CRISPR-mediated gene editing for the surgeon scientist.
CRISPR to the Rescue: Advances in Gene Editing for the FMR1 Gene
- BiologyBrain sciences
A review of reports using CRISPR-editing strategies to target Fragile X syndrome (FXS) molecular dysregulation, and highlights how differences in FXS and FragileX-associated Tremor/Ataxia Syndrome (FXTAS) might alter treatment strategies for each syndrome.
Applications of PNA-laden nanoparticles for hematological disorders
- Biology, ChemistryCellular and Molecular Life Sciences
This review comprehensively covers the advances made in peptide nucleic acid-based TFOs for site-specific gene editing and their therapeutic applications.
CRISPR/Cas system: A game changing genome editing technology, to treat human genetic diseases.
Genome-Wide Analysis of Off-Target CRISPR/Cas9 Activity in Single-Cell-Derived Human Hematopoietic Stem and Progenitor Cell Clones
The collective somatic mutational burden observed within Cas9 RNP-edited human HSPC clones is indistinguishable from naturally occurring levels of background genetic heterogeneity.
CRISPR/Cas-Based Gene Editing Strategies for DOCK8 Immunodeficiency Syndrome
- Medicine, BiologyFrontiers in Genome Editing
The findings encourage the pursuit of CRISPR/Cas-based gene editing approaches, which may constitute more precise, affordable, and low-risk definitive treatment options for DOCK8 deficiency.
CRISPR/Cas9 for Sickle Cell Disease: Applications, Future Possibilities, and Challenges.
- Biology, MedicineAdvances in experimental medicine and biology
Growth-based approaches to correct the underlying mutation in patient-derived hematopoietic stem/progenitor cells, induce fetal hemoglobin expression to circumvent sickling of red blood cells, or create corrected induced pluripotent stem cells (iPSCs) among other approaches are summarized.
Delivering on the promise of gene editing for cystic fibrosis
- Medicine, BiologyGenes & diseases
Development of clustered regularly interspaced short palindromic repeats/CRISPR-associated technology for potential clinical applications
- BiologyWorld journal of clinical cases
This study reviews the recently reported application platforms for screening, diagnosis, and treatment of different diseases based on CRISPR/Cas systems and would be a valuable reference for future genome-editing practices.
Extinction of all infectious HIV in cell culture by the CRISPR-Cas12a system with only a single crRNA
- BiologyNucleic acids research
It is disclosed that DNA cleavage by the Cas12a endonuclease and subsequent DNA repair causes mutations with a sequence profile that is distinct from that of Cas9, the first report that documents these striking differences.
SHOWING 1-10 OF 104 REFERENCES
In vivo genome editing restores hemostasis in a mouse model of hemophilia
It is shown that ZFNs are able to induce DSBs efficiently when delivered directly to mouse liver and that, when co-delivered with an appropriately designed gene-targeting vector, they can stimulate gene replacement through both homology-directed and Homology-independent targeted gene insertion at the ZFN-specified locus.
CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes
- BiologyProtein & Cell
It is found that CRISPR/Cas9 could effectively cleave the endogenous β-globin gene (HBB), however, the efficiency of homologous recombination directed repair (HDR) of HBB was low and the edited embryos were mosaic.
Enhanced proofreading governs CRISPR-Cas9 targeting accuracy
A new hyper-accurate Cas9 variant (HypaCas9) is designed that demonstrates high genome-wide specificity without compromising on-target activity in human cells and offers a more comprehensive model to rationalize and modify the balance between target recognition and nuclease activation for precision genome editing.
Rationally engineered Cas9 nucleases with improved specificity
- Biology, EngineeringScience
Using targeted deep sequencing and unbiased whole-genome off-target analysis to assess Cas9-mediated DNA cleavage in human cells, it is demonstrated that “enhanced specificity” SpCas9 (eSpCas9) variants reduce off- target effects and maintain robust on-target cleavage, which could be broadly useful for genome-editing applications requiring a high level of specificity.
FACS-Assisted CRISPR-Cas9 Genome Editing Facilitates Parkinson's Disease Modeling
- BiologyStem cell reports
Multiplex Genome Engineering Using CRISPR/Cas Systems
Two different type II CRISPR/Cas systems are engineered and it is demonstrated that Cas9 nucleases can be directed by short RNAs to induce precise cleavage at endogenous genomic loci in human and mouse cells, demonstrating easy programmability and wide applicability of the RNA-guided nuclease technology.
Adenoviral vector DNA for accurate genome editing with engineered nucleases
- Biology, EngineeringNature Methods
It is reported that the nature of the donor DNA greatly affects the specificity and accuracy of the editing process following site-specific genomic cleavage by transcription activator–like effector nucleases (TALENs) and clustered, regularly interspaced, short palindromic repeats (CRISPR)-Cas9 nucleases.
RNA-Guided Human Genome Engineering via Cas9
The type II bacterial CRISPR system is engineer to function with custom guide RNA (gRNA) in human cells to establish an RNA-guided editing tool for facile, robust, and multiplexable human genome engineering.
Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
E engineered fusions of CRISPR/Cas9 and a cytidine deaminase enzyme that retain the ability to be programmed with a guide RNA, do not induce dsDNA breaks, and mediate the direct conversion of cytidine to uridine, thereby effecting a C→T (or G→A) substitution.
Efficient targeting of a SCID gene by an engineered single-chain homing endonuclease
- BiologyNucleic acids research
The first demonstration that an engineered meganuclease can induce targeted recombination at an endogenous locus in up to 6% of transfected human cells is provided, ranking this new generation of endonucleases among the best molecular scissors available for genome surgery strategies.