Starting the conversation on gene therapy for phenylketonuria: Current perspectives of patients, caregivers, and advocates
Author:
Publisher
Elsevier BV
Subject
Endocrinology,Genetics,Molecular Biology
Reference44 articles.
1. Updated, web-based nutrition management guideline for PKU: an evidence and consensus based approach;Singh;Mol. Genet. Metab.,2016
2. The complete European guidelines on phenylketonuria: diagnosis and treatment;van Wegberg;Orphanet J. Rare. Dis.,2017
3. Neurocognitive evidence for revision of treatment targets and guidelines for phenylketonuria;Jahja;J. Pediatr.,2014
4. Systematic review and meta-analysis of neuropsychiatric symptoms and executive functioning in adults with phenylketonuria;Bilder;Dev. Neuropsychol.,2016
5. Psychiatric and cognitive aspects of phenylketonuria: the limitations of diet and promise of new treatments;Ashe;Front. Psychiatry,2019
Cited by 2 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. Breaking genetic shackles: The advance of base editing in genetic disorder treatment;Frontiers in Pharmacology;2024-03-06
2. Phenylalanine hydroxylase mRNA rescues the phenylketonuria phenotype in mice;Frontiers in Bioengineering and Biotechnology;2022-10-07
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