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TRIAMF: A New Method for Delivery of Cas9 Ribonucleoprotein Complex to Human Hematopoietic Stem Cells

Scientific reports, 2018-11, Vol.8 (1), p.16304-11, Article 16304

2018

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Transducer of Regulated CREB-Binding Proteins (TORCs) Induce PGC-1α Transcription and Mitochondrial Biogenesis in Muscle Cells

Proceedings of the National Academy of Sciences - PNAS, 2006-09, Vol.103 (39), p.14379-14384

2006

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CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease

The New England journal of medicine, 2023-08, Vol.389 (9), p.820-832

2023

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Collaborative science to advance gene therapies in resource-limited parts of the world

Molecular therapy, 2021-11, Vol.29 (11), p.3101-3102

2021

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Muscle-specific expression of PPARγ coactivator-1α improves exercise performance and increases peak oxygen uptake

Journal of applied physiology (1985), 2008-05, Vol.104 (5), p.1304-1312

2008

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Receptor interactions involved in adenoviral-mediated gene delivery after systemic administration in non-human primates

Human gene therapy, 2003-11, Vol.14 (17), p.1595-1604

2003

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In Vivo Hepatic Adenoviral Gene Delivery Occurs Independently of the Coxsackievirus–Adenovirus Receptor

Molecular therapy, 2002-06, Vol.5 (6), p.770-779

2002

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A molecular glue degrader of the WIZ transcription factor for fetal hemoglobin induction

Science (American Association for the Advancement of Science), 2024-07, Vol.385 (6704), p.91-99

2024

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Targeting Adenoviral Vectors by Using the Extracellular Domain of the Coxsackie-Adenovirus Receptor: Improved Potency via Trimerization

Journal of Virology, 2002-02, Vol.76 (4), p.1892-1903

2002

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Identification of novel modulators of mitochondrial function by a genome-wide RNAi screen in Drosophila melanogaster

Genome Research, 2008-01, Vol.18 (1), p.123-136

2008

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Adenovirus serotype 5 fiber shaft influences in vivo gene transfer in mice

Human gene therapy, 2003-05, Vol.14 (8), p.777-787

2003

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Interaction of systemically delivered adenovirus vectors with Kupffer cells in mouse liver

Human gene therapy, 2008-05, Vol.19 (5), p.547-554

2008

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Estrogen-related receptor α is essential for the expression of antioxidant protection genes and mitochondrial function

Biochemical and biophysical research communications, 2007-05, Vol.357 (1), p.231-236

2007

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Effect of adenovirus serotype 5 fiber and penton modifications on in vivo tropism in rats

Molecular therapy, 2004-08, Vol.10 (2), p.344-354

2004

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Gene Therapy With Inducible Nitric Oxide Synthase Protects Against Myocardial Infarction via a Cyclooxygenase-2-Dependent Mechanism

Circulation research, 2003-04, Vol.92 (7), p.741-748

2003

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Adenovirus Vector Pseudotyping in Fiber-Expressing Cell Lines: Improved Transduction of Epstein-Barr Virus-Transformed B Cells

Journal of Virology, 2000-01, Vol.74 (1), p.354-362

2000

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Adenovirus Type 5 Viral Particles Pseudotyped with Mutagenized Fiber Proteins Show Diminished Infectivity of Coxsackie B-Adenovirus Receptor-Bearing Cells

Journal of Virology, 2001-03, Vol.75 (6), p.2972-2981

2001

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Treatment of Individuals with Severe Sickle Cell Disease with OTQ923, an Autologous, Ex Vivo, CRISPR/Cas9-Edited, CD34+ Hematopoietic Stem and Progenitor Cell Product, Leads to Durable Engraftment and Fetal Hemoglobin Induction

Blood, 2022-11, Vol.140 (Supplement 1), p.1906-1908

2022

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CRISPR/Cas9 Genome-Edited Autologous Hematopoietic Stem Cell Transplantation Therapies for Sickle Cell Disease

Blood, 2022-11, Vol.140 (Supplement 1), p.12982-12983

2022

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381. The Adenoviral Fiber Shaft Is a Major Determinant of Kupffer Cell Necrosis

Molecular therapy, 2006-05, Vol.13 (S1), p.S145-S145

2006

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Overexpression of soluble Fas attenuates transplant arteriosclerosis in rat aortic allografts

Circulation (New York, N.Y.), 2002-09, Vol.106 (12), p.1536-1542

2002

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Targeted Degradation of the Wiz Transcription Factor for Gamma Globin De-Repression

Blood, 2023-11, Vol.142 (Supplement 1), p.2-2

2023

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In vivo transduction of photoreceptors or ciliary body by intravitreal injection of pseudotyped adenoviral vectors

Molecular therapy, 2003-01, Vol.7 (1), p.27-34

2003

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Inhibition of accelerated graft arteriosclerosis by gene transfer of soluble fibroblast growth factor receptor-1 in rat aortic transplants

Arteriosclerosis, thrombosis, and vascular biology, 2004-06, Vol.24 (6), p.1081-1086

2004

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Phenotypic correction of hypercholesterolemia in apoE-deficient mice by adenovirus-mediated in vivo gene transfer

Arteriosclerosis, thrombosis, and vascular biology, 1995-04, Vol.15 (4), p.479-484

1995

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TRIAMF: A New Method for Delivery of Cas9 Ribonucleoprotein Complex to Human Hematopoietic Stem Cells

Transducer of Regulated CREB-Binding Proteins (TORCs) Induce PGC-1α Transcription and Mitochondrial Biogenesis in Muscle Cells

CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease

Collaborative science to advance gene therapies in resource-limited parts of the world

Muscle-specific expression of PPARγ coactivator-1α improves exercise performance and increases peak oxygen uptake

Receptor interactions involved in adenoviral-mediated gene delivery after systemic administration in non-human primates

In Vivo Hepatic Adenoviral Gene Delivery Occurs Independently of the Coxsackievirus–Adenovirus Receptor

A molecular glue degrader of the WIZ transcription factor for fetal hemoglobin induction

Targeting Adenoviral Vectors by Using the Extracellular Domain of the Coxsackie-Adenovirus Receptor: Improved Potency via Trimerization

Identification of novel modulators of mitochondrial function by a genome-wide RNAi screen in Drosophila melanogaster

Adenovirus serotype 5 fiber shaft influences in vivo gene transfer in mice

Interaction of systemically delivered adenovirus vectors with Kupffer cells in mouse liver

Estrogen-related receptor α is essential for the expression of antioxidant protection genes and mitochondrial function

Effect of adenovirus serotype 5 fiber and penton modifications on in vivo tropism in rats

Gene Therapy With Inducible Nitric Oxide Synthase Protects Against Myocardial Infarction via a Cyclooxygenase-2-Dependent Mechanism

Adenovirus Vector Pseudotyping in Fiber-Expressing Cell Lines: Improved Transduction of Epstein-Barr Virus-Transformed B Cells

Adenovirus Type 5 Viral Particles Pseudotyped with Mutagenized Fiber Proteins Show Diminished Infectivity of Coxsackie B-Adenovirus Receptor-Bearing Cells

Treatment of Individuals with Severe Sickle Cell Disease with OTQ923, an Autologous, Ex Vivo, CRISPR/Cas9-Edited, CD34+ Hematopoietic Stem and Progenitor Cell Product, Leads to Durable Engraftment and Fetal Hemoglobin Induction

CRISPR/Cas9 Genome-Edited Autologous Hematopoietic Stem Cell Transplantation Therapies for Sickle Cell Disease

381. The Adenoviral Fiber Shaft Is a Major Determinant of Kupffer Cell Necrosis

Overexpression of soluble Fas attenuates transplant arteriosclerosis in rat aortic allografts

Targeted Degradation of the Wiz Transcription Factor for Gamma Globin De-Repression

In vivo transduction of photoreceptors or ciliary body by intravitreal injection of pseudotyped adenoviral vectors

Inhibition of accelerated graft arteriosclerosis by gene transfer of soluble fibroblast growth factor receptor-1 in rat aortic transplants

Phenotypic correction of hypercholesterolemia in apoE-deficient mice by adenovirus-mediated in vivo gene transfer

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