Cargando…

Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease

Gene editing with the CRISPR-Cas9 system could revolutionize hematopoietic stem cell (HSC)-targeted gene therapy for hereditary diseases, including sickle cell disease (SCD). Conventional delivery of editing tools by electroporation limits HSC fitness due to its toxicity; therefore, efficient and no...

Descripción completa

Detalles Bibliográficos
Autores principales:	Uchida, Naoya, Drysdale, Claire M., Nassehi, Tina, Gamer, Jackson, Yapundich, Morgan, DiNicola, Julia, Shibata, Yoshitaka, Hinds, Malikiya, Gudmundsdottir, Bjorg, Haro-Mora, Juan J., Demirci, Selami, Tisdale, John F.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2021
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8005818/ https://www.ncbi.nlm.nih.gov/pubmed/33816645 http://dx.doi.org/10.1016/j.omtm.2021.02.022

Ejemplares similares

βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid Cells
por: Demirci, Selami, et al.
Publicado: (2020)

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)

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)

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

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

Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell Disease
por: Germino-Watnick, Paula, et al.
Publicado: (2022)

Definitive hematopoietic stem/progenitor cells from human embryonic stem cells through serum/feeder-free organoid-induced differentiation
por: Demirci, Selami, et al.
Publicado: (2020)

Correction to: Definitive hematopoietic stem/progenitor cells from human embryonic stem cells through serum/feeder-free organoid-induced differentiation
por: Demirci, Selami, 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)

Decitabine Suspends Human CD34(+) Cell Differentiation and Proliferation during Lentiviral Transduction
por: Uchida, Naoya, et al.
Publicado: (2014)

Toward effective hematopoietic stem cell gene therapies: Optimized conditioning regimen and stem cell source in harmony
por: Demirci, Selami, et al.
Publicado: (2023)

Immunoresponse to Gene-Modified Hematopoietic Stem Cells
por: Drysdale, Claire M., et al.
Publicado: (2019)

Hematopoietic stem cells from pluripotent stem cells: Clinical potential, challenges, and future perspectives
por: Demirci, Selami, et al.
Publicado: (2020)

Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model
por: Uchida, Naoya, et al.
Publicado: (2023)

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype
por: Weber, Leslie, et al.
Publicado: (2018)

Integration-specific In Vitro Evaluation of Lentivirally Transduced Rhesus CD34(+) Cells Correlates With In Vivo Vector Copy Number
por: Uchida, Naoya, et al.
Publicado: (2013)

Forced enhancer-promoter rewiring to alter gene expression in animal models
por: Peslak, Scott A., et al.
Publicado: (2023)

Pre-existing immunity does not impair the engraftment of CRISPR-Cas9-edited cells in rhesus macaques conditioned with busulfan or radiation
por: Essawi, Khaled, et al.
Publicado: (2023)

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)

Urinary cell‐free extrachromosomal circular DNAs: A possible biomarker for chronic kidney disease
por: Demirci, Selami
Publicado: (2022)

Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of β-Thalassemia and Sickle Cell Disease
por: Negre, Olivier, et al.
Publicado: (2015)

Role for Gag-CA Interdomain Linker in Primate Lentiviral Replication
por: Doi, Naoya, et al.
Publicado: (2019)

Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector
por: Kabadi, Ami M., et al.
Publicado: (2014)

Hit and go CAS9 delivered through a lentiviral based self-limiting circuit
por: Petris, Gianluca, et al.
Publicado: (2017)

Lentiviral Vectors for Delivery of Gene-Editing Systems Based on CRISPR/Cas: Current State and Perspectives
por: Dong, Wendy, et al.
Publicado: (2021)

Safe and efficient peripheral blood stem cell collection in patients with sickle cell disease using plerixafor
por: Uchida, Naoya, et al.
Publicado: (2020)

In Vivo Knockout of the Vegfa Gene by Lentiviral Delivery of CRISPR/Cas9 in Mouse Retinal Pigment Epithelium Cells
por: Holmgaard, Andreas, et al.
Publicado: (2017)

Lovo‐cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB‐206 study
por: Kanter, Julie, et al.
Publicado: (2022)

Production of lentiviral vectors
por: Merten, Otto-Wilhelm, et al.
Publicado: (2016)

Lentiviral Vector Bioprocessing
por: Perry, Christopher, et al.
Publicado: (2021)

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy
por: Brendel, Christian, et al.
Publicado: (2020)

Robust generation of erythroid and multilineage hematopoietic progenitors from human iPSCs using a scalable monolayer culture system
por: Ruiz, Juan Pablo, et al.
Publicado: (2019)

Integrase-Deficient Lentiviral Vector as an All-in-One Platform for Highly Efficient CRISPR/Cas9-Mediated Gene Editing
por: Ortinski, Pavel I., et al.
Publicado: (2017)

Expression Profiles of Vpx/Vpr Proteins Are Co-related with the Primate Lentiviral Lineage
por: Sakai, Yosuke, et al.
Publicado: (2016)

CRISPR-Cas9-directed gene tagging using a single integrase-defective lentiviral vector carrying a transposase-based Cas9 off switch
por: Thomsen, Emil Aagaard, et al.
Publicado: (2022)

The Inside Out of Lentiviral Vectors
por: Durand, Stéphanie, et al.
Publicado: (2011)

Clinical use of lentiviral vectors
por: Milone, Michael C., et al.
Publicado: (2018)

Roles for endocytosis in lentiviral replication
por: Marsh, Mark, et al.
Publicado: (1997)

Cannot write session to /tmp/vufind_sessions/sess_gapp1nfiakkokilimj9em8orsc