Mechanism and applications of CRISPR/Cas-9-mediated genome editing

M Asmamaw, B Zawdie - Biologics: targets and therapy, 2021 - Taylor & Francis
Clustered regularly interspaced short palindromic repeat (CRISPR) and their associated
protein (Cas-9) is the most effective, efficient, and accurate method of genome editing tool in …

Novel CRISPR–Cas systems: an updated review of the current achievements, applications, and future research perspectives

S Nidhi, U Anand, P Oleksak, P Tripathi, JA Lal… - International journal of …, 2021 - mdpi.com
According to Darwin's theory, endless evolution leads to a revolution. One such example is
the Clustered Regularly Interspaced Palindromic Repeats (CRISPR)–Cas system, an …

Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice

E Kenjo, H Hozumi, Y Makita, KA Iwabuchi… - Nature …, 2021 - nature.com
Genome editing therapy for Duchenne muscular dystrophy (DMD) holds great promise,
however, one major obstacle is delivery of the CRISPR-Cas9/sgRNA system to skeletal …

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

L Amoasii, JCW Hildyard, H Li, E Sanchez-Ortiz… - Science, 2018 - science.org
Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and
function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD …

[HTML][HTML] Targeted disruption of HLA genes via CRISPR-Cas9 generates iPSCs with enhanced immune compatibility

H Xu, BO Wang, M Ono, A Kagita, K Fujii, N Sasakawa… - Cell stem cell, 2019 - cell.com
Induced pluripotent stem cells (iPSCs) have strong potential in regenerative medicine
applications; however, immune rejection caused by HLA mismatching is a concern. B2M …

Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping

P Gee, MSY Lung, Y Okuzaki, N Sasakawa… - Nature …, 2020 - nature.com
Prolonged expression of the CRISPR-Cas9 nuclease and gRNA from viral vectors may
cause off-target mutagenesis and immunogenicity. Thus, a transient delivery system is …

CRISPR-based technologies for the manipulation of eukaryotic genomes

AC Komor, AH Badran, DR Liu - Cell, 2017 - cell.com
The CRISPR-Cas9 RNA-guided DNA endonuclease has contributed to an explosion of
advances in the life sciences that have grown from the ability to edit genomes within living …

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

CE Nelson, CH Hakim, DG Ousterout, PI Thakore… - Science, 2016 - science.org
Duchenne muscular dystrophy (DMD) is a devastating disease affecting about 1 out of 5000
male births and caused by mutations in the dystrophin gene. Genome editing has the …

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

C Long, L Amoasii, AA Mireault, JR McAnally, H Li… - Science, 2016 - science.org
CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic
diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in …

CRISPR/Cas9 in genome editing and beyond

H Wang, M La Russa, LS Qi - Annual review of biochemistry, 2016 - annualreviews.org
The Cas9 protein (CRISPR-associated protein 9), derived from type II CRISPR (clustered
regularly interspaced short palindromic repeats) bacterial immune systems, is emerging as a …