Cargando…

High-efficiency editing in hematopoietic stem cells and the HUDEP-2 cell line based on in vitro mRNA synthesis

Introduction: Genome editing tools, such as CRISPR/Cas, TALE nucleases and, more recently, double-strand-break-independent editors, have been successfully used for gene therapy and reverse genetics. Among various challenges in the field, tolerable and efficient delivery of editors to target cells an...

Descripción completa

Detalles Bibliográficos
Autores principales:	Papaioannou, Nikoletta Y., Patsali, Petros, Naiisseh, Basma, Papasavva, Panayiota L., Koniali, Lola, Kurita, Ryo, Nakamura, Yukio, Christou, Soteroula, Sitarou, Maria, Mussolino, Claudio, Cathomen, Toni, Kleanthous, Marina, Lederer, Carsten W.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2023
Materias:	Genome Editing
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10030607/ https://www.ncbi.nlm.nih.gov/pubmed/36969374 http://dx.doi.org/10.3389/fgeed.2023.1141618

Ejemplares similares

Distinct miRNA Signatures and Networks Discern Fetal from Adult Erythroid Differentiation and Primary from Immortalized Erythroid Cells
por: Papasavva, Panayiota L., et al.
Publicado: (2021)

The Scope for Thalassemia Gene Therapy by Disruption of Aberrant Regulatory Elements
por: Patsali, Petros, et al.
Publicado: (2019)

Relative and Absolute Quantification of Aberrant and Normal Splice Variants in HBB(IVSI−110 (G > A)) β-Thalassemia
por: Patsali, Petros, et al.
Publicado: (2020)

CRISPR Editing Enables Consequential Tag-Activated MicroRNA-Mediated Endogene Deactivation
por: Papasavva, Panayiota L., et al.
Publicado: (2022)

Rapid and Sensitive Assessment of Globin Chains for Gene and Cell Therapy of Hemoglobinopathies
por: Loucari, Constantinos C., et al.
Publicado: (2018)

Therapy Development by Genome Editing of Hematopoietic Stem Cells
por: Koniali, Lola, et al.
Publicado: (2021)

Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies
por: Lederer, Carsten W., et al.
Publicado: (2022)

Effect of HBB genotype on survival in a cohort of transfusion-dependent thalassemia patients in Cyprus
por: Kountouris, Petros, et al.
Publicado: (2020)

Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases
por: Papasavva, Panayiota, et al.
Publicado: (2019)

Genome Editing With TALEN, CRISPR-Cas9 and CRISPR-Cas12a in Combination With AAV6 Homology Donor Restores T Cell Function for XLP
por: Houghton, Benjamin C., et al.
Publicado: (2022)

Robust CRISPR/Cas9 Genome Editing of the HUDEP-2 Erythroid Precursor Line Using Plasmids and Single-Stranded Oligonucleotide Donors
por: Moir-Meyer, Gemma, et al.
Publicado: (2018)

Disruptive Technology: CRISPR/Cas-Based Tools and Approaches
por: Patsali, Petros, et al.
Publicado: (2019)

A novel mutation in the erythroid transcription factor KLF1 is likely responsible for ameliorating β‐thalassemia major
por: Fanis, Pavlos, et al.
Publicado: (2019)

Genetic Modifiers at the Crossroads of Personalised Medicine for Haemoglobinopathies
por: Stephanou, Coralea, et al.
Publicado: (2019)

Traceless Targeting and Isolation of Gene-Edited Immortalized Keratinocytes from Epidermolysis Bullosa Simplex Patients
por: Aushev, Magomet, et al.
Publicado: (2017)

Recent Approaches for Manipulating Globin Gene Expression in Treating Hemoglobinopathies
por: Mussolino, Claudio, et al.
Publicado: (2021)

The molecular spectrum and distribution of haemoglobinopathies in Cyprus: a 20-year retrospective study
por: Kountouris, Petros, et al.
Publicado: (2016)

T-CAST: An optimized CAST-Seq pipeline for TALEN confirms superior safety and efficacy of obligate-heterodimeric scaffolds
por: Rhiel, Manuel, et al.
Publicado: (2023)

Genome Editing of the SNAI1 Gene in Rhabdomyosarcoma: A Novel Model for Studies of Its Role
por: Ulman, Aleksandra, et al.
Publicado: (2020)

A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity
por: Mussolino, Claudio, et al.
Publicado: (2011)

Sex-specific transcriptional profiles identified in β-thalassemia patients
por: Nanou, Aikaterini, et al.
Publicado: (2021)

Editorial: Mutation-Specific Gene Editing for Blood Disorders
por: Lederer, Carsten Werner, et al.
Publicado: (2021)

Chimerization Enables Gene Synthesis and Lentiviral Delivery of Customizable TALE-Based Effectors
por: Fang, Yongxing, et al.
Publicado: (2020)

Targeted genome editing restores T cell differentiation in a humanized X-SCID pluripotent stem cell disease model
por: Alzubi, Jamal, et al.
Publicado: (2017)

Measurement of lentiviral vector titre and copy number by cross-species duplex quantitative PCR
por: Christodoulou, I, et al.
Publicado: (2016)

Inactivation of Hepatitis B Virus Replication in Cultured Cells and In Vivo with Engineered Transcription Activator-Like Effector Nucleases
por: Bloom, Kristie, et al.
Publicado: (2013)

TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity
por: Mussolino, Claudio, et al.
Publicado: (2014)

TALEN-mediated functional correction of human iPSC-derived macrophages in context of hereditary pulmonary alveolar proteinosis
por: Kuhn, Alexandra, et al.
Publicado: (2017)

Inhibition of replication of hepatitis B virus using transcriptional repressors that target the viral DNA
por: Bloom, Kristie, et al.
Publicado: (2019)

Hematopoietic stem and progenitors cells gene editing: Beyond blood disorders
por: Buffa, Valentina, et al.
Publicado: (2023)

Gene Editing of Hematopoietic Stem Cells: Hopes and Hurdles Toward Clinical Translation
por: Ferrari, Samuele, et al.
Publicado: (2021)

Genome editing in cereal crops: an overview
por: Matres, Jerlie Mhay, et al.
Publicado: (2021)

Current approaches and potential challenges in the delivery of gene editing cargos into hematopoietic stem and progenitor cells
por: Murugesan, Ramya, et al.
Publicado: (2023)

Prime editing in hematopoietic stem cells—From ex vivo to in vivo CRISPR-based treatment of blood disorders
por: Wolff, Jonas Holst, et al.
Publicado: (2023)

Allele-Specific Disruption of a Common STAT3 Autosomal Dominant Allele Is Not Sufficient to Restore Downstream Signaling in Patient-Derived T Cells
por: König, Saskia, et al.
Publicado: (2022)

Global and Local Manipulation of DNA Repair Mechanisms to Alter Site-Specific Gene Editing Outcomes in Hematopoietic Stem Cells
por: Benitez, Elizabeth K., et al.
Publicado: (2020)

Efficient and error-free correction of sickle mutation in human erythroid cells using prime editor-2
por: George, Anila, et al.
Publicado: (2022)

New Edition
Publicado: (1952)

New Editions
Publicado: (1951)

Grand Challenges in Genome Editing in Plants
por: Yang, Bing
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_ikr6lhlkbhmk6f7pig6nl8d5os