1. 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...
  2. Evaluating the impact of systemic AAV9.cIDUA administration on brain pathology in MPS I dogs

    Molecular Genetics and Metabolism 114(2):S20 (2015)

  3. Adeno-associated virus vector-mediated gene therapy can effectively treat CNS and cardiac lesions and induce immune tolerance to the therapeutic enzyme in large animal models of mucopolysaccharidosis type I

    Molecular Genetics and Metabolism 114(2):S126 (2015)

  4. Intrathecal injection of lentiviral vector results in high expression in the brain of mucopolysaccharidosis VII dogs but the pattern of expression is different than for AAV9 or AAV-rh10

    Molecular Genetics and Metabolism 114(2):S95 (2015)

  5. 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...
  6. 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...
  7. 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...
  8. 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...
  9. In vivo evaluation of adeno-associated virus gene transfer in airways of mice with acute or chronic respiratory infection.

    Human Gene Therapy 25(11):966 (2014) PMID 25144316 PMCID PMC4235593

    Patients with cystic fibrosis (CF) often suffer chronic lung infection with concomitant inflammation, a setting that may reduce the efficacy of gene transfer. While gene therapy development for CF often involves viral-based vectors, little is known about gene transfer in the context of an infect...
  10. In vivo evaluation of adeno-associated virus gene transfer in airways of mice with acute or chronic respiratory infection.

    Human Gene Therapy 25(11):966 (2014) PMID 25144316 PMCID PMC4235593

    Abstract Patients with cystic fibrosis (CF) often suffer chronic lung infection with concomitant inflammation, a setting that may reduce the efficacy of gene transfer. While gene therapy development for CF often involves viral-based vectors, little is known about gene transfer in the context of ...
  11. In vivo evaluation of adeno-associated virus gene transfer in airways of mice with acute or chronic respiratory infection.

    Human Gene Therapy 25(11):966 (2014) PMID 25144316 PMCID PMC4235593

    Patients with cystic fibrosis (CF) often suffer chronic lung infection with concomitant inflammation, a setting that may reduce the efficacy of gene transfer. While gene therapy development for CF often involves viral-based vectors, little is known about gene transfer in the context of an infect...
  12. Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I.

    PNAS 111(41):14894 (2014) PMID 25267637 PMCID PMC4205647

    Patients with mucopolysaccharidosis type I (MPS I), a genetic deficiency of the lysosomal enzyme α-l-iduronidase (IDUA), exhibit accumulation of glycosaminoglycans in tissues, with resulting diverse clinical manifestations including neurological, ocular, skeletal, and cardiac disease. MPS I is c...
  13. Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I.

    PNAS 111(41):14894 (2014) PMID 25267637 PMCID PMC4205647

    Patients with mucopolysaccharidosis type I (MPS I), a genetic deficiency of the lysosomal enzyme α-l-iduronidase (IDUA), exhibit accumulation of glycosaminoglycans in tissues, with resulting diverse clinical manifestations including neurological, ocular, skeletal, and cardiac disease. MPS I is c...
  14. Professional regulation: a potentially valuable tool in responding to "stem cell tourism".

    Stem cell reports 3(3):379 (2014) PMID 25241736 PMCID PMC4266009

    The growing international market for unproven stem cell-based interventions advertised on a direct-to-consumer basis over the internet ("stem cell tourism") is a source of concern because of the risks it presents to patients as well as their supporters, domestic health care systems, and the stem...
  15. Professional regulation: a potentially valuable tool in responding to "stem cell tourism".

    Stem cell reports 3(3):379 (2014) PMID 25241736

    The growing international market for unproven stem cell-based interventions advertised on a direct-to-consumer basis over the internet ("stem cell tourism") is a source of concern because of the risks it presents to patients as well as their supporters, domestic health care systems, and the stem...
  16. Professional Regulation: A Potentially Valuable Tool in Responding to “Stem Cell Tourism”

    Stem cell reports 3(3):379 (2014)

    The growing international market for unproven stem cell-based interventions advertised on a direct-to-consumer basis over the internet (“stem cell tourism”) is a source of concern because of the risks it presents to patients as well as their supporters, domestic health care systems, an...
  17. Professional regulation: a potentially valuable tool in responding to "stem cell tourism".

    Stem cell reports 3(3):379 (2014) PMID 25241736 PMCID PMC4266009

    The growing international market for unproven stem cell-based interventions advertised on a direct-to-consumer basis over the internet ("stem cell tourism") is a source of concern because of the risks it presents to patients as well as their supporters, domestic health care systems, and the stem...
  18. Formation of newly synthesized adeno-associated virus capsids in the cell nucleus.

    HUMAN GENE THERAPY, Part B: Methods 25(3):179 (2014) PMID 24933465

    Adeno-associated virus (AAV) particles inside the nucleus of a HEK 293 cell are shown by electron microscopy. Cells have been triple-transfected for vector production and were analyzed for capsid formation three days later. Newly assembled particle are visible as seemingly unstructured conglomer...
  19. Formation of newly synthesized adeno-associated virus capsids in the cell nucleus.

    HUMAN GENE THERAPY, Part B: Methods 25(3):179 (2014) PMID 24933465

    Adeno-associated virus (AAV) particles inside the nucleus of a HEK 293 cell are shown by electron microscopy. Cells have been triple-transfected for vector production and were analyzed for capsid formation three days later. Newly assembled particle are visible as seemingly unstructured conglomer...
  20. Formation of newly synthesized adeno-associated virus capsids in the cell nucleus.

    HUMAN GENE THERAPY, Part B: Methods 25(3):179 (2014) PMID 24933465

    Adeno-associated virus (AAV) particles inside the nucleus of a HEK 293 cell are shown by electron microscopy. Cells have been triple-transfected for vector production and were analyzed for capsid formation three days later. Newly assembled particle are visible as seemingly unstructured conglomer...