1. Recollections from a Pioneer Who Provided the Foundation for the Success of Gene Therapy in Treating Severe Combined Immune Deficiencies.

    Human Gene Therapy Clinical Development 27(2):53 (2016) PMID 27314911

  2. AAV Natural Infection Induces Broad Cross-Neutralizing Antibody Responses to Multiple AAV Serotypes in Chimpanzees.

    Human Gene Therapy Clinical Development 27(2):79 (2016) PMID 27314914 PMCID PMC4932656

    Cross-sectional studies of primates have revealed that natural neutralizing antibody (NAb) responses to adeno-associated viruses (AAV) span multiple serotypes. This differs from the phenotype of the NAb response to an AAV vector delivered to seronegative nonhuman primates that is typically restr...
  3. Stable liver-specific expression of human IDOL in humanized mice raises plasma cholesterol.

    Cardiovascular Research 110(1):23 (2016) PMID 26786161 PMCID PMC4798044

    IDOL (inducible degrader of the low-density lipoprotein receptor, LDLR) is an E3 ubiquitin ligase that promotes the ubiquitination and degradation of the LDLR. IDOL is a potential therapeutic target for the development of a novel class of low-density lipoprotein cholesterol (LDL-C)-lowering ther...
  4. Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia.

    Human Gene Therapy 27(5):345 (2016) PMID 26790480 PMCID PMC4841085

    Isolated methylmalonic acidemia (MMA), a group of autosomal recessive inborn errors of metabolism, is most commonly caused by complete (mut(0)) or partial (mut(-)) deficiency of the enzyme methylmalonyl-CoA mutase (MUT). The severe metabolic instability and increased mortality experienced by man...
  5. ARCII: A phase II trial of the HIV protease inhibitor Nelfinavir in combination with chemoradiation for locally advanced inoperable pancreatic cancer.

    Radiotherapy and Oncology 119(2):306 (2016) PMID 27117177 PMCID PMC4917892

    Nelfinavir can enhance intrinsic radiosensitivity, reduce hypoxia and improve vascularity. We conducted a phase II trial combining nelfinavir with chemoradiotherapy (CRT) for locally advanced inoperable pancreatic cancer (LAPC). Radiotherapy (50.4Gy/28 fractions; boost to 59.4Gy/33 fractions) wa...
  6. Interview with Inder Verma, PhD.

    Human Gene Therapy Clinical Development 27(1):5 (2016) PMID 27003750

  7. A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.

    Nature Biotechnology 34(3):334 (2016) PMID 26829317 PMCID PMC4786489

    Many genetic liver diseases in newborns cause repeated, often lethal, metabolic crises. Gene therapy using nonintegrating viruses such as adeno-associated virus (AAV) is not optimal in this setting because the nonintegrating genome is lost as developing hepatocytes proliferate. We reasoned that ...
  8. Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.

    Molecular Therapy 24(2):206 (2016) PMID 26447927 PMCID PMC4817811

    Mucopolysaccharidosis VII (MPS VII) is a lysosomal storage disease arising from mutations in β-d-glucuronidase (GUSB), which results in glycosaminoglycan (GAG) accumulation and a variety of clinical manifestations including neurological disease. Herein, MPS VII dogs were injected intravenously (...
  9. Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids.

    Molecular Therapy 23(12):1877 (2015) PMID 26412589 PMCID PMC4700115

    Vectors based on the clade E family member adeno-associated virus (AAV) serotype 8 have shown promise in patients with hemophilia B and have emerged as best in class for human liver gene therapies. We conducted a thorough evaluation of liver-directed gene therapy using vectors based on several n...
  10. Human immune system mice immunized with Plasmodium falciparum circumsporozoite protein induce protective human humoral immunity against malaria.

    Journal of Immunological Methods 427:42 (2015) PMID 26410104

    In this study, we developed human immune system (HIS) mice that possess functional human CD4+ T cells and B cells, named HIS-CD4/B mice. HIS-CD4/B mice were generated by first introducing HLA class II genes, including DR1 and DR4, along with genes encoding various human cytokines and human B cel...
  11. A Journey in the Development of Gene Therapy for Inherited Disorders of the Bone Marrow.

    Human Gene Therapy Clinical Development 26(4):203 (2015) PMID 26684443

  12. Structure of neurotropic adeno-associated virus AAVrh.8.

    Journal of Structural Biology 192(1):21 (2015) PMID 26334681 PMCID PMC4617535

    Adeno-associated virus rhesus isolate 8 (AAVrh.8) is a leading vector for the treatment of neurological diseases due to its efficient transduction of neuronal cells and reduced peripheral tissue tropism. Toward identification of the capsid determinants for these properties, the structure of AAVr...
  13. There and Back Again: Mitchell Finer on the Journey of Biotech from Start-Up to Success.

    Human Gene Therapy Clinical Development 26(3):140 (2015) PMID 26390088

  14. Human Gene Therapy Clinical Development: Where the Academy and Industry Meet.

    Human Gene Therapy Clinical Development 26(3):139 (2015) PMID 26390087

  15. Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.

    The lancet. Respiratory medicine 3(9):684 (2015) PMID 26149841 PMCID PMC4673100

    Lung delivery of plasmid DNA encoding the CFTR gene complexed with a cationic liposome is a potential treatment option for patients with cystic fibrosis. We aimed to assess the efficacy of non-viral CFTR gene therapy in patients with cystic fibrosis. We did this randomised, double-blind, placebo...
  16. Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.

    Molecular Therapy 23(8):1298 (2015) PMID 26022732 PMCID PMC4817868

    The potential host immune response to a nonself protein poses a fundamental challenge for gene therapies targeting recessive diseases. We demonstrate in both dogs and nonhuman primates that liver-directed gene transfer using an adeno-associated virus (AAV) vector in neonates induces a persistent...
  17. 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...
  18. 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...
  19. The Next Chapter.

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

  20. 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...