Study on a novel strategy to treatment of phenylketonuria.
To replace the low phenylalanine (phe) diet treatment and to improve the quality of life of the phenylketonuria (PKU) patients, A phenylalanine ammonia-lyase (PAL) cDNA from petroselinum crispum was subcloned into expression vectors, pMG36e and pNZ8048, and transformed L. lactis by electroporation. The pMG36e PAL/L. lactis and pNZ8048 PAL/L.L. were screened and characterized by using PCR and HPLC, and prepared as a liquid type preparation that were given orally to treat hyperphenylalaninemia-rats. The phe levels of the rat plasma sample were determined by HPLC. The data showed that the plasma phe levels in hyperphenylalanemia (HPA) rats receiving preparations made from the engineered L. lactis were significantly reduced compared with non-treated HPA rats. The effect of the pNZ8048PAL/L.L. showed a higher expression of PAL and better cure results than pMG36ePAL/L.L. These results point a potential way for PKU treatment.
Liu
Jingzhong
J
Basic Medical Research Center, Beijing Chaoyang Hospital, Affiliated The Capital University of Medical Sciences, PR China.
Jia
Xingyuan
X
Zhang
Jing
J
Xiang
Hua
H
Hu
Wei
W
Zhou
Yan
Y
D000284:Administration, Oral; D000818:Animals; D015316:Genetic Therapy / Q000379:methods*; D013294:Lactococcus lactis / Q000235:genetics; D028528:Petroselinum / Q000201:enzymology; D010649:Phenylalanine / Q000097:blood; D010650:Phenylalanine Ammonia-Lyase / Q000008:administration & dosage* / Q000235:genetics / Q000627:therapeutic use; D010661:Phenylketonurias / Q000628:therapy*; D010957:Plasmids / Q000008:administration & dosage / Q000235:genetics / Q000627:therapeutic use; D051381:Rats; D014170:Transformation, Genetic; D016896:Treatment Outcome
D010649:Phenylalanine; D010650:Phenylalanine Ammonia-Lyase