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Seamless Gene Correction in the Human Cystic Fibrosis Transmembrane Conductance Regulator Locus by Vector Replacement and Vector Insertion Events

Frontiers in genome editing, 2022-04, Vol.4, p.843885-843885

2022

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TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs

Molecular therapy. Nucleic acids, 2016-01, Vol.5 (1), p.e273-e273, Article e273

2016

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Induction of therapeutic levels of HbF in genome‐edited primary β039‐thalassaemia haematopoietic stem and progenitor cells

British journal of haematology, 2021-01, Vol.192 (2), p.395-404

2021

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Induction of therapeutic levels of HbF in genome-edited primary β 0 39-thalassaemia haematopoietic stem and progenitor cells

British journal of haematology, 2021-01, Vol.192 (2), p.395-404

2021

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Recombinant adeno-associated viral vector encoding human VEGF165 induces neomicrovessel formation in the adult mouse brain

Frontiers in bioscience, 2006-09, Vol.11, p.3190

2006

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Permanent coronary artery occlusion: cardiovascular MR imaging is platform for percutaneous transendocardial delivery and assessment of gene therapy in canine model

Radiology, 2008-11, Vol.249 (2), p.560

2008

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Adeno-associated viral vector-mediated gene transfer of VEGF normalizes skeletal muscle oxygen tension and induces arteriogenesis in ischemic rat hindlimb

Molecular therapy, 2003-01, Vol.7 (1), p.44-51

2003

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Antibodies to Human Fetal Erythroid Cells from a Nonimmune Phage Antibody Library

Proceedings of the National Academy of Sciences - PNAS, 2001-02, Vol.98 (5), p.2682-2687

2001

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228. AAV Mediated Hypoxia-Inducible VEGF Expression and Neovascular Formation in Ischemic Pig Myocardium

Molecular therapy, 2006-05, Vol.13 (S1), p.S87-S87

2006

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332. Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells

Molecular therapy, 2016-05, Vol.24, p.S132-S132

2016

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Cloning MafF by Recognition Site Screening with the NFE2 Tandem Repeat of HS2: Analysis of Its Role in Globin and GCSl Genes Regulation

Blood cells, molecules, & diseases, 2002-09, Vol.29 (2), p.145-158

2002

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Induction of therapeutic levels of HbF in genome-edited primary β0 39-thalassaemia haematopoietic stem and progenitor cells

British journal of haematology, 2021-01, Vol.192 (2), p.395-404

2021

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Multiple Protein 4.1 Isoforms Produced by Alternative Splicing in Human Erythroid Cells

Proceedings of the National Academy of Sciences - PNAS, 1988-12, Vol.85 (23), p.9062-9065

1988

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Molecular basis of hereditary elliptocytosis due to protein 4.1 deficiency

The New England journal of medicine, 1986-09, Vol.315 (11), p.680

1986

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926. Recombinant AAV Vectors Pseudotyped with Serotype-1 Capsid Mediates Early Onset of Gene Expression in Mouse Hearts

Molecular therapy, 2005-05, Vol.11 (S1), p.S358-S358

2005

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Molecular Cloning of Protein 4.1, a Major Structural Element of the Human Erythrocyte Membrane Skeleton

Proceedings of the National Academy of Sciences - PNAS, 1986-12, Vol.83 (24), p.9512-9516

1986

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Beta Thalassemia and Translation of Globin Messenger RNA

Proceedings of the National Academy of Sciences - PNAS, 1971-10, Vol.68 (10), p.2514-2518

1971

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Biogenesis of the Platelet Receptor for Fibrinogen: Evidence for Separate Precursors for Glycoproteins IIb and IIIa

Proceedings of the National Academy of Sciences - PNAS, 1986-03, Vol.83 (5), p.1480-1484

1986

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Survival of a hydropic infant with homozygous α-thalassemia-1

The Journal of pediatrics, 1986-05, Vol.108 (5), p.713-716

1986

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Human Placental Na+,K+-ATPase α Subunit: cDNA Cloning, Tissue Expression, DNA Polymorphism, and Chromosomal Localization

Proceedings of the National Academy of Sciences - PNAS, 1987-11, Vol.84 (22), p.7901-7905

1987

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Chromosomal Localization of the Human Apolipoprotein B Gene and Detection of Homologous RNA in Monkey Intestine

Proceedings of the National Academy of Sciences - PNAS, 1986-01, Vol.83 (2), p.419-422

1986

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Hemoglobin constant spring defined by specific oligonucleotide hybridization and hemoglobin D Punjab ( beta super(121) arrow right super(Gln)) in a Batak Indonesian family

American journal of hematology, 1988-01, Vol.29 (1), p.22-26

1988

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Assignment of Human $\beta $-, $\gamma $-, and $\delta $-globin Genes to the Short Arm of Chromosome 11 by Chromosome Sorting and DNA Restriction Enzyme Analysis

Proceedings of the National Academy of Sciences - PNAS, 1979-11, Vol.76 (11), p.5804-5808

1979

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Prediction of severity of disease in homozygous β thalassemia (β thal)

Pediatric research, 1971-08, Vol.5 (8), p.407-407

1971

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Hemoglobin constant spring defined by specific oligonucleotide hybridization and hemoglobin D Punjab (β121→Gln) in a Batak Indonesian family

American journal of hematology, 1988-09, Vol.29 (1), p.22-26

1988

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Seamless Gene Correction in the Human Cystic Fibrosis Transmembrane Conductance Regulator Locus by Vector Replacement and Vector Insertion Events

TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs

Induction of therapeutic levels of HbF in genome‐edited primary β039‐thalassaemia haematopoietic stem and progenitor cells

Induction of therapeutic levels of HbF in genome-edited primary β 0 39-thalassaemia haematopoietic stem and progenitor cells

Recombinant adeno-associated viral vector encoding human VEGF165 induces neomicrovessel formation in the adult mouse brain

Permanent coronary artery occlusion: cardiovascular MR imaging is platform for percutaneous transendocardial delivery and assessment of gene therapy in canine model

Adeno-associated viral vector-mediated gene transfer of VEGF normalizes skeletal muscle oxygen tension and induces arteriogenesis in ischemic rat hindlimb

Antibodies to Human Fetal Erythroid Cells from a Nonimmune Phage Antibody Library

228. AAV Mediated Hypoxia-Inducible VEGF Expression and Neovascular Formation in Ischemic Pig Myocardium

332. Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells

Cloning MafF by Recognition Site Screening with the NFE2 Tandem Repeat of HS2: Analysis of Its Role in Globin and GCSl Genes Regulation

Induction of therapeutic levels of HbF in genome-edited primary β0 39-thalassaemia haematopoietic stem and progenitor cells

Multiple Protein 4.1 Isoforms Produced by Alternative Splicing in Human Erythroid Cells

Molecular basis of hereditary elliptocytosis due to protein 4.1 deficiency

926. Recombinant AAV Vectors Pseudotyped with Serotype-1 Capsid Mediates Early Onset of Gene Expression in Mouse Hearts

Molecular Cloning of Protein 4.1, a Major Structural Element of the Human Erythrocyte Membrane Skeleton

Beta Thalassemia and Translation of Globin Messenger RNA

Biogenesis of the Platelet Receptor for Fibrinogen: Evidence for Separate Precursors for Glycoproteins IIb and IIIa

Survival of a hydropic infant with homozygous α-thalassemia-1

Human Placental Na+,K+-ATPase α Subunit: cDNA Cloning, Tissue Expression, DNA Polymorphism, and Chromosomal Localization

Chromosomal Localization of the Human Apolipoprotein B Gene and Detection of Homologous RNA in Monkey Intestine

Hemoglobin constant spring defined by specific oligonucleotide hybridization and hemoglobin D Punjab ( beta super(121) arrow right super(Gln)) in a Batak Indonesian family

Assignment of Human $\beta $-, $\gamma $-, and $\delta $-globin Genes to the Short Arm of Chromosome 11 by Chromosome Sorting and DNA Restriction Enzyme Analysis

Prediction of severity of disease in homozygous β thalassemia (β thal)

Hemoglobin constant spring defined by specific oligonucleotide hybridization and hemoglobin D Punjab (β121→Gln) in a Batak Indonesian family

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