Disruptive Technology: CRISPR/Cas-Based Tools and Approaches

Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges

(2019) Valentina Ghiaccio et al.   Molecular Diagnosis & Therapy

Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases

(2019) Panayiota Papasavva et al.   Molecular Diagnosis & Therapy

Base editing with a Cpf1–cytidine deaminase fusion

(2018) Xiaosa Li et al.   NATURE BIOTECHNOLOGY

CRISPOR: intuitive guide selection for CRISPR/Cas9 genome editing experiments and screens

(2018) Jean-Paul Concordet et al.   NUCLEIC ACIDS RESEARCH

A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

(2018) Jonathan D. Finn et al.   Cell Reports

Evolved Cas9 variants with broad PAM compatibility and high DNA specificity

(2018) Johnny H. Hu et al.   NATURE

CRISPR-Mediated Base Editing Enables Efficient Disruption of Eukaryotic Genes through Induction of STOP Codons

(2017) Pierre Billon et al.   MOLECULAR CELL

CRISPR/Cas9-Mediated CCR5 Ablation in Human Hematopoietic Stem/Progenitor Cells Confers HIV-1 Resistance In Vivo

(2017) Lei Xu et al.   MOLECULAR THERAPY

Engineered Cpf1 variants with altered PAM specificities

(2017) Linyi Gao et al.   NATURE BIOTECHNOLOGY

Advancing chimeric antigen receptor T cell therapy with CRISPR/Cas9

(2017) Jiangtao Ren et al.   Protein & Cell

Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype

(2016) Christian Brendel et al.   JOURNAL OF CLINICAL INVESTIGATION

CRISPR-Mediated Base Editing without DNA Double-Strand Breaks

(2016) Brian S. Plosky   MOLECULAR CELL

Genome-editing Technologies for Gene and Cell Therapy

(2016) Morgan L Maeder et al.   MOLECULAR THERAPY

Genome-wide specificities of CRISPR-Cas Cpf1 nucleases in human cells

(2016) Benjamin P Kleinstiver et al.   NATURE BIOTECHNOLOGY

RS-1 enhances CRISPR/Cas9- and TALEN-mediated knock-in efficiency

(2016) Jun Song et al.   Nature Communications

Post-translational Regulation of Cas9 during G1 Enhances Homology-Directed Repair

(2016) Tony Gutschner et al.   Cell Reports

Elimination of HIV-1 Genomes from Human T-lymphoid Cells by CRISPR/Cas9 Gene Editing

(2016) Rafal Kaminski et al.   Scientific Reports

A Genome-wide CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) Screen Identifies NEK7 as an Essential Component of NLRP3 Inflammasome Activation

(2015) Jonathan L. Schmid-Burgk et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Engineered CRISPR-Cas9 nucleases with altered PAM specificities

(2015) Benjamin P. Kleinstiver et al.   NATURE

Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation

(2014) Luke A. Gilbert et al.   CELL

Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery

(2014) Steven Lin et al.   eLife

Targeting Hepatitis B Virus With CRISPR/Cas9

(2014) Christoph Seeger et al.   Molecular Therapy-Nucleic Acids

CRISPR interference (CRISPRi) for sequence-specific control of gene expression

(2013) Matthew H Larson et al.   Nature Protocols

DNA double-strand-break complexity levels and their possible contributions to the probability for error-prone processing and repair pathway choice

(2013) A. Schipler et al.   NUCLEIC ACIDS RESEARCH