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Non-coding RNA mutations unveiled as new cause of retinitis pigmentosa
Retinitis pigmentosa (RP) is a genetic eye disorder affecting around one in 5,000 people worldwide. It typically begins with ...
Novel Gene and Variant Discovery in Human Genetic Disorders: From Coding and Non-Coding RNA variants
Rapid advances in sequencing technologies have transformed our ability to diagnose human genetic disorders, yet many patients still lack a molecular ...
Researchers from Proqr Therapeutics NV recently presented preclinical data on AX-0810, a single-stranded RNA editing oligonucleotide (EON) targeting RNA coding for NTCP cotransporter ...
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