CRISPR Genome Editing & Therapy

CRISPR-Cas system enables the editing of genes to create or correct mutations. This feed focuses on the application of this system for gene editing and therapy in human diseases.

August 11, 2020
Open Access

AAV-Genome Population Sequencing of Vectors Packaging CRISPR Components Reveals Design-Influenced Heterogeneity

Molecular Therapy. Methods & Clinical Development
Ngoc Tam TranPhillip W L Tai
August 21, 2020
Open Access

Safe CRISPR-Cas9 Inhibition of HIV-1 with High Specificity and Broad-Spectrum Activity by Targeting LTR NF-κB Binding Sites

Molecular Therapy. Nucleic Acids
Cheng-Han ChungBrian Wigdahl
September 10, 2020
Open Access

Gene-Editing Technologies Paired With Viral Vectors for Translational Research Into Neurodegenerative Diseases

Frontiers in Molecular Neuroscience
Joseph Edward RittinerBoris Kantor
August 19, 2020
Open Access

Uncoupling gene expression noise along the central dogma using genome engineered human cell lines

Nucleic Acids Research
Tyler QuartonLeonidas Bleris
August 26, 2020
Review
Open Access

Personalized Immuno-oncology

Medical Principles and Practice : International Journal of the Kuwait University, Health Science Centre
Kewal K Jain
September 7, 2020
Review

Adenoviral vectors for in vivo delivery of CRISPR-Cas gene editors

Journal of Controlled Release : Official Journal of the Controlled Release Society
Paul BoucherDavid T Curiel
August 25, 2020
Open Access

Futuristic CRISPR-based biosensing in the cloud and internet of things era: an overview

Multimedia Tools and Applications
Abdullahi Umar IbrahimMehmet Ozsoz
August 25, 2020

GERG Study 2019: Reproducibility of indel formation rates by comparing guideRNA format and delivery method

Journal of Biomolecular Techniques : JBT
Elizabeth SergisonKevin Knudtson
August 25, 2020
Open Access

Promoter Orientation within an AAV-CRISPR Vector Affects Cas9 Expression and Gene Editing Efficiency

The CRISPR Journal
Lewis E FryRobert E MacLaren
August 22, 2020
Open Access

PINCER: improved CRISPR/Cas9 screening by efficient cleavage at conserved residues

Nucleic Acids Research
Brendan VeenemanWenyan Zhong
August 11, 2020
Review
Open Access

Ready for Repair? Gene Editing Enters the Clinic for the Treatment of Human Disease

Molecular Therapy. Methods & Clinical Development
Martijn P T ErnstW W M Pim Pijnappel
September 17, 2020

Huntingtin protein maintains balanced energetics in mouse cardiomyocytes

Nucleosides, Nucleotides & Nucleic Acids
Marta TomczykRyszard T Smolenski
August 26, 2020
Open Access

Antisense Oligonucleotide- and CRISPR-Cas9-Mediated Rescue of mRNA Splicing for a Deep Intronic CLRN1 Mutation

Molecular Therapy. Nucleic Acids
Anna-Lena PanagiotopoulosElvir Becirovic
August 19, 2020
Open Access

Identification of Functional HLA-A*01 :01-Restricted EBV-LMP2-Specific T-cell Receptors

The Journal of Infectious Diseases
Wesley HuismanInge Jedema
August 11, 2020
Open Access

CRISPR-Cas9-Mediated In Vivo Gene Integration at the Albumin Locus Recovers Hemostasis in Neonatal and Adult Hemophilia B Mice

Molecular Therapy. Methods & Clinical Development
Qingnan WangYang Yang
September 17, 2020
Open Access

Trinucleotide Repeat-Targeting dCas9 as a Therapeutic Strategy for Fuchs' Endothelial Corneal Dystrophy

Translational Vision Science & Technology
Ziye RongV Vinod Mootha

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