22 Result(s)
within
Ronald G. Crystal
Cancer Research
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Article
Gene therapy in a murine model of chronic eosinophilic leukemia-not otherwise specified (CEL-NOS)
Chronic eosinophilic leukemia-not otherwise specified (CEL-NOS) is a rare, aggressive, fatal disease characterized by blood eosinophilia and dysfunction of organs infiltrated with eosinophils. Clinically, the ...
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Reference Work Entry In depth
Chronic Obstructive Pulmonary Disease and Lung Cancer
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Living Reference Work Entry In depth
Chronic Obstructive Pulmonary Disease and Lung Cancer
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Article
Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia
In Friedreich's ataxia, caused by mutation of the gene encoding the mitochondrial protein frataxin, the major cause of mortality is heart failure. Using mice lacking frataxin in the heart, Hélène Puccio and he...
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Article
A SNAIL1–SMAD3/4 transcriptional repressor complex promotes TGF-β mediated epithelial–mesenchymal transition
TGF-β mediates epithelial-mesenchymal transitions (EMT) during tumorigenesis but the molecular mechanisms driving this effect have been unclear. The transcriptional repressor SNAIL1 and the downstream effector...
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Reference Work Entry In depth
Chronic Obstructive Pulmonary Disease and Lung Cancer
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Article
Cytokine-mediated deployment of SDF-1 induces revascularization through recruitment of CXCR4+ hemangiocytes
The mechanisms through which hematopoietic cytokines accelerate revascularization are unknown. Here, we show that the magnitude of cytokine-mediated release of SDF-1 from platelets and the recruitment of nonen...
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Article
Genetic medicines: treatment strategies for hereditary disorders
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.
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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...
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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...
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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...
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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...
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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 ...
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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...
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Article
In vivo and ex vivo gene therapy strategies to treat tumors using adenovirus gene transfer vectors
The adaptation of gene therapy strategies to treat tumors has broadened the potential armamentarium of anticancer strategies to include approaches for local control of tumor growth as well as to enhance syste...
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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.
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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...
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Article
IL-6 release and airway administration of human CFTR cDNA adenovirus vector
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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 ...
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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...
