Cell encapsulation as a potential nondietary therapy for maternal phenylketonuria

Donna A Santillan; Mark K Santillan; Stephen K Hunter

doi:10.1016/j.ajog.2009.05.035

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Cell encapsulation as a potential nondietary therapy for maternal phenylketonuria

Journal article

Peer reviewed

Cell encapsulation as a potential nondietary therapy for maternal phenylketonuria

Donna A Santillan, Mark K Santillan and Stephen K Hunter

American journal of obstetrics and gynecology, Vol.201(3), pp.289.e1-289.e6

2009

DOI: 10.1016/j.ajog.2009.05.035

PMID: 19631922

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Abstract

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. 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. 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. 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.

alginate microspheres

cell encapsulation

immunoisolation

phenylalanine hydroxylase

phenylketonuria

Details

Title: Subtitle: Cell encapsulation as a potential nondietary therapy for maternal phenylketonuria
Creators: Donna A Santillan - University of Iowa, Obstetrics and Gynecology
Mark K Santillan - University of Iowa, Obstetrics and Gynecology
Stephen K Hunter - University of Iowa, Obstetrics and Gynecology
Resource Type: Journal article
Publication Details: American journal of obstetrics and gynecology, Vol.201(3), pp.289.e1-289.e6
Publisher: Mosby, Inc
DOI: 10.1016/j.ajog.2009.05.035
PMID: 19631922
ISSN: 0002-9378
eISSN: 1097-6868
Language: English
Date published: 2009
Academic Unit: Roy J. Carver Department of Biomedical Engineering; Obstetrics and Gynecology
Record Identifier: 9983557492902771

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