Iowa Research Online

Title

Cell encapsulation as a potential nondietary therapy for maternal phenylketonuria.

Authors

Donna A. Santillan, University of Iowa, Iowa City, IAFollow
Mark Santillan, University of IowaFollow
Stephen K. Hunter, University of IowaFollow

Document Type

Article

Peer Reviewed

1

Publication Date

9-1-2009

Journal/Book/Conference Title

American journal of obstetrics and gynecology

Volume

201

Abstract

OBJECTIVE: The objective of this work was to determine whether cells overexpressing phenylalanine (Phe) hydroxylase (PAH) can significantly reduce Phe in vitro for potential use as a therapy for preventing maternal phenylketonuria.

STUDY DESIGN: Human 293T and WRL68 cell lines were transiently and stably transfected to overexpress PAH. Cells were encapsulated within microspheres of sodium alginate. Timed measurements of Phe in media were performed using tandem mass spectrometry.

RESULTS: Both nonencapsulated and encapsulated transiently transfected cells overexpressing PAH significantly reduced the Phe concentration in media by approximately 50% in comparison to mock-transfected cells. Cell line clones stably expressing PAH significantly decreased the Phe concentration in the media by up to 85% compared with media alone.

CONCLUSION: Both unencapsulated and encapsulated cells overexpressing PAH significantly reduce Phe in vitro. Studies using phenylketonuria model mice will be important in determining the ability of our therapy to prevent the teratogenic effects of elevated maternal Phe in maternal phenylketonuria.

Keywords

Alginates, Animals, Cell Line, Drug Carriers, Female, Glucuronic Acid, Hexuronic Acids, Humans, Mice, Microspheres, Phenylalanine Hydroxylase, Phenylketonurias, Pregnancy, Pregnancy Complications