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Article
Open AccessCRISPR base editing-mediated correction of a tau mutation rescues cognitive decline in a mouse model of tauopathy
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Article
Open AccessTargeted dual base editing with Campylobacter jejuni Cas9 by single AAV-mediated delivery
Various CRISPR‒Cas9 orthologs are used in genome engineering. One of the smallest Cas9 orthologs is cjCas9 derived from Campylobacter jejuni, which is a highly specific genome editing tool. Here, we developed cjC...
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Article
Open AccessEfficient CRISPR editing with a hypercompact Cas12f1 and engineered guide RNAs delivered by adeno-associated virus
Gene therapy would benefit from a miniature CRISPR system that fits into the small adeno-associated virus (AAV) genome and has high cleavage activity and specificity in eukaryotic cells. One of the most compac...
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Chapter
Muscular Dystrophy Therapy Using Viral Vector-based CRISPR/Cas
Duchenne muscular dystrophy (DMD) is a fatal X-linked genetic disorder caused by mutations in the DMD gene, which encodes the dystrophin protein. A lack of dystrophin disrupts the skeletal musculature, resulting ...
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Article
Correction to: Recent advances in CRISPR technologies for genome editing
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Article
Recent advances in CRISPR technologies for genome editing
The discovery of clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein (Cas) system, and its development into a set of powerful tools for manipulating the genome, has rev...
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Article
Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy
Adenine base editing is used to treat a mouse model of Duchenne muscular dystrophy and to create defined mutations in mouse embryos.
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Article
Open AccessCRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration
LbCpf1, derived from Lachnospiraceae bacterium ND2006, is a CRISPR RNA-guided endonuclease and holds promise for therapeutic applications. Here we show that LbCpf1 can be used for therapeutic gene editing in a mo...
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Article
Open AccessLong-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is an incurable X-linked muscle-wasting disease caused by mutations in the dystrophin gene. Gene therapy using highly functional microdystrophin genes and recombinant adeno-as...
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Article
Open AccessIn vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni
Several CRISPR-Cas9 orthologues have been used for genome editing. Here, we present the smallest Cas9 orthologue characterized to date, derived from Campylobacter jejuni (CjCas9), for efficient genome editing in ...
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Article
Open AccessAbnormal splicing switch of DMD’s penultimate exon compromises muscle fibre maintenance in myotonic dystrophy
Myotonic Dystrophy type 1 (DM1) is a dominant neuromuscular disease caused by nuclear-retained RNAs containing expanded CUG repeats. These toxic RNAs alter the activities of RNA splicing factors resulting in a...
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Protocol
Gene Therapy in Skeletal Muscle Mediated by Adeno-Associated Virus Vectors
Adeno-associated virus (AAV) is the most promising gene delivery vehicle for muscle-directed gene therapy. AAV’s natural tropism to muscle cells, long-term persistent transgene expression, multiple serotypes, ...
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Article
ADP-overexpressing adenovirus elicits enhanced cytopathic effect by induction of apoptosis
Replication-competent adenoviruses (Ad's) are emerging as a promising new modality for treatment of cancer. Selective replication of viral agents in tumor may lead to improved efficacy over nonreplicating Ad's...
