In vitro read-through of phenylalanine hydroxylase (PAH) nonsense mutations using aminoglycosides: a potential therapy for phenylketonuria

Nutrition in phenylketonuria

(2011) A. MacDonald et al.   MOLECULAR GENETICS AND METABOLISM

Rescue of Melanocortin 4 Receptor (MC4R) Nonsense Mutations by Aminoglycoside-Mediated Read-Through

(2011) Harald Brumm et al.   Obesity

Repairing faulty genes by aminoglycosides: Development of new derivatives of geneticin (G418) with enhanced suppression of diseases-causing nonsense mutations

(2010) Igor Nudelman et al.   BIOORGANIC & MEDICINAL CHEMISTRY

Nonsense suppressor therapies rescue peroxisome lipid metabolism and assembly in cells from patients with specific PEX gene mutations

(2010) Patricia K. Dranchak et al.   JOURNAL OF CELLULAR BIOCHEMISTRY

Therapeutic liver repopulation for phenylketonuria

(2010) Cary O. Harding et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Suboptimal outcomes in patients with PKU treated early with diet alone: Revisiting the evidence

(2010) G.M. Enns et al.   MOLECULAR GENETICS AND METABOLISM

Nonaminoglycoside compounds induce readthrough of nonsense mutations

(2009) Liutao Du et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Development of Novel Aminoglycoside (NB54) with Reduced Toxicity and Enhanced Suppression of Disease-Causing Premature Stop Mutations

(2009) Igor Nudelman et al.   JOURNAL OF MEDICINAL CHEMISTRY

Converting an injectable protein therapeutic into an oral form: Phenylalanine ammonia lyase for phenylketonuria

(2009) Tse Siang Kang et al.   MOLECULAR GENETICS AND METABOLISM

Correction of Murine PKU Following AAV-mediated Intramuscular Expression of a Complete Phenylalanine Hydroxylating System

(2008) Zhaobing Ding et al.   MOLECULAR THERAPY