Complete restoration of phenylalanine oxidation in phenylketonuria mouse by a self-complementary adeno-associated virus vector

Characterization of a Recombinant Adeno-Associated Virus Type 2 Reference Standard Material

(2010) Martin Lock et al.   HUMAN GENE THERAPY

Long-term correction of murine phenylketonuria by viral gene transfer: liver versus muscle

(2010) Beat Thöny   JOURNAL OF INHERITED METABOLIC DISEASE

Effect of Adeno-Associated Virus Serotype and Genomic Structure on Liver Transduction and Biodistribution in Mice of Both Genders

(2009) Astrid Pañeda et al.   HUMAN GENE THERAPY

Comparison of Adeno-Associated Virus Pseudotype 1, 2, and 8 Vectors Administered by Intramuscular Injection in the Treatment of Murine Phenylketonuria

(2009) Alexandre Rebuffat et al.   HUMAN GENE THERAPY

Liver-restricted expression of the canine factor VIII gene facilitates prevention of inhibitor formation in factor VIII-deficient mice

(2009) Akira Ishiwata et al.   JOURNAL OF GENE MEDICINE

AAV gene therapy as a means to increase apolipoprotein (Apo) A-I and high-density lipoprotein-cholesterol levels: correction of murine ApoA-I deficiency

(2009) Stefan F. C. Vaessen et al.   JOURNAL OF GENE MEDICINE

Enhancing transduction of the liver by adeno-associated viral vectors

(2008) A C Nathwani et al.   GENE THERAPY

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

(2008) Zhaobing Ding et al.   MOLECULAR THERAPY

Optimization of Self-complementary AAV Vectors for Liver-directed Expression Results in Sustained Correction of Hemophilia B at Low Vector Dose

(2007) Zhijian Wu et al.   MOLECULAR THERAPY