Phenylalanine hydroxylase mRNA rescues the phenylketonuria phenotype in mice

In vivo prime editing of a metabolic liver disease in mice

(2022) Desirée Böck et al.   Science Translational Medicine

Development of an mRNA replacement therapy for phenylketonuria

(2022) Carlos G. Perez-Garcia et al.   Molecular Therapy-Nucleic Acids

Starting the conversation on gene therapy for phenylketonuria: Current perspectives of patients, caregivers, and advocates

(2022) Debra S. Regier et al.   Molecular Genetics and Metabolism Reports

mRNA-based therapy proves superior to the standard of care for treating hereditary tyrosinemia 1 in a mouse model

(2022) Maximiliano L. Cacicedo et al.   Molecular Therapy-Methods & Clinical Development

Effect of BH4 on blood phenylalanine and tyrosine variations in patients with phenylketonuria

(2021) Wegberg van AMJ et al.   MOLECULAR GENETICS AND METABOLISM

Use of an AAV serotype Anc80 to provide durable cure of phenylketonuria in a mouse model

(2021) Robert A. Kaiser et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Challenges Posed by Immune Responses to AAV Vectors: Addressing Root Causes

(2021) Bradley A. Hamilton et al.   Frontiers in Immunology

mRNA-Based Vaccines

(2021) Frank Kowalzik et al.   Vaccines

Phenylketonuria

(2021) Francjan J. van Spronsen et al.   Nature Reviews Disease Primers

Optimization of non-coding regions for a non-modified mRNA COVID-19 vaccine

(2021) Makda S. Gebre et al.   NATURE

Efficacy and safety of the CVnCoV SARS-CoV-2 mRNA vaccine candidate in ten countries in Europe and Latin America (HERALD): a randomised, observer-blinded, placebo-controlled, phase 2b/3 trial

(2021) Peter G Kremsner et al.   LANCET INFECTIOUS DISEASES

The Genetic Landscape and Epidemiology of Phenylketonuria

(2020) Alicia Hillert et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Evaluation of the mRNA-1273 Vaccine against SARS-CoV-2 in Nonhuman Primates

(2020) Kizzmekia S. Corbett et al.   NEW ENGLAND JOURNAL OF MEDICINE

Bone Status in Patients with Phenylketonuria: A Systematic Review

(2020) María José de Castro et al.   Nutrients

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

COVID-19 vaccine BNT162b1 elicits human antibody and TH1 T cell responses

(2020) Ugur Sahin et al.   NATURE

Phenylalanine effects on brain function in adult phenylketonuria

(2020) Andrea Pilotto et al.   NEUROLOGY

Dual mRNA therapy restores metabolic function in long-term studies in mice with propionic acidemia

(2020) Lei Jiang et al.   Nature Communications

First 1.5 years of pegvaliase clinic: Experiences and outcomes

(2020) Stephanie Sacharow et al.   Molecular Genetics and Metabolism Reports

Synthetic human ABCB4 mRNA therapy rescues severe liver disease phenotype in a BALB/c.Abcb4 mouse model of PFIC3

(2020) Guangyan Wei et al.   JOURNAL OF HEPATOLOGY

Safety and Efficacy of the BNT162b2 mRNA Covid-19 Vaccine

(2020) Fernando P. Polack et al.   NEW ENGLAND JOURNAL OF MEDICINE

Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

(2019) Piotr S. Kowalski et al.   MOLECULAR THERAPY

Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates

(2019) Xuling Zhu et al.   AMERICAN JOURNAL OF HUMAN GENETICS

State-of-the-Art 2019 on Gene therapy for Phenylketonuria

(2019) Hiu Man Grisch-Chan et al.   HUMAN GENE THERAPY

The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs

(2019) Akin Akinc et al.   Nature Nanotechnology

mRNA as a Novel Treatment Strategy for Hereditary Spastic Paraplegia Type 5

(2019) Stefan Hauser et al.   Molecular Therapy-Methods & Clinical Development

AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria

(2019) Daelyn Y. Richards et al.   Molecular Therapy-Methods & Clinical Development

G6PC mRNA Therapy Positively Regulates Fasting Blood Glucose and Decreases Liver Abnormalities in a Mouse Model of Glycogen Storage Disease 1a

(2018) Daniel S. Roseman et al.   MOLECULAR THERAPY

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

(2018) David Adams et al.   NEW ENGLAND JOURNAL OF MEDICINE

Association of immune response with efficacy and safety outcomes in adults with phenylketonuria administered pegvaliase in phase 3 clinical trials

(2018) Soumi Gupta et al.   EBioMedicine

Efficacy, safety and population pharmacokinetics of sapropterin in PKU patients

(2017) Ania C. Muntau et al.   Orphanet Journal of Rare Diseases

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

(2017) Ding An et al.   Cell Reports

Phenylalanine hydroxylase: Function, structure, and regulation

(2013) Marte I. Flydal et al.   IUBMB LIFE

Chronic kidney disease in adolescent and adult patients with phenylketonuria

(2012) Julia B. Hennermann et al.   JOURNAL OF INHERITED METABOLIC DISEASE

The interplay between genotype, metabolic state and cofactor treatment governs phenylalanine hydroxylase function and drug response

(2011) M. Staudigl et al.   HUMAN MOLECULAR GENETICS

Activation of Phenylalanine Hydroxylase Induces Positive Cooperativity toward the Natural Cofactor

(2010) Søren W. Gersting et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

A robust and high-throughput Cre reporting and characterization system for the whole mouse brain

(2009) Linda Madisen et al.   NATURE NEUROSCIENCE