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Moving away from one disease at a time: Screening, trial design, and regulatory implications of novel platform technologies

American journal of medical genetics. Part C, Seminars in medical genetics, 2023-03, Vol.193 (1), p.30-43

2023

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Data sharing to advance gene‐targeted therapies in rare diseases

American journal of medical genetics. Part C, Seminars in medical genetics, 2023-03, Vol.193 (1), p.87-98

2023

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CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

The New England journal of medicine, 2021-01, Vol.384 (3), p.252-260

2021

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Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

The New England journal of medicine, 2017-11, Vol.377 (21), p.2024-2035

2017

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Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

The New England journal of medicine, 2017-11, Vol.377 (21), p.2013-2023

2017

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Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR

American journal of respiratory and critical care medicine, 2018-01, Vol.197 (2), p.214-224

2018

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Pharmacokinetic and Drug–Drug Interaction Profiles of the Combination of Tezacaftor/Ivacaftor

Clinical and translational science, 2019-05, Vol.12 (3), p.267-275

2019

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Phase 1B, randomized, double‐blind, dose‐escalation trial of CPG 10101 in patients with chronic hepatitis C virus

Hepatology (Baltimore, Md.), 2007-11, Vol.46 (5), p.1341-1349

2007

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Effect of CC Chemokine Receptor 2 CCR2 Blockade on Serum C-Reactive Protein in Individuals at Atherosclerotic Risk and With a Single Nucleotide Polymorphism of the Monocyte Chemoattractant Protein-1 Promoter Region

The American journal of cardiology, 2011-03, Vol.107 (6), p.906-911

2011

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Inhibition of Human Neutrophil IL-8 Production by Hydrogen Peroxide and Dysregulation in Chronic Granulomatous Disease

The Journal of immunology (1950), 2005-01, Vol.174 (1), p.411-417

2005

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Enhancement of Hematopoietic Stem Cell Repopulating Capacity and Self-Renewal in the Absence of the Transcription Factor C/EBPα

Immunity (Cambridge, Mass.), 2004-12, Vol.21 (6), p.853-863

2004

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Continuous intravesical lidocaine treatment for interstitial cystitis/bladder pain syndrome: safety and efficacy of a new drug delivery device

Science translational medicine, 2012-07, Vol.4 (143), p.143ra100

2012

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Biological Role of the CCAAT/Enhancer-binding Protein Family of Transcription Factors

The Journal of biological chemistry, 1998-10, Vol.273 (44), p.28545-28548

1998

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Immunodeficiency Diseases Caused by Defects in Phagocytes

The New England journal of medicine, 2000-12, Vol.343 (23), p.1703-1714

2000

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The Role of C/EBPε in the Terminal Stages of Granulocyte Differentiation

Stem cells (Dayton, Ohio), 2001-03, Vol.19 (2), p.125-133

Lekstrom‐Himes, Julie A.

2001

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Association of Major Histocompatibility Complex Determinants with the Development of Symptomatic and Asymptomatic Genital Herpes Simplex Virus Type 2 Infections

The Journal of infectious diseases, 1999-05, Vol.179 (5), p.1077-1085

1999

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Immunodeficiency Diseases Caused by Defects in Phagocytes

The New England journal of medicine, 2000-12, Vol.343 (23), p.1703-1714

2000

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The Quantity of Latent Viral DNA Correlates with the Relative Rates at Which Herpes Simplex Virus Types 1 and 2 Cause Recurrent Genital Herpes Outbreaks

Journal of Virology, 1998-04, Vol.72 (4), p.2760-2764

1998

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Human platelets exhibit chemotaxis using functional N-formyl peptide receptors

Experimental hematology, 2005, Vol.33 (1), p.73-84

2005

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Gamma Interferon Impedes the Establishment of Herpes Simplex Virus Type 1 Latent Infection but Has No Impact on Its Maintenance or Reactivation in Mice

Journal of Virology, 2000-07, Vol.74 (14), p.6680-6683

2000

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Platelets deliver costimulatory signals to antigen-presenting cells: A potential bridge between injury and immune activation

Experimental hematology, 2004-02, Vol.32 (2), p.135-139

2004

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Molecular analysis of the mouse S100A9 gene and evidence that the myeloid specific transcription factor C/EBPepsilon is not required for the regulation of the S100A9/A8 gene expression in neutrophils

Journal of cellular biochemistry, 2001-03, Vol.80 (4), p.606-616

2001

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Metabolic Response of Mice to a Postnatal Ablation of CCAAT/Enhancer-binding Protein α

The Journal of biological chemistry, 2005-11, Vol.280 (46), p.38689-38699

2005

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Novel Combinatorial Interactions of GATA-1, PU.1, and C/EBPε Isoforms Regulate Transcription of the Gene Encoding Eosinophil Granule Major Basic Protein

The Journal of biological chemistry, 2002-11, Vol.277 (45), p.43481-43494

2002

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Periodic Illness Associated with Epstein-Barr Virus Infection

Clinical infectious diseases, 1996-01, Vol.22 (1), p.22-27

1996

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Moving away from one disease at a time: Screening, trial design, and regulatory implications of novel platform technologies

Data sharing to advance gene‐targeted therapies in rare diseases

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR

Pharmacokinetic and Drug–Drug Interaction Profiles of the Combination of Tezacaftor/Ivacaftor

Phase 1B, randomized, double‐blind, dose‐escalation trial of CPG 10101 in patients with chronic hepatitis C virus

Effect of CC Chemokine Receptor 2 CCR2 Blockade on Serum C-Reactive Protein in Individuals at Atherosclerotic Risk and With a Single Nucleotide Polymorphism of the Monocyte Chemoattractant Protein-1 Promoter Region

Inhibition of Human Neutrophil IL-8 Production by Hydrogen Peroxide and Dysregulation in Chronic Granulomatous Disease

Enhancement of Hematopoietic Stem Cell Repopulating Capacity and Self-Renewal in the Absence of the Transcription Factor C/EBPα

Continuous intravesical lidocaine treatment for interstitial cystitis/bladder pain syndrome: safety and efficacy of a new drug delivery device

Biological Role of the CCAAT/Enhancer-binding Protein Family of Transcription Factors

Immunodeficiency Diseases Caused by Defects in Phagocytes

The Role of C/EBPε in the Terminal Stages of Granulocyte Differentiation

Association of Major Histocompatibility Complex Determinants with the Development of Symptomatic and Asymptomatic Genital Herpes Simplex Virus Type 2 Infections

Immunodeficiency Diseases Caused by Defects in Phagocytes

The Quantity of Latent Viral DNA Correlates with the Relative Rates at Which Herpes Simplex Virus Types 1 and 2 Cause Recurrent Genital Herpes Outbreaks

Human platelets exhibit chemotaxis using functional N-formyl peptide receptors

Gamma Interferon Impedes the Establishment of Herpes Simplex Virus Type 1 Latent Infection but Has No Impact on Its Maintenance or Reactivation in Mice

Platelets deliver costimulatory signals to antigen-presenting cells: A potential bridge between injury and immune activation

Molecular analysis of the mouse S100A9 gene and evidence that the myeloid specific transcription factor C/EBPepsilon is not required for the regulation of the S100A9/A8 gene expression in neutrophils

Metabolic Response of Mice to a Postnatal Ablation of CCAAT/Enhancer-binding Protein α

Novel Combinatorial Interactions of GATA-1, PU.1, and C/EBPε Isoforms Regulate Transcription of the Gene Encoding Eosinophil Granule Major Basic Protein

Periodic Illness Associated with Epstein-Barr Virus Infection

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