Combining Single Strand Oligodeoxynucleotides and CRISPR/Cas9 to Correct Gene Mutations in β-Thalassemia-induced Pluripotent Stem Cells

Regulation of Gene Editing Activity Directed by Single-Stranded Oligonucleotides and CRISPR/Cas9 Systems

(2015) Pawel Bialk et al.   PLoS One

Improved Hematopoietic Differentiation Efficiency of Gene-Corrected Beta-Thalassemia Induced Pluripotent Stem Cells by CRISPR/Cas9 System

(2015) Bing Song et al.   STEM CELLS AND DEVELOPMENT

Both TALENs and CRISPR/Cas9 directly target the HBB IVS2–654 (C > T) mutation in β-thalassemia-derived iPSCs

(2015) Peng Xu et al.   Scientific Reports

Naïve Induced Pluripotent Stem Cells Generated From β-Thalassemia Fibroblasts Allow Efficient Gene Correction With CRISPR/Cas9

(2015) Yuanyuan Yang et al.   Stem Cells Translational Medicine

Targeted Gene Correction Minimally Impacts Whole-Genome Mutational Load in Human-Disease-Specific Induced Pluripotent Stem Cell Clones

(2014) Keiichiro Suzuki et al.   Cell Stem Cell

TALEN-Mediated Generation and Genetic Correction of Disease-Specific Human Induced Pluripotent Stem Cells

(2014) Sivaprakash Ramalingam et al.   CURRENT GENE THERAPY

Recent trends for novel options in experimental biological therapy of β-thalassemia

(2014) Alessia Finotti et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 andpiggyBac

(2014) Fei Xie et al.   GENOME RESEARCH

CRISPR/Cas9 for genome editing: progress, implications and challenges

(2014) F. Zhang et al.   HUMAN MOLECULAR GENETICS

Precise Gene Modification Mediated by TALEN and Single-Stranded Oligodeoxynucleotides in Human Cells

(2014) Xiaoling Wang et al.   PLoS One

Rapid generation of mouse models with defined point mutations by the CRISPR/Cas9 system

(2014) Masafumi Inui et al.   Scientific Reports

Seamless correction of the sickle cell disease mutation of theHBBgene in human induced pluripotent stem cells using TALENs

(2013) Ning Sun et al.   BIOTECHNOLOGY AND BIOENGINEERING

Transcription Activator-like Effector Nuclease (TALEN)-mediated Gene Correction in Integration-free β-Thalassemia Induced Pluripotent Stem Cells

(2013) Ning Ma et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Genetic correction of β-thalassemia patient-specific iPS cells and its use in improving hemoglobin production in irradiated SCID mice

(2012) Yixuan Wang et al.   CELL RESEARCH

Optimized TAL effector nucleases (TALENs) for use in treatment of sickle cell disease

(2012) Ning Sun et al.   Molecular BioSystems

Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease

(2011) J. Zou et al.   BLOOD

Homologous Recombination in Human Embryonic Stem Cells: A Tool for Advancing Cell Therapy and Understanding and Treating Human Disease

(2011) Andrew D. Leavitt et al.   CTS-Clinical and Translational Science

An unbiased genome-wide analysis of zinc-finger nuclease specificity

(2011) Richard Gabriel et al.   NATURE BIOTECHNOLOGY

Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection

(2011) Vikram Pattanayak et al.   NATURE METHODS

In Situ Genetic Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells Using Engineered Zinc Finger Nucleases

(2011) Vittorio Sebastiano et al.   STEM CELLS

Beta-thalassemia

(2010) Antonio Cao et al.   GENETICS IN MEDICINE

Generation of induced pluripotent stem cells from human β-thalassemia fibroblast cells

(2009) Yixuan Wang et al.   CELL RESEARCH

Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases

(2009) L. Ye et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA