Cargando…

Highly efficient therapeutic gene editing of human hematopoietic stem cells

Re-expression of the paralogous γ-globin genes (HBG1/2) could be a universal strategy to ameliorate the severe β-globin disorders sickle cell disease (SCD) and β-thalassemia by induction of fetal hemoglobin (HbF, α(2)γ(2))(1). Previously we and others have shown that core sequences at the BCL11A ery...

Descripción completa

Detalles Bibliográficos
Autores principales:	Wu, Yuxuan, Zeng, Jing, Roscoe, Benjamin P., Liu, Pengpeng, Yao, Qiuming, Lazzarotto, Cicera R., Clement, M. Kendell, Cole, Mitchel A., Luk, Kevin, Baricordi, Cristina, Shen, Anne H., Ren, Chunyan, Esrick, Erica B., Manis, John P., Dorfman, David M., Williams, David A., Biffi, Alessandra, Brugnara, Carlo, Biasco, Luca, Brendel, Christian, Pinello, Luca, Tsai, Shengdar Q., Wolfe, Scot A., Bauer, Daniel E.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	2019
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6512986/ https://www.ncbi.nlm.nih.gov/pubmed/30911135 http://dx.doi.org/10.1038/s41591-019-0401-y

Ejemplares similares

Retroviral Integrations in Gene Therapy Trials
por: Biasco, Luca, et al.
Publicado: (2012)

AmpUMI: design and analysis of unique molecular identifiers for deep amplicon sequencing
por: Clement, Kendell, et al.
Publicado: (2018)

A comprehensive single cell transcriptional landscape of human hematopoietic progenitors
por: Pellin, Danilo, et al.
Publicado: (2019)

IS-Seq: a bioinformatics pipeline for integration sites analysis with comprehensive abundance quantification methods
por: Yan, Aimin, et al.
Publicado: (2023)

An APOBEC3A-Cas9 base editor with minimized bystander and off-target activities
por: Gehrke, Jason M., et al.
Publicado: (2018)

Rational targeting of a NuRD subcomplex guided by comprehensive in situ mutagenesis
por: Sher, Falak, et al.
Publicado: (2019)

CRISPRO: identification of functional protein coding sequences based on genome editing dense mutagenesis
por: Schoonenberg, Vivien A. C., et al.
Publicado: (2018)

Investigational curative gene therapy approaches to sickle cell disease
por: Williams, David A., et al.
Publicado: (2021)

Precise therapeutic gene correction by a simple nuclease-induced double-strand break
por: Iyer, Sukanya, et al.
Publicado: (2019)

Assessment of computational methods for the analysis of single-cell ATAC-seq data
por: Chen, Huidong, et al.
Publicado: (2019)

Enhanced Cas12a editing in mammalian cells and zebrafish
por: Liu, Pengpeng, et al.
Publicado: (2019)

Orthogonal Cas9–Cas9 chimeras provide a versatile platform for genome editing
por: Bolukbasi, Mehmet Fatih, et al.
Publicado: (2018)

Publisher Correction: Orthogonal Cas9-Cas9 chimeras provide a versatile platform for genome editing
por: Bolukbasi, Mehmet Fatih, et al.
Publicado: (2018)

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

Efficient Homology-Directed Repair with Circular Single-Stranded DNA Donors
por: Iyer, Sukanya, et al.
Publicado: (2022)

Base editing as a genetic treatment for spinal muscular atrophy
por: Alves, Christiano R. R., et al.
Publicado: (2023)

Variant-aware saturating mutagenesis using multiple Cas9 nucleases identifies regulatory elements at trait-associated loci
por: Canver, Matthew C., et al.
Publicado: (2017)

In vivo CRISPR editing with no detectable genome-wide off-target mutations
por: Akcakaya, Pinar, et al.
Publicado: (2018)

Prime editing in mice reveals the essentiality of a single base in driving tissue-specific gene expression
por: Gao, Pan, et al.
Publicado: (2021)

A brown fat-enriched adipokine, ASRA, is a leptin receptor antagonist that stimulates appetite
por: Huang, Lei, et al.
Publicado: (2023)

Long-term lymphoid progenitors independently sustain naïve T and NK cell production in humans
por: Izotova, Natalia, et al.
Publicado: (2021)

Augmenting and directing long-range CRISPR-mediated activation in human cells
por: Tak, Y. Esther, et al.
Publicado: (2021)

Reducing the inherent auto-inhibitory interaction within the pegRNA enhances prime editing efficiency
por: Ponnienselvan, Karthikeyan, et al.
Publicado: (2023)

Production and characterization of virus-free, CRISPR-CAR T cells capable of inducing solid tumor regression
por: Mueller, Katherine P, et al.
Publicado: (2022)

Transcription factor competition at the γ-globin promoters controls hemoglobin switching
por: Liu, Nan, et al.
Publicado: (2021)

CHANGE-seq reveals genetic and epigenetic effects on CRISPR-Cas9 genome-wide activity
por: Lazzarotto, Cicera R., et al.
Publicado: (2020)

A Rare Presentation of Rickets Mimicking Sacroiliitis: A Case Report and Literature Review
por: Ali, Rafal S, et al.
Publicado: (2022)

Analyzing the Genotoxicity of Retroviral Vectors in Hematopoietic Cell Gene Therapy
por: Biasco, Luca, et al.
Publicado: (2017)

A methodology to assess the intrinsic discriminative ability of a distance function and its interplay with clustering algorithms for microarray data analysis
por: Giancarlo, Raffaele, et al.
Publicado: (2013)

Predicting chromatin organization using histone marks
por: Huang, Jialiang, et al.
Publicado: (2015)

GiniClust: detecting rare cell types from single-cell gene expression data with Gini index
por: Jiang, Lan, et al.
Publicado: (2016)

Loss-of-function lesions impact B-cell development and fitness but are insufficient to drive CLL in mouse models
por: Hacken, Elisa ten, et al.
Publicado: (2022)

Lipophilic dye-compatible brain clearing technique allowing correlative magnetic resonance/high-resolution fluorescence imaging in rat models of glioblastoma
por: Peviani, Marco, et al.
Publicado: (2020)

High throughput single-cell detection of multiplex CRISPR-edited gene modifications
por: ten Hacken, Elisa, et al.
Publicado: (2020)

Optimal crystal surface for efficient channeling in the new generation of hadron machines
por: Baricordi, S
Publicado: (2007)

LONP-1 and ATFS-1 sustain deleterious heteroplasmy by promoting mtDNA replication in dysfunctional mitochondria
por: Yang, Qiyuan, et al.
Publicado: (2022)

A motif-independent metric for DNA sequence specificity
por: Pinello, Luca, et al.
Publicado: (2011)

GRAFIMO: Variant and haplotype aware motif scanning on pangenome graphs
por: Tognon, Manuel, et al.
Publicado: (2021)

Engineered CRISPR-Cas12a variants with increased activities and improved targeting ranges for gene, epigenetic and base editing
por: Kleinstiver, Benjamin P., et al.
Publicado: (2019)

CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells
por: Brault, Julie, et al.
Publicado: (2023)

Cannot write session to /tmp/vufind_sessions/sess_ekkn41gf9nqoec86rluuljvdfp