Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH-Reference-Cited by-同舟云学术

Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH

Published:2020-06 Issue: Volume:17 Page:568-580
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Ahmed Seemin S.,Rubin Hillard,Wang Minglun,Faulkner Deiby,Sengooba Arnold,Dollive Serena N.,Avila Nancy,Ellsworth Jeff L.,Lamppu Diana,Lobikin Maria,Lotterhand Jason,Adamson-Small Laura,Wright Teresa,Seymour Albert,Francone Omar L.

Funder

Homology Medicines

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference57 articles.

1. Serotonin and dopamine synthesis in phenylketonuria;Curtius;Adv. Exp. Med. Biol.,1981

2. Phenylketonuria: an inborn error of phenylalanine metabolism;Williams;Clin. Biochem. Rev.,2008

3. Diagnosis, classification, and genetics of phenylketonuria and tetrahydrobiopterin (BH4) deficiencies;Blau;Mol. Genet. Metab.,2011

4. Neuropsychologic functions of early treated patients with phenylketonuria, on and off diet: results of a cross-national and cross-sectional study;Burgard;Pediatr. Res.,1997

5. Long-term enzymatic and phenotypic correction in the phenylketonuria mouse model by adeno-associated virus vector-mediated gene transfer;Oh;Pediatr. Res.,2004

Cited by 19 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Current Advances and Material Innovations in the Search for Novel Treatments of Phenylketonuria;Advanced Healthcare Materials;2024-05-31

2. Inborn errors of amino acid metabolism – from underlying pathophysiology to therapeutic advances;Disease Models & Mechanisms;2023-11-01

3. Genome-wide assays to characterize rAAV integration into human genomic DNA in vivo;2023-08-23

4. State‐of‐the‐art 2023 on gene therapy for phenylketonuria;Journal of Inherited Metabolic Disease;2023-08-03

5. Liver directed adeno‐associated viral vectors to treat metabolic disease;Journal of Inherited Metabolic Disease;2023-06-05