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Rational design and applications of piperazine and cyclohexane ionizable lipids for PKU and SSADH deficiency

Jiyeon Son1,2, Hyunjung Yoo1, Joonyoung Park1

  • 1Department of Research & Development, GC Biopharma Corp., Yongin-si, Gyeonggi-do 16924, Republic of Korea.

Molecular Therapy. Nucleic Acids
|December 15, 2025
PubMed

Keywords:
MT: Delivery StrategiesPKURNA therapeuticsSSADHionizable lipidlipid nanoparticle

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