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Advancing Familial Hypercholesterolemia Diagnosis: Activity-Normalized Prime Editing for Comprehensive LDLR Variant Classification
CardiologyChuyển hóa mỡ máuGenetic variant classification

Advancing Familial Hypercholesterolemia Diagnosis: Activity-Normalized Prime Editing for Comprehensive LDLR Variant Classification

By MedXY|Sep 6, 2026

A novel prime editing screening method enables precise functional classification of LDLR gene variants, enhancing diagnosis and therapeutic strategies for familial hypercholesterolemia.

Allergy & ImmunologyPrime Editing

Precision Gene Correction via Prime Editing Restores NADPH Oxidase Activity in Patients with p47phox-Deficient Chronic Granulomatous Disease

By MedXY
|
Dec 19, 2025

A Phase 1/2 clinical trial demonstrates that PM359, an autologous prime-edited CD34+ cell therapy, successfully corrects the delGT mutation in NCF1, restoring essential microbicidal function in patients with p47-CGD without the off-target r

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