Cargando…

βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid Cells

Lentiviral addition of βT87Q-globin, a modified β-globin with an anti-sickling mutation, is currently being used in gene therapy trials for sickle cell disease (SCD) and β-thalassemia patients. βT87Q-globin interferes with sickle hemoglobin (HbS) polymerization. Here, we generated the SCD mutation i...

Descripción completa

Detalles Bibliográficos
Autores principales:	Demirci, Selami, Gudmundsdottir, Bjorg, Li, Quan, Haro-Mora, Juan J., Nassehi, Tina, Drysdale, Claire, Yapundich, Morgan, Gamer, Jackson, Seifuddin, Fayaz, Tisdale, John F., Uchida, Naoya
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7210457/ https://www.ncbi.nlm.nih.gov/pubmed/32405513 http://dx.doi.org/10.1016/j.omtm.2020.04.013

Ejemplares similares

Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease
por: Uchida, Naoya, et al.
Publicado: (2021)

High-Efficiency Lentiviral Transduction of Human CD34(+) Cells in High-Density Culture with Poloxamer and Prostaglandin E2
por: Uchida, Naoya, et al.
Publicado: (2019)

Low-Dose Busulfan Reduces Human CD34(+) Cell Doses Required for Engraftment in c-kit Mutant Immunodeficient Mice
por: Leonard, Alexis, et al.
Publicado: (2019)

Preclinical evaluation for engraftment of CD34(+) cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models
por: Uchida, Naoya, et al.
Publicado: (2021)

CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease
por: Demirci, Selami, et al.
Publicado: (2021)

Biallelic correction of sickle cell disease‐derived induced pluripotent stem cells (iPSCs) confirmed at the protein level through serum‐free iPS‐sac/erythroid differentiation
por: Haro‐Mora, Juan J., et al.
Publicado: (2020)

Serum-free Erythroid Differentiation for Efficient Genetic Modification and High-Level Adult Hemoglobin Production
por: Uchida, Naoya, et al.
Publicado: (2018)

Genetic reversal of the globin switch concurrently modulates both fetal and sickle hemoglobin and reduces red cell sickling
por: De Souza, Daniel C., et al.
Publicado: (2023)

Salubrinal induces fetal hemoglobin expression via the stress-signaling pathway in human sickle erythroid progenitors and sickle cell disease mice
por: Lopez, Nicole H., et al.
Publicado: (2022)

SGK1 inhibition induces fetal hemoglobin expression and delays polymerization in sickle erythroid cells
por: Hara, Yannis, et al.
Publicado: (2023)

Sickle cell anemia: clinical diversity and beta S-globin haplotypes
por: Loggetto, Sandra Regina
Publicado: (2013)

THE STATE OF HEMOGLOBIN IN SICKLED ERYTHROCYTES
por: Stetson, Chandler A.
Publicado: (1966)

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype
por: Weber, Leslie, et al.
Publicado: (2020)

Sickle Cell Disease and Fetal Hemoglobin
por: Rivera, Alicia
Publicado: (2018)

Biochemical and Molecular analysis of the beta-globin gene on Saudi sickle cell anemia
por: Alenzi, Faris Q., et al.
Publicado: (2019)

Frequency of beta S globin gene haplotypes among sickle cell patients in Nigeria
por: Adabale, Abosede, et al.
Publicado: (2021)

Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease
por: Poletti, Valentina, et al.
Publicado: (2018)

GELS OF NORMAL AND SICKLED HEMOGLOBIN : COMPARATIVE STUDY
por: White, James G., et al.
Publicado: (1970)

Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders
por: Uchida, Naoya, et al.
Publicado: (2019)

Pure Erythroid Leukemia in a Sickle Cell Patient Treated with Hydroxyurea
por: Yadav, Dhiraj Kumar, et al.
Publicado: (2020)

Genetic variants of PKLR are associated with acute pain in sickle cell disease
por: Wang, Xunde, et al.
Publicado: (2022)

Hemoglobin switching in sheep and goats. VI. Commitment of erythroid colony-forming cells to the synthesis of betaC globin
Publicado: (1976)

Hemoglobin switching in sheep and goats. V. Effect of erythropoietin concentration on in vitro erythroid colony growth and globin synthesis
Publicado: (1975)

Influence of βS-Globin Haplotypes and Hydroxyurea on Arginase I Levels in Sickle Cell Disease
por: Moreira, J. A., et al.
Publicado: (2016)

Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations
por: Woodard, Kaitly J., et al.
Publicado: (2022)

A Triazole Disulfide Compound Increases the Affinity of Hemoglobin for Oxygen and Reduces the Sickling of Human Sickle Cells
por: Nakagawa, Akito, et al.
Publicado: (2018)

Fetal hemoglobin and hemolysis markers in sickle cell anemia()
por: Colella, Marina Pereira, et al.
Publicado: (2015)

Rapid and inefficient kinetics of sickle hemoglobin fiber growth
por: Castle, Brian T., et al.
Publicado: (2019)

Hemoglobin level and macular thinning in sickle cell disease
por: Hussnain, S Amal, et al.
Publicado: (2019)

Erythroid Adhesion Molecules in Sickle Cell Anaemia Infants: Insights Into Early Pathophysiology
por: Brousse, Valentine, et al.
Publicado: (2014)

High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation
por: Magis, Wendy, et al.
Publicado: (2022)

Beta-S globin haplotypes in patients with sickle cell anemia: one approach to understand the diversity in Brazil
por: Okumura, Jéssika Viviani, et al.
Publicado: (2013)

New variants in beta globin gene among the Palestinian refugees with sickle cell disease in Lebanon
por: Moussa, Esraa Y., et al.
Publicado: (2018)

Fluorescence Lifetime Measurement of Prefibrillar Sickle Hemoglobin Oligomers as a Platform for Drug Discovery in Sickle Cell Disease
por: Vunnam, Nagamani, et al.
Publicado: (2022)

THE PHYSICAL STATE OF HEMOGLOBIN IN SICKLE-CELL ANEMIA ERYTHROCYTES IN VIVO
por: Döbler, Johanna, et al.
Publicado: (1968)

Sickle Retinopathy in a Person with Hemoglobin S/New York Disease
por: Calder, Donovan, et al.
Publicado: (2012)

The Sickle Effect: The Silent Titan Affecting Glycated Hemoglobin Reliability
por: Gordon, Domonick K, et al.
Publicado: (2020)

Fetal hemoglobin rescues ineffective erythropoiesis in sickle cell disease
por: El Hoss, Sara, et al.
Publicado: (2020)

Hemoglobin and End-Organ Damage in Individuals with Sickle Cell Disease
por: Ershler, William B., et al.
Publicado: (2023)

Influence of βS-globin haplotypes hydroxyurea on tumor necrosis factor-alpha levels in sickle cell anemia
por: Laurentino, Marília Rocha, et al.
Publicado: (2014)

Cannot write session to /tmp/vufind_sessions/sess_cl3558dcp3jp5k2nojslcjkq5i