1. Feasibility study of a randomized controlled trial comparing docetaxel chemotherapy and androgen deprivation therapy with sequential prostatic biopsies from patients with advanced non-castration-resistant prostate cancer.

    Urologic Oncology: Seminars and Original Invest... 33(8):337.e1 (2015) PMID 26092557

    Sequential tissue biopsies taken during clinical trials of novel systemic anticancer therapies for advanced prostate cancer (PCa) may aid pharmacodynamic evaluation and biomarker discovery. We conducted a single institution phase-II open-labeled randomized study to assess the safety, tolerabilit...
  2. The Next Chapter.

    Human Gene Therapy 26(6):331 (2015) PMID 26091068

  3. Preexisting Neutralizing Antibodies to Adeno-Associated Virus Capsids in Large Animals Other Than Monkeys May Confound In Vivo Gene Therapy Studies.

    HUMAN GENE THERAPY, Part B: Methods 26(3):103 (2015) PMID 26067568 PMCID PMC4492586

    Adeno-associated virus (AAV) vectors are currently being tested not only in small animal models such as mice but also in large animal models, including pigs, dogs, and horses. Natural exposure to AAV occurs in most of the species used in these studies and potentially elicits a neutralizing humor...
  4. Coronary artery disease performance measures and statin use in patients with recent percutaneous coronary intervention or recent coronary artery bypass grafting (from the NCDR PINNACLE registry).

    The American Journal of Cardiology 115(8):1013 (2015) PMID 25721483

    The association between coronary revascularization strategy (percutaneous coronary intervention [PCI] or coronary artery bypass grafting [CABG]) and compliance with coronary artery disease (CAD) performance measures is not well studied. Our analysis studied patients enrolled in the Practice Inno...
  5. Motor neuron transduction after intracisternal delivery of AAV9 in a cynomolgus macaque.

    HUMAN GENE THERAPY, Part B: Methods 26(2):43 (2015) PMID 25885277

    The image shows a section of the lumbar spinal cord from a cynomolgus macaque that had received AAV9.CB.EGFP via the cisterna magna. Expression of GFP in multiple motor neurons is visible. Injection into the cerebrospinal fluid has been shown to be an effective route of vector administration for...
  6. Modelling the propagation of social response during a disease outbreak.

    Journal of Royal Society Interface 12(104) (2015) PMID 25589575

    Epidemic trajectories and associated social responses vary widely between populations, with severe reactions sometimes observed. When confronted with fatal or novel pathogens, people exhibit a variety of behaviours from anxiety to hoarding of medical supplies, overwhelming medical infrastructure...
  7. Modelling the propagation of social response during a disease outbreak.

    Journal of Royal Society Interface 12(104):20141105 (2015) PMID 25589575

    Epidemic trajectories and associated social responses vary widely between populations, with severe reactions sometimes observed. When confronted with fatal or novel pathogens, people exhibit a variety of behaviours from anxiety to hoarding of medical supplies, overwhelming medical infrastructure...
  8. Perspectives on best practices for gene therapy programs.

    Human Gene Therapy 26(3):127 (2015) PMID 25654329 PMCID PMC4367233

    With recent successes in gene therapy trials for hemophilia and retinal diseases, the promise and prospects for gene therapy are once again garnering significant attention. To build on this momentum, the National Institute of Neurological Disorders and Stroke and the Muscular Dystrophy Associati...
  9. β-Defensin 1 Plays a Role in Acute Mucosal Defense against Candida albicans.

    Journal of Immunology 194(4):1788 (2015) PMID 25595775

    Candida is an opportunistic fungal pathogen that colonizes the mucosal tract of humans. Pathogenic infection occurs in the presence of conditions causing perturbations to the commensal microbiota or host immunity. Early innate immune responses by the epithelium, including antimicrobial peptides ...
  10. β-Defensin 1 plays a role in acute mucosal defense against Candida albicans.

    Journal of Immunology 194(4):1788 (2015) PMID 25595775 PMCID PMC4323878

    Candida is an opportunistic fungal pathogen that colonizes the mucosal tract of humans. Pathogenic infection occurs in the presence of conditions causing perturbations to the commensal microbiota or host immunity. Early innate immune responses by the epithelium, including antimicrobial peptides ...
  11. β-Defensin 1 Plays a Role in Acute Mucosal Defense against Candida albicans.

    Journal of Immunology 194(4):1788 (2015) PMID 25595775

    Candida is an opportunistic fungal pathogen that colonizes the mucosal tract of humans. Pathogenic infection occurs in the presence of conditions causing perturbations to the commensal microbiota or host immunity. Early innate immune responses by the epithelium, including antimicrobial peptides ...
  12. β-Defensin 1 Plays a Role in Acute Mucosal Defense against Candida albicans.

    Journal of Immunology 194(4):1788 (2015) PMID 25595775 PMCID PMC4323878

    Candida is an opportunistic fungal pathogen that colonizes the mucosal tract of humans. Pathogenic infection occurs in the presence of conditions causing perturbations to the commensal microbiota or host immunity. Early innate immune responses by the epithelium, including antimicrobial peptides ...
  13. β-Defensin 1 Plays a Role in Acute Mucosal Defense against Candida albicans.

    Journal of Immunology 194(4):1788 (2015) PMID 25595775

    Candida is an opportunistic fungal pathogen that colonizes the mucosal tract of humans. Pathogenic infection occurs in the presence of conditions causing perturbations to the commensal microbiota or host immunity. Early innate immune responses by the epithelium, including antimicrobial peptides ...
  14. A call to arms for improved vector analytics!

    HUMAN GENE THERAPY, Part B: Methods 26(1):1 (2015) PMID 25679056

  15. A call to arms for improved vector analytics!

    HUMAN GENE THERAPY, Part B: Methods 26(1):1 (2015) PMID 25679056

  16. Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model.

    Nature Communications 6:7339 (2015) PMID 26081744

    Diseases of lipid metabolism are a major cause of human morbidity, but no animal model entirely recapitulates human lipoprotein metabolism. Here we develop a xenograft mouse model using hepatocytes from a patient with familial hypercholesterolaemia caused by loss-of-function mutations in the low...
  17. Hypothesis of Long-Term Outcome after Coronary Revascularization in Japanese Patients Compared to Multiethnic Groups in the US.

    PLoS ONE 10(5):e0128252 (2015) PMID 26023784 PMCID PMC4449105

    Ethnicity has a significant impact on coronary artery disease (CAD). This study investigated the long-term outcomes of Japanese patients undergoing revascularization compared with US patients belonging to multiple ethnic groups. We evaluated clinical outcomes, based on ethnicity, of patients inc...
  18. Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I.

    Molecular Therapy 22(12):2018 (2014) PMID 25027660

    Enzyme replacement therapy has revolutionized the treatment of the somatic manifestations of lysosomal storage diseases (LSD), although it has been ineffective in treating central nervous system (CNS) manifestations of these disorders. The development of neurotrophic vectors based on novel serot...
  19. Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I.

    Molecular Therapy 22(12):2018 (2014) PMID 25027660

    Enzyme replacement therapy has revolutionized the treatment of the somatic manifestations of lysosomal storage diseases (LSD), although it has been ineffective in treating central nervous system (CNS) manifestations of these disorders. The development of neurotrophic vectors based on novel serot...
  20. Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I.

    Molecular Therapy 22(12):2018 (2014) PMID 25027660 PMCID PMC4429692

    Enzyme replacement therapy has revolutionized the treatment of the somatic manifestations of lysosomal storage diseases (LSD), although it has been ineffective in treating central nervous system (CNS) manifestations of these disorders. The development of neurotrophic vectors based on novel serot...