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

AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

(2009) F. Mingozzi et al.   BLOOD

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

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

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

(2009) M. L. Brantly et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Optimized adeno-associated virus 8 produces hepatocyte-specific Cre-mediated recombination without toxicity or affecting liver regeneration

(2008) Karen J. Ho et al.   AMERICAN JOURNAL OF PHYSIOLOGY-GASTROINTESTINAL AND LIVER PHYSIOLOGY

Potential of AAV vectors in the treatment of metabolic disease

(2008) I E Alexander et al.   GENE THERAPY

Enhancing transduction of the liver by adeno-associated viral vectors

(2008) A C Nathwani et al.   GENE THERAPY

Immunity to adeno-associated virus vectors in animals and humans: a continued challenge

(2008) A K Zaiss et al.   GENE THERAPY

Bioluminescent virion shells: new tools for quantitation of AAV vector dynamics in cells and live animals

(2008) A Asokan et al.   GENE THERAPY

Identification of pharmacological chaperones as potential therapeutic agents to treat phenylketonuria

(2008) Angel L. Pey et al.   JOURNAL OF CLINICAL INVESTIGATION

Recent developments in adeno‐associated virus vector technology

(2008) Hildegard Büning et al.   JOURNAL OF GENE MEDICINE

What we know that could influence future treatment of phenylketonuria

(2008) C. N. Sarkissian et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Safety and Efficacy of Regional Intravenous (RI) Versus Intramuscular (IM) Delivery of rAAV1 and rAAV8 to Nonhuman Primate Skeletal Muscle

(2008) Alice Toromanoff et al.   MOLECULAR THERAPY

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

(2008) Zhaobing Ding et al.   MOLECULAR THERAPY

Recombinant Adeno-associated Virus Transduction and Integration

(2008) Brian R Schultz et al.   MOLECULAR THERAPY

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli 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

AAV8, 9, Rh10, Rh43 Vector Gene Transfer in the Rat Brain: Effects of Serotype, Promoter and Purification Method

(2007) Ronald L Klein et al.   MOLECULAR THERAPY