Related references
Note: Only part of the references are listed.Drag-and-drop genome insertion of large sequences without double-strand DNA cleavage using CRISPR-directed integrases
Matthew T. N. Yarnall et al.
NATURE BIOTECHNOLOGY (2023)
CRISPR Editing Enables Consequential Tag-Activated MicroRNA-Mediated Endogene Deactivation
Panayiota L. Papasavva et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2022)
Inference of CRISPR Edits from Sanger Trace Data
David Conant et al.
CRISPR JOURNAL (2022)
Reconstructed glycosylase base editors GBE2.0 with enhanced C-to-G base editing efficiency and purity
Naxin Sun et al.
MOLECULAR THERAPY (2022)
MAJOR CRISPR PATENT DECISION WON'T END TANGLED DISPUTE
Heidi Ledford
NATURE (2022)
A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders
Francesca Tucci et al.
NATURE COMMUNICATIONS (2022)
Generation of C-to-G transversion in mouse embryos via CG editors
Tianqi Cao et al.
TRANSGENIC RESEARCH (2022)
Base editing in human cells with monomeric DddA-TALE fusion deaminases
Young Geun Mok et al.
NATURE COMMUNICATIONS (2022)
Ultra-deep sequencing validates safety of CRISPR/Cas9 genome editing in human hematopoietic stem and progenitor cells
M. Kyle Cromer et al.
NATURE COMMUNICATIONS (2022)
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression
Panagiotis Antoniou et al.
NATURE COMMUNICATIONS (2022)
Conversion of HbS to Hb G-Makassar By Adenine Base Editing Is Compatible with Normal Hemoglobin Function
S. Haihua Chu et al.
BLOOD (2021)
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
H. Frangoul et al.
NEW ENGLAND JOURNAL OF MEDICINE (2021)
Quantitative evaluation of chromosomal rearrangements in gene-edited human stem cells by CAST-Seq
Giandomenico Turchiano et al.
CELL STEM CELL (2021)
Distinct miRNA Signatures and Networks Discern Fetal from Adult Erythroid Differentiation and Primary from Immortalized Erythroid Cells
Panayiota L. Papasavva et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2021)
Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editing
Mitchell L. Leibowitz et al.
NATURE GENETICS (2021)
Base editing of haematopoietic stem cells rescues sickle cell disease in mice
Gregory A. Newby et al.
NATURE (2021)
Therapy Development by Genome Editing of Hematopoietic Stem Cells
Lola Koniali et al.
CELLS (2021)
Base and Prime Editing Technologies for Blood Disorders
Panagiotis Antoniou et al.
FRONTIERS IN GENOME EDITING (2021)
Opportunities and Challenges in the Delivery of mRNA-Based Vaccines
Abishek Wadhwa et al.
PHARMACEUTICS (2020)
Directed evolution of adenine base editors with increased activity and therapeutic application
Nicole M. Gaudelli et al.
NATURE BIOTECHNOLOGY (2020)
Dual base editor catalyzes both cytosine and adenine base conversions in human cells
Xiaohui Zhang et al.
NATURE BIOTECHNOLOGY (2020)
Therapeutic base editing of human hematopoietic stem cells
Jing Zeng et al.
NATURE MEDICINE (2020)
A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing
Beverly Y. Mok et al.
NATURE (2020)
Chemically modified mRNA nucleofection of primary human T cells
Nikolaus Thuille et al.
JOURNAL OF IMMUNOLOGICAL METHODS (2020)
Fetal Hemoglobin in Sickle Hemoglobinopathies: High HbF Genotypes and Phenotypes
Martin H. Steinberg
JOURNAL OF CLINICAL MEDICINE (2020)
Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9-Modified CD34+Hematopoietic Stem and Progenitor Cells
Haydar Frangoul et al.
BLOOD (2020)
A catalogue of biochemically diverse CRISPR-Cas9 orthologs
Giedrius Gasiunas et al.
NATURE COMMUNICATIONS (2020)
The genome editing revolution: review
Ahmad M. Khalil
JOURNAL OF GENETIC ENGINEERING AND BIOTECHNOLOGY (2020)
Highly efficient therapeutic gene editing of human hematopoietic stem cells
Yuxuan Wu et al.
NATURE MEDICINE (2019)
TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction
Christopher T. Lux et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2019)
Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas- and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells
Petros Patsali et al.
HAEMATOLOGICA (2019)
A novel mutation in the erythroid transcription factor KLF1 is likely responsible for ameliorating β-thalassemia major
Pavlos Fanis et al.
HUMAN MUTATION (2019)
Gene editing of PKLR gene in human hematopoietic progenitors through 5′ and 3′ UTR modified TALEN mRNA
Oscar Quintana-Bustamante et al.
PLOS ONE (2019)
The Scope for Thalassemia Gene Therapy by Disruption of Aberrant Regulatory Elements
Petros Patsali et al.
JOURNAL OF CLINICAL MEDICINE (2019)
Emergence of synthetic mRNA: In vitro synthesis of mRNA and its applications in regenerative medicine
Hyokyoung Kwon et al.
BIOMATERIALS (2018)
Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34+ Hematopoietic Stem and Progenitor Cells
M. Kyle Cromer et al.
MOLECULAR THERAPY (2018)
Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements
Michael Kosicki et al.
NATURE BIOTECHNOLOGY (2018)
Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction
Luke W. Koblan et al.
NATURE BIOTECHNOLOGY (2018)
Natural regulatory mutations elevate the fetal globin gene via disruption of BCL11A or ZBTB7A binding
Gabriella E. Martyn et al.
NATURE GENETICS (2018)
p53 inhibits CRISPR-Cas9 engineering in human pluripotent stem cells
Robert J. Ihry et al.
NATURE MEDICINE (2018)
CRISPR-Cas9 genome editing induces a p53-mediated DNA damage response
Emma Haapaniemi et al.
NATURE MEDICINE (2018)
CRISPR/Cas9 genome editing in human hematopoietic stem cells
Rasmus O. Bak et al.
NATURE PROTOCOLS (2018)
Engineering the Delivery System for CRISPR-Based Genome Editing
Zachary Glass et al.
TRENDS IN BIOTECHNOLOGY (2018)
Rapid and Sensitive Assessment of Globin Chains for Gene and Cell Therapy of Hemoglobinopathies
Constantinos C. Loucari et al.
HUMAN GENE THERAPY METHODS (2018)
Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification
Sriram Vaidyanathan et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2018)
EditR: A Method to Quantify Base Editing from Sanger Sequencing
Mitchell G. Kluesner et al.
CRISPR JOURNAL (2018)
Programmable base editing of A.T to G.C in genomic DNA without DNA cleavage
Nicole M. Gaudelli et al.
NATURE (2017)
Orexin activation counteracts decreases in nonexercise activity thermogenesis (NEAT) caused by high-fat diet
P. E. Bunney et al.
PHYSIOLOGY & BEHAVIOR (2017)
Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned
Richard A. Morgan et al.
CELL STEM CELL (2017)
Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
Alexis C. Komor et al.
NATURE (2016)
Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases
Carmen F. Bjurstrom et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2016)
BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis
Matthew C. Canver et al.
NATURE (2015)
Efficient delivery of nuclease proteins for genome editing in human stem cells and primary cells
Jia Liu et al.
NATURE PROTOCOLS (2015)
Controlled delivery of β-globin-targeting TALENs and CRISPR/Cas9 into mammalian cells for genome editing using microinjection
Renee N. Cottle et al.
SCIENTIFIC REPORTS (2015)
TALENs-mediated gene disruption of FLT3 in leukemia cells: Using genome-editing approach for exploring the molecular basis of gene abnormality
Jue Wang et al.
SCIENTIFIC REPORTS (2015)
CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes
Puping Liang et al.
PROTEIN & CELL (2015)
KLF1 mutations are relatively more common in a thalassemia endemic region and ameliorate the severity of β-thalassemia
Dun Liu et al.
BLOOD (2014)
Multiplex genomic structure variation mediated by TALEN and ssODN
Sanyuan Ma et al.
BMC GENOMICS (2014)
Determinants and implications of mRNA poly(A) tail size - Does this protein make my tail look big?
Aimee L. Jalkanen et al.
SEMINARS IN CELL & DEVELOPMENTAL BIOLOGY (2014)
TALENs and ZFNs are associated with different mutation signatures
Yongsub Kim et al.
NATURE METHODS (2013)
INNOVATION TALENs: a widely applicable technology for targeted genome editing
J. Keith Joung et al.
NATURE REVIEWS MOLECULAR CELL BIOLOGY (2013)
Establishment of Immortalized Human Erythroid Progenitor Cell Lines Able to Produce Enucleated Red Blood Cells
Ryo Kurita et al.
PLOS ONE (2013)
Ten novel mutations in the erythroid transcription factor KLF1 gene associated with increased fetal hemoglobin levels in adults
Alice E. Gallienne et al.
HAEMATOLOGICA-THE HEMATOLOGY JOURNAL (2012)
A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
Martin Jinek et al.
SCIENCE (2012)
A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity
Claudio Mussolino et al.
NUCLEIC ACIDS RESEARCH (2011)
Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors
Jens Boch et al.
SCIENCE (2009)
A Simple Cipher Governs DNA Recognition by TAL Effectors
Matthew J. Moscou et al.
SCIENCE (2009)
Highly efficient endogenous human gene correction using designed zinc-finger nucleases
FD Urnov et al.
NATURE (2005)
Novel anti-reverse cap analogs with superior translational properties
J Jemielity et al.
RNA (2003)
Share Paper
