标题
Development of an mRNA replacement therapy for phenylketonuria
作者
关键词
MT: Delivery Strategies, mRNA, replacement, lipid nanoparticle, PKU, phenylketonuria, liver, LNP, therapy, hepatocytes, PAH
出版物
Molecular Therapy-Nucleic Acids
Volume 28, Issue -, Pages 87-98
出版商
Elsevier BV
发表日期
2022-02-28
DOI
10.1016/j.omtn.2022.02.020
参考文献
相关参考文献
注意：仅列出部分参考文献，下载原文获取全部文献信息。- Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH
- (2020) Seemin S. Ahmed et al. Molecular Therapy-Methods & Clinical Development
- Phenylketonuria: Current Treatments and Future Developments
- (2019) Uta Lichter-Konecki et al. DRUGS
- Low Seroprevalence of Neutralizing Antibodies Targeting Two Clade F AAV in Humans
- (2018) Jeff L. Ellsworth et al. Human Gene Therapy Clinical Development
- mRNA-Based Protein Replacement Therapy for the Heart
- (2018) Ajit Magadum et al. MOLECULAR THERAPY
- New protein structures provide an updated understanding of phenylketonuria
- (2017) Eileen K. Jaffe MOLECULAR GENETICS AND METABOLISM
- The complete European guidelines on phenylketonuria: diagnosis and treatment
- (2017) A. M. J. van Wegberg et al. Orphanet Journal of Rare Diseases
- Systemic delivery of factor IX messenger RNA for protein replacement therapy
- (2017) Suvasini Ramaswamy et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Adeno-Associated Virus Gene Therapy for Liver Disease
- (2016) Lisa M. Kattenhorn et al. HUMAN GENE THERAPY
- Alternative therapies to address the unmet medical needs of patients with phenylketonuria
- (2015) Nenad Blau et al. EXPERT OPINION ON PHARMACOTHERAPY
- Follow up of phenylketonuria patients
- (2011) M. Demirkol et al. MOLECULAR GENETICS AND METABOLISM
- Expression of therapeutic proteins after delivery of chemically modified mRNA in mice
- (2011) Michael S D Kormann et al. NATURE BIOTECHNOLOGY
- The reality of dietary compliance in the management of phenylketonuria
- (2010) Anita MacDonald et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Phenylketonuria
- (2010) Nenad Blau et al. LANCET
- Optimizing the use of sapropterin (BH4) in the management of phenylketonuria
- (2009) Nenad Blau et al. MOLECULAR GENETICS AND METABOLISM
- Converting an injectable protein therapeutic into an oral form: Phenylalanine ammonia lyase for phenylketonuria
- (2009) Tse Siang Kang et al. MOLECULAR GENETICS AND METABOLISM
- Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria
- (2008) Phillip Lee et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART A
- Defining tetrahydrobiopterin (BH4)-responsiveness in PKU
- (2008) Nenad Blau JOURNAL OF INHERITED METABOLIC DISEASE
- Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors
- (2008) Sharon C Cunningham et al. MOLECULAR THERAPY
- Preclinical evaluation of multiple species of PEGylated recombinant phenylalanine ammonia lyase for the treatment of phenylketonuria
- (2008) C. N. Sarkissian et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
Discover Peeref hubs
Discuss science. Find collaborators. Network.
Join a conversationPublish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn More