The use of new CRISPR tools in cardiovascular research and medicine

Nishiga, Masataka; Liu, Chun; Qi, Lei S.; Wu, Joseph C.

doi:10.1038/s41569-021-00669-3

Cited by 31 publications

(24 citation statements)

References 159 publications

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“…With the advance in nanotechnology and chemistry, rationalized system designs with translational and favorable manufacturing practices need to be fully considered. Third, stimuli-responsive non-viral systems can be further extended to other more recently engineered genome editing machineries, such as prime editors and base editors [ 7 , 76 , 237 , 238 ] . Lastly, current investigations of stimuli-responsive CRISPR-Cas9 delivery systems are still limited to preclinical evaluations therefore, efforts to advance the technologies into clinical settings should be actively explored.…”

Section: Discussionmentioning

confidence: 99%

Stimuli-responsive nanoformulations for CRISPR-Cas9 genome editing

et al. 2022

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The CRISPR-Cas9 technology has changed the landscape of genome editing and has demonstrated extraordinary potential for treating otherwise incurable diseases. Engineering strategies to enable efficient intracellular delivery of CRISPR-Cas9 components has been a central theme for broadening the impact of the CRISPR-Cas9 technology. Various non-viral delivery systems for CRISPR-Cas9 have been investigated given their favorable safety profiles over viral systems. Many recent efforts have been focused on the development of stimuli-responsive non-viral CRISPR-Cas9 delivery systems, with the goal of achieving efficient and precise genome editing. Stimuli-responsive nanoplatforms are capable of sensing and responding to particular triggers, such as innate biological cues and external stimuli, for controlled CRISPR-Cas9 genome editing. In this Review, we overview the recent advances in stimuli-responsive nanoformulations for CRISPR-Cas9 delivery, highlight the rationale of stimuli and formulation designs, and summarize their biomedical applications.

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Section: Discussionmentioning

confidence: 99%

Stimuli-responsive nanoformulations for CRISPR-Cas9 genome editing

et al. 2022

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“…229 However, despite these advances, nanoparticle delivery in the heart has had only limited success (if any), partially because cardiomyocytes lack unique cell surface markers for nanoparticle uptake. 230 Major efforts are needed to modify nanoparticles toward efficient tissue-specific delivery.…”

Section: Delivery Strategies For Therapeutic Gene Editing In the Heartmentioning

confidence: 99%

CRISPR Modeling and Correction of Cardiovascular Disease

Liu

Olson

2022

Circulation Research

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Cardiovascular disease remains the leading cause of morbidity and mortality in the developed world. In recent decades, extraordinary effort has been devoted to defining the molecular and pathophysiological characteristics of the diseased heart and vasculature. Mouse models have been especially powerful in illuminating the complex signaling pathways, genetic and epigenetic regulatory circuits, and multicellular interactions that underlie cardiovascular disease. The advent of CRISPR genome editing has ushered in a new era of cardiovascular research and possibilities for genetic correction of disease. Next-generation sequencing technologies have greatly accelerated the identification of disease-causing mutations, and advances in gene editing have enabled the rapid modeling of these mutations in mice and patient-derived induced pluripotent stem cells. The ability to correct the genetic drivers of cardiovascular disease through delivery of gene editing components in vivo, while still facing challenges, represents an exciting therapeutic frontier. In this review, we provide an overview of cardiovascular disease mechanisms and the potential applications of CRISPR genome editing for disease modeling and correction. We also discuss the extent to which mice can faithfully model cardiovascular disease and the opportunities and challenges that lie ahead.

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“…However, for dCas9-FokI and SpCas9 nickase, the target sites are remarkably limited in 1 For SpCas9 nickase and FokI-dCas9, the Cas9 variants are used in pairs to increase gene-editing specificity. 2 The amino acid substitutions in LZ3 Cas9 are not explicitly described in the original paper by Schmid-Burgk et al We collected the mutations from the deposited plasmid (https://www.addgene.org/140561/) (accessed on 8 June 2022). 3 miCas9 improves gene-editing specificity not through reducing non-specific recognition between gRNA and target DNA site but rather through enhanced homology-directed repair.…”

Section: Dcas9-fokimentioning

confidence: 99%

“…CRISPR–Cas9 is a powerful gene-editing tool and has a wide range of applications in biotechnology and medicine [ 1 , 2 , 3 , 4 ]. It is increasingly popular thanks to its simplicity, scalability, and affordability.…”

Section: Introductionmentioning

confidence: 99%

Recent Advances in Improving Gene-Editing Specificity through CRISPR–Cas9 Nuclease Engineering

Huang

Yang

Zhang

et al. 2022

Cells

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CRISPR–Cas9 is the state-of-the-art programmable genome-editing tool widely used in many areas. For safe therapeutic applications in clinical medicine, its off-target effect must be dramatically minimized. In recent years, extensive studies have been conducted to improve the gene-editing specificity of the most popular CRISPR–Cas9 nucleases using different strategies. In this review, we summarize and discuss these strategies and achievements, with a major focus on improving the gene-editing specificity through Cas9 protein engineering.

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The use of new CRISPR tools in cardiovascular research and medicine

Cited by 31 publications

References 159 publications

Stimuli-responsive nanoformulations for CRISPR-Cas9 genome editing

Stimuli-responsive nanoformulations for CRISPR-Cas9 genome editing

CRISPR Modeling and Correction of Cardiovascular Disease

Recent Advances in Improving Gene-Editing Specificity through CRISPR–Cas9 Nuclease Engineering

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