapy.
Wave Life Sciences shares early trial data on an RNA editing treatment for alpha-1 antitrypsin deficiency, targeting monthly dosing.
A landmark multi-omics atlas of human kidney tissue reveals how proximal tubule cells shift between repair and disease states after injury.
A novel anti-swelling hydrogel loaded with dasatinib, quercetin, and nucleus pulposus exosomes clears senescent cells and restores disc integrity in rats and goats.
Single-cell sequencing reveals ferroptosis drives pulpitis; thymosin ฮฑ1 restores GPX4, cuts iron overload, and reduces inflammation in cells and rats.
Injectable hydrogel from traditional Chinese herbs shows superior wound repair by regulating immune response and promoting tissue regeneration.
Biotech startup Nionyx Bio takes top prize at BIO-Europe Spring for innovative gene therapy targeting kidney disease using proprietary AAV technology.
Novel iodide-enhanced hydrogel penetrates gum pockets to eliminate harmful bacteria while protecting regenerative stem cells.
NO-primed extracellular vesicles transfer functional mitochondria to damaged kidney cells, restoring energy production and reducing injury.
Mitochondria-targeted antioxidant polymer shows superior protection against liver transplant injury compared to standard treatments.
New research reveals stem cells from urine contain telomerase activity that could combat aging by maintaining telomeres and rejuvenating tissues.
Intellia Therapeutics reports a landmark Phase 3 win for CRISPR gene editing, potentially offering a one-time cure for a debilitating genetic disease.
A Phase 2 trial evaluates MBA-P01, a novel botulinum toxin A, for safety and optimal dosing in treating frown lines.
A CRISPR-based adenine base editor restores CFTR function in airway cells and intestinal organoids from CF patients with a previously undruggable mutation.
Enhanced stem cells reduced inflammation and improved bladder function in rats with nerve damage.
Amgen expands late-stage testing of its long-acting obesity drug MariTide, including a trial for patients switching from rival GLP-1 medications.
Breakthrough crystal technology enables transplanted insulin-producing cells to survive and function for a full year without traditional immunosuppression.