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  1. No Access

    Article

    A Pro → Leu substitution in codon 369 of the alpha-1-antitrypsin deficiency variant PI MHeerlen

    The molecular defect has been elucidated in the alpha-1-antitrypsin (PI) gene of a patient with a serum level of only 5 mg/100 ml and a PI M-like phenotype, designated PI MHeerlen. The restriction fragment pat...

    Marten H. Hofker, Toshihiro Nukiwa, Heleen M. B. van Paassen… in Human Genetics (1989)

  2. No Access

    Article

    Genetic basis of variable exon 9 skip** in cystic fibrosis transmembrane conductance regulator mRNA

    Variable in–frame skip** of exon 9 in cystic fibrosis transmembrane conductance regulator (CFTR) mRNA transcripts (exon 9−) occurs in the respiratory epithelium. To explore the genetic basis of this event, we e...

    Chin-Shyan Chu, Bruce C. Trapnell, Sheila Curristin, Garry R. Cutting… in Nature Genetics (1993)

  3. No Access

    Article

    Direct in vivo gene transfer to ependymal cells in the central nervous system using recombinant adenovirus vectors

    To evaluate the potential for adenovirus–mediated central nervous system (CMS) gene transfer, the replication deficient recombinant adenovirus vectors Ad.RSVβgal (coding for β–galactosidase) and Ad–α1AT (codin...

    Gianluigi Bajocchi, Sanford H. Feldman, Ronald G. Crystal… in Nature Genetics (1993)

  4. No Access

    Article

    Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis

    We have administered a recombinant adenovirus vector (AdCFTR) containing the normal human CFTR cDNA to the nasal and bronchial epithelium of four individuals with cystic fibrosis (CF). We show that this vector ca...

    Ronald G. Crystal, Noel G. McElvaney, Melissa A. Rosenfeld… in Nature Genetics (1994)

  5. No Access

    Article

    The gene as the drug

    Caplen et al. (pages 39–46) demonstrate that it is feasible to use liposomes to transfer a human gene in vivo. Can this vector mimic the ability of viruses to effectively transfer genetic information into target ...

    Ronald G. Crystal in Nature Medicine (1995)

  6. No Access

    Article

    IL-6 release and airway administration of human CFTR cDNA adenovirus vector

    Noel G. McElvaney, Ronald G. Crystal in Nature Medicine (1995)

  7. No Access

    Article

    Ectopic expression of thyrotropin releasing hormone (TRH) receptors in liver modulates organ function to regulate blood glucose by TRH

    Maintenance of blood glucose by the liver is normally initiated by extracellular regulatory molecules such as glucagon and vasopressin triggering specific hepatocyte receptors to activate the cAMP or phosphoin...

    Gerhard Wolff, Andrea Mastrangeli, Marcos Heinflink, Erik Falck-Pedersen… in Nature Genetics (1996)

  8. No Access

    Article

    Managed care for viruses

    The finding of a common receptor for coxsackievirus and adenovirus should help improve strategies for drug design and gene therapy.

    Ronald G. Crystal in Nature Medicine (1997)

  9. No Access

    Article

    Use of L-plastin promoter to develop an adenoviral system that confers transgene expression in ovarian cancer cells but not in normal mesothelial cells

    The objective of this study was to develop an adenoviral vector system that would generate a pattern of expression of exogenous therapeutic genes appropriate for the treatment of ovarian cancer. For this purpo...

    Injae Chung, Peter E Schwartz, Ronald G Crystal, Giuseppe Pizzorno… in Cancer Gene Therapy (1999)

  10. No Access

    Article

    Regional treatment of hepatic micrometastasis by adenovirus vector-mediated delivery of interleukin-2 and interleukin-12 cDNAs to the hepatic parenchyma

    We hypothesize that adenovirus (Ad) vector-mediated delivery of the human interleukin-2 (IL-2) cDNA (AdIL2) or the murine IL-12 cDNA heterodimer (AdIL12) would produce high concentrations of cytokines in the l...

    Edward A Hirschowitz, Hassan A Naama, Denis Evoy… in Cancer Gene Therapy (1999)

  11. No Access

    Article

    Dendritic cells genetically modified to express CD40 ligand and pulsed with antigen can initiate antigen-specific humoral immunity independent of CD4+ T cells

    We have investigated whether dendritic cells genetically modified to express CD40 ligand and pulsed with antigen can trigger B cells to produce antigen-specific antibodies without CD4+ T-cell help. Dendritic cell...

    Toshiaki Kikuchi, Stefan Worgall, Ravi Singh, Malcolm A.S. Moore… in Nature Medicine (2000)

  12. No Access

    Protocol

    Adenoviral-Vector-Mediated Gene Transfer to Dendritic Cells

    Dendritic cells (DC) are the most potent antigen presenting cells capable of initiating T-cell-dependent immune responses (1-5). This biologic potential can be harnessed to elicit effective antigen-specific immun...

    Wenru Song, Ronald G. Crystal in Dendritic Cell Protocols (2001)

  13. No Access

    Article

    Impaired recruitment of bone-marrow–derived endothelial and hematopoietic precursor cells blocks tumor angiogenesis and growth

    The role of bone marrow (BM)-derived precursor cells in tumor angiogenesis is not known. We demonstrate here that tumor angiogenesis is associated with recruitment of hematopoietic and circulating endothelial ...

    David Lyden, Koichi Hattori, Sergio Dias, Carla Costa, Pamela Blaikie… in Nature Medicine (2001)

  14. No Access

    Article

    Placental growth factor reconstitutes hematopoiesis by recruiting VEGFR1+ stem cells from bone-marrow microenvironment

    The mechanism by which angiogenic factors recruit bone marrow (BM)-derived quiescent endothelial and hematopoietic stem cells (HSCs) is not known. Here, we report that functional vascular endothelial growth fa...

    Koichi Hattori, Beate Heissig, Yan Wu, Sergio Dias, Rafael Tejada… in Nature Medicine (2002)

  15. No Access

    Article

    Sampling-Dependent Up-regulation of Gene Expression in Sequential Samples of Human Airway Epithelial Cells

    As part of a study of in vivo gene expression levels in the human airway epithelium in response to chronic cigarette smoking, we have identified a number of genes whose expression levels are altered in a time-...

    Adriana Heguy, Ben-Gary Harvey, Timothy P O’Connor, Neil R Hackett… in Molecular Medicine (2003)

  16. No Access

    Article

    Chemokine-mediated interaction of hematopoietic progenitors with the bone marrow vascular niche is required for thrombopoiesis

    The molecular pathways involved in the differentiation of hematopoietic progenitors are unknown. Here we report that chemokine-mediated interactions of megakaryocyte progenitors with sinusoidal bone marrow end...

    Scott T Avecilla, Koichi Hattori, Beate Heissig, Rafael Tejada… in Nature Medicine (2004)

  17. No Access

    Article

    Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency

    X-linked immunodeficiency with hyper-IgM (HIGM1), characterized by failure of immunoglobulin isotype switching, is caused by mutations of the CD40 ligand (CD40L), which is normally expressed on activated CD4+ T c...

    Minoru Tahara, Robert G Pergolizzi, Hiroyasu Kobayashi, Anja Krause… in Nature Medicine (2004)

  18. No Access

    Chapter

    Cancer-Related Gene Therapy Clinical Trials

    Gene therapy represents a strategy using transfer of genetic information to modify a population of target cells for therapeutic purposes. The transferred genetic material has typically included double-stranded...

    Robert J. Korst, Ronald G. Crystal in Cancer Gene Therapy (2005)

  19. No Access

    Article

    Gene expression profiling of human alveolar macrophages of phenotypically normal smokers and nonsmokers reveals a previously unrecognized subset of genes modulated by cigarette smoking

    Cigarette smoking is the leading cause of the respiratory diseases collectively known as chronic obstructive pulmonary disease (COPD). While the pathogenesis of COPD is complex, there is abundant evidence that...

    Adriana Heguy, Timothy P. O’Connor, Karsta Luettich… in Journal of Molecular Medicine (2006)

  20. No Access

    Article

    Genetic medicines: treatment strategies for hereditary disorders

  21. Of the approximately 25,000 genes that comprise the human genome, mutations in more than 1,800 have already been identified as causing hereditary disorders.

    ...

    22. Timothy P. O'Connor, Ronald G. Crystal in Nature Reviews Genetics (2006)

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