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@genesndiseases.bsky.socialOct 12, 2026, 6:56 AM

Could #GeneTherapy move #Phenylketonuria treatment beyond #DietaryRestriction?

This review explores #CRISPRCas9, base and prime editing, gene replacement and mRNA therapies to restore PAH activity, while addressing challenges.

#OpenAccess #GenesAndDiseases: doi.org/10.1016/j.ge...

@jamanetworkopen.comOct 9, 2026, 2:30 PM

Among children with early-treated #Phenylketonuria, phenylalanine control worsened with age, and classic phenylketonuria was associated with poorer long-term metabolic control. ja.ma/4rTgpYy

Two graphs. A: Overall estimated phenylalanine (Phe) concentration increases from ~100 to ~600 µmol/L from age 0-18. B: Phe by diagnosis. Mild PKU rises to ~500 µmol/L by age 18; Classic PKU rises to ~700 µmol/L, consistently higher.
@itmat.bsky.socialSep 18, 2026, 1:29 AM

Improving the safety and efficiency of adenine base-editing therapies www.nature.com/articles/s41... @kiranmusunuru.bsky.social @ahrensnicklas.bsky.social #CRISPR #Phenylketonuria #PseudoxanthomaElasticum #HereditaryTyrosinemia