Latest developed strategies to minimize the off-target effects in CRISPR-Cas-mediated genome editing
Gene editing that makes target gene modification in the genome by deletion or addition has
revolutionized the era of biomedicine. Clustered regularly interspaced short palindromic …
revolutionized the era of biomedicine. Clustered regularly interspaced short palindromic …
Therapeutic genome editing: prospects and challenges
Recent advances in the development of genome editing technologies based on
programmable nucleases have substantially improved our ability to make precise changes …
programmable nucleases have substantially improved our ability to make precise changes …
A high-throughput platform to identify small-molecule inhibitors of CRISPR-Cas9
The precise control of CRISPR-Cas9 activity is required for a number of genome
engineering technologies. Here, we report a generalizable platform that provided the first …
engineering technologies. Here, we report a generalizable platform that provided the first …
A self-deleting AAV-CRISPR system for in vivo genome editing
Adeno-associated viral (AAV) vectors packaging the CRISPR-Cas9 system (AAV-CRISPR)
can efficiently modify disease-relevant genes in somatic tissues with high efficiency. AAV …
can efficiently modify disease-relevant genes in somatic tissues with high efficiency. AAV …
Overcoming gene-delivery hurdles: physiological considerations for nonviral vectors
With the use of contemporary tools and techniques, it has become possible to more
precisely tune the biochemical mechanisms associated with using nonviral vectors for gene …
precisely tune the biochemical mechanisms associated with using nonviral vectors for gene …
Immunity to CRISPR Cas9 and Cas12a therapeutics
WL Chew - Wiley Interdisciplinary Reviews: Systems Biology …, 2018 - Wiley Online Library
Genome‐editing therapeutics are poised to treat human diseases. As we enter clinical trials
with the most promising CRISPR‐Cas9 and CRISPR‐Cas12a (Cpf1) modalities, the risks …
with the most promising CRISPR‐Cas9 and CRISPR‐Cas12a (Cpf1) modalities, the risks …
Genome engineering using adeno-associated virus: basic and clinical research applications
In addition to their broad potential for therapeutic gene delivery, adeno-associated virus
(AAV) vectors possess the innate ability to stimulate homologous recombination in …
(AAV) vectors possess the innate ability to stimulate homologous recombination in …
Guide RNA engineering for versatile Cas9 functionality
CM Nowak, S Lawson, M Zerez… - Nucleic Acids Research, 2016 - academic.oup.com
Abstract The Clustered Regularly Interspaced Short Palindromic Repeats system allows a
single guide RNA (sgRNA) to direct a protein with combined helicase and nuclease activity …
single guide RNA (sgRNA) to direct a protein with combined helicase and nuclease activity …
Hit and go CAS9 delivered through a lentiviral based self-limiting circuit
G Petris, A Casini, C Montagna, F Lorenzin… - Nature …, 2017 - nature.com
In vivo application of the CRISPR-Cas9 technology is still limited by unwanted Cas9
genomic cleavages. Long-term expression of Cas9 increases the number of genomic loci …
genomic cleavages. Long-term expression of Cas9 increases the number of genomic loci …
RISC-y business: Limitations of short hairpin RNA-mediated gene silencing in the brain and a discussion of CRISPR/Cas-based alternatives
Manipulating gene expression within and outside the nervous system is useful for
interrogating gene function and developing therapeutic interventions for a variety of …
interrogating gene function and developing therapeutic interventions for a variety of …