CRISPR/Cas9 system: A promising technology for the treatment of inherited and neoplastic hematological diseases
Explore this paper's citation graph
Summary
The utilization of the CRISPR/Cas9 system as a possible treatment option for hemoglobinopathies and hematological tumors and its therapeutic potential for genome editing in cells from hematopoietic origin are focused on.
- Type
- article
- Published
- 2018-05-01
- Cited by
- 19
- References
- 91
- Access
- Open access
- OpenAlex
- https://openalex.org/W2807464733
- Semantic Scholar
- https://api.semanticscholar.org/CorpusID:91068969
Keywords
CRISPR, Genome editing, Zinc finger nuclease, Cas9, Biology
References
- Gene Therapy for Hemoglobinopathies
- Both TALENs and CRISPR/Cas9 directly target the HBB IVS2–654 (C > T) mutation in β-thalassemia-derived iPSCs
- Generation of mouse models of myeloid malignancy with combinatorial genetic lesions using CRISPR-Cas9 genome editing
- BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis
- Pharmacological inhibition of DNA-PK stimulates Cas9-mediated genome editing
- Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease.
- Regulated expression of genes inserted at the human chromosomal beta-globin locus by homologous recombination.
- IthaGenes: An Interactive Database for Haemoglobin Variations and Epidemiology
- Reawakening fetal hemoglobin: prospects for new therapies for the β-globin disorders.
- GUIDE-Seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases
- Inhibition of non-homologous end joining increases the efficiency of CRISPR/Cas9-mediated precise [TM: inserted] genome editing
- In Situ Genetic Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells Using Engineered Zinc Finger Nucleases
- Correction of Sickle Cell Disease in Adult Mice by Interference with Fetal Hemoglobin Silencing
- Heterocellular hereditary persistence of fetal haemoglobin affects the haematological parameters of β‐thalassaemia trait
- Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells
- High frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells
- Improved Hematopoietic Differentiation Efficiency of Gene-Corrected Beta-Thalassemia Induced Pluripotent Stem Cells by CRISPR/Cas9 System
- Production of gene-corrected adult beta globin protein in human erythrocytes differentiated from patient iPSCs after genome editing of the sickle point mutation
- Control of large, established tumor xenografts with genetically retargeted human T cells containing CD28 and CD137 domains
- Improving CRISPR-Cas nuclease specificity using truncated guide RNAs
Cited by
- CRISPR/Cas9-modified hematopoietic stem cells—present and future perspectives for stem cell transplantation
- Targeted deletion of BCL11A gene by CRISPR-Cas9 system for fetal hemoglobin reactivation: A promising approach for gene therapy of beta thalassemia disease.
- Hematopoietic stem cell gene therapy: The optimal use of lentivirus and gene editing approaches.
- Recent advances in CRISPR/Cas9-mediated knock-ins in mammalian cells.
- CRISPR/Cas9 technology: towards a new generation of improved CAR-T cells for anticancer therapies.
- Comparative analysis of lentiviral gene transfer approaches designed to promote fetal hemoglobin production for the treatment of β-hemoglobinopathies.
- Gene editing technology for improving life quality: A dream coming true?
- Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34+ HSPCs by CRISPR/Cas9 for the induction of fetal hemoglobin
- Progress and challenges in CRISPR-mediated therapeutic genome editing for monogenic diseases
- Hematopoietic stem cell gene therapy for the treatment of β-hemoglobinopathies
- Diversification of the CRISPR Toolbox: Applications of CRISPR-Cas Systems Beyond Genome Editing
- The Potential of CRISPR/Cas9 Gene Editing as a Treatment Strategy for Inherited Diseases
- A Mutation-Agnostic Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy
- Applying stem cell therapy in intractable diseases: a narrative review of decades of progress and challenges
- CRISPR medicine for blood disorders: Progress and challenges in delivery
- CRISPR-CAS9-MEDIATED EX VIVO GENE EDITING FOR INHERITED HEMATOLOGICAL DISORDERS: ADVANCEMENTS, CHALLENGES, AND CLINICAL POTENTIAL
- Optimizing cancer treatment: the synergistic potential of CAR-T cell therapy and CRISPR/Cas9
- CRISPR-Cas9: a prominent genome editing tool in the management of inherited blood disorders and hematological malignancies.
- Most Recent Clinical Advances in CAR T Cell and Gene Therapy 2017/2018
Related papers
- Genome engineering with targetable nucleases (467.1)
- Comparing Zinc Finger Nucleases and Transcription Activator-Like Effector Nucleases for Gene Targeting in Drosophila
- Current and future delivery systems for engineered nucleases: ZFN, TALEN and RGEN.
- Temperature effect on CRISPR-Cas9 mediated genome editing.
- Programmable site-specific DNA endonucleases:powerful tools for genome engineering
- Biological and Biomedical Applications of Engineered Nucleases
- JoVE Video Dataset
- NgAgo:A New Genome Editor
- A guide to genome engineering with programmable nucleases
- Mouse Genome Engineering Using Designer Nucleases