2020
DOI: 10.1002/advs.201903432
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Dual Supramolecular Nanoparticle Vectors Enable CRISPR/Cas9‐Mediated Knockin of Retinoschisin 1 Gene—A Potential Nonviral Therapeutic Solution for X‐Linked Juvenile Retinoschisis

Abstract: The homology-independent targeted integration (HITI) strategy enables effective CRISPR/Cas9-mediated knockin of therapeutic genes in nondividing cells in vivo, promising general therapeutic solutions for treating genetic diseases like Xlinked juvenile retinoschisis. Herein, supramolecular nanoparticle (SMNP) vectors are used for codelivery of two DNA plasmids-CRISPR-Cas9 genome-editing system and a therapeutic gene, Retinoschisin 1 (RS1)-enabling clustered regularly interspaced short palindromic repeats (CRISP… Show more

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Cited by 40 publications
(42 citation statements)
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“…In another study, PAMAM was grafted with adamantane (Ad-PAMAM) to form a dendrimer building block ( Figure 3 C) [ 38 ]. Beta-cyclodextrin (β-CD) is a cyclic oligosaccharide composing of seven glucopyranose units.…”
Section: Nonviral Delivery Systems For Genome Editingmentioning
confidence: 99%
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“…In another study, PAMAM was grafted with adamantane (Ad-PAMAM) to form a dendrimer building block ( Figure 3 C) [ 38 ]. Beta-cyclodextrin (β-CD) is a cyclic oligosaccharide composing of seven glucopyranose units.…”
Section: Nonviral Delivery Systems For Genome Editingmentioning
confidence: 99%
“…Successful tumor suppression and gene knockout have been observed following intratumoral administration in various studies [ 127 , 135 ]. Along these lines, intravitreal injection of Cas9/PAMAM polymeric nanoparticles has been used to knock in therapeutic genes in the retina [ 38 ], and local infusion of Cas13a/crRNA/lipid-coated polymeric nanoparticle into the bladder cavity has been used to reduce systemic off-target effects. These strategies promise safe gene-editing delivery systems for specific types of disease, including skin cancer, head and neck cancer, and retinal degeneration diseases.…”
Section: Challenges and Future Perspectivesmentioning
confidence: 99%
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“…CRISPR element can be delivered to the eye as DNA (Cas9-sgRNA plasmid), mRNA (Cas9 mRNA, and sgRNA as two separate entities) or protein (Cas9 protein with sgRNA as ribonucleoprotein complex; RNP). Other than the viral-based in vivo delivery, nanoparticle-mediated delivery has been in use and considered to be safe [266][267][268][269]. The CRISPR can introduce as well as correct point mutations, insertions, and deletions in the cultured cells as well as in livings (mice, rats, monkeys, pigs, etc.)…”
Section: Crispr-gene Editingmentioning
confidence: 99%
“…More recently, others have shown the potential of HITI for use with other Cas proteins; an AAV based HITI strategy making use of SaCas9 was shown to restore FIX serum levels to a greater extent than the equivalent HDR strategy in a mouse model of haemophilia (71). Furthermore, HITI can be used to aid in gene augmentation therapies -targeting genes to safe harbour loci for sustained expression without the risk of insertional mutagenesis (72). The ability of FIVER to report HITI editing will be beneficial in developing new and improved HITI-based therapeutics.…”
mentioning
confidence: 99%