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BioWorld - Wednesday, April 15, 2026
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Biomarkers

New link between PRRX1 gene and open-angle glaucoma

Oct. 21, 2025
No Comments
Primary open-angle glaucoma (POAG) is among the leading causes of irreversible blindness. Early detection and treatment are crucial for vision preservation, and research to identify genes associated with POAG is ongoing.
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Illustration demonstrating structure of the human eye and organization of retinal cells
Genetic/congenital

rAAV2-U1a-hELP1 rescues RGC function in familial dysautonomia

Oct. 20, 2025
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Familial dysautonomia (FD) is an autosomal recessive neurodevelopmental disorder.
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Purple-tinted test tubes and dropper
Biomarkers

miR-1265 emerges as a biomarker of meningioma

Oct. 17, 2025
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Meningiomas are usually grade I benign tumors that can turn into aggressive grade II or III malignant tumors. Genome-wide methylation analysis has shown that microRNA 1265 (miR-1265) is hypermethylated in meningiomas.


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Fallopian tubes, ovaries and uterus
Biomarkers

AKR1B1 and AKR1B10 as prognostic markers in endometrial cancer

Oct. 17, 2025
No Comments
Endometrial cancer is the most prevalent gynecological malignancy in high-income countries, and the discovery of new biomarkers may aid in earlier detection and improve the prognosis of patients.
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Art concept for inflammation in the intestines
Gastrointestinal

Orchard’s OTL-104 is safe and efficacious in Crohn’s disease model

Oct. 16, 2025
No Comments

NOD2 deficiency is involved in the pathogenesis of Crohn’s disease (CD) due to failure of gut innate immunity and loss of intestinal tissue homeostasis. Orchard Therapeutics Ltd. has presented preclinical data on OTL-104, human hematopoietic stem cell (HSC) gene therapy for the treatment of NOD2-deficient CD.


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Neurology/psychiatric

Selective GABA-A α3 modulator shows efficacy in essential tremor

Oct. 15, 2025
Essential tremor is a movement disorder marked by involuntary, rhythmic shaking in the hands but sometimes affecting the head, voice and other areas. Its exact cause is still unknown. Recent research suggests that dysfunction in the neurotransmitter systems, particularly involving γ-aminobutyric acid (GABA), contributes significantly to the disorder.
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Cancer

[68Ga]NOTA-R-54 arises as imaging probe for CXCR4+ tumors

Oct. 15, 2025
No Comments
CXC chemokine receptor 4 (CXCR4) is a receptor overexpressed in several tumor types and associated with tumor aggressiveness and risk of metastasis, resistance and recurrence. A novel PET radiopharmaceutical tracer and CXCR4 ligand, [68Ga]NOTA-R-54, was developed and tested for potential use in the treatment of lung cancer.
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LDL particle binding to the LDL receptor
Genetic/congenital

Epigenetic approach shows durable PCSK9 silencing in primates

Oct. 15, 2025
By Mar de Miguel
No Comments
A preclinical study presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, showed a new epigenetic editing technology that enables durable gene silencing using ELXRs, short for Epigenetic Long-Term X-Repressors. With this approach, scientists at Scribe Therapeutics Inc. successfully inhibited the expression of the PCSK9 gene, a key regulator of cholesterol metabolism, in human cells, mice and nonhuman primates.
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Cardiovascular

EDIT-401 lowers LDL cholesterol by modulating LDLR

Oct. 14, 2025
No Comments
When the levels of LDL cholesterol are elevated, there is increased risk for atherosclerotic cardiovascular disease. Lowering these levels contributes to decreasing the risk of major adverse cardiovascular events. EDIT-401 is a CRISPR-based LDL cholesterol-lowering gene-editing approach developed by Editas Medicine Inc.
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Hand holding radioactive symbol
Cancer

[177Lu]LNC-1018 performs well in CCK2R+ tumors

Oct. 14, 2025
No Comments
Recent evidence suggests the gastrin/cholecystokinin-2 receptor (CCK2R) is a potential target for radiolabeled theranostics in cancer, but the stability of minigastrin analogues remains unresolved. Researchers from the National University of Singapore aimed to develop a peptide with improved tumor uptake and prolonged retention in CCK2R+ cancers.
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