Stimuli-responsive nanoformulations for CRISPR-Cas9 genome editing

Fang, Tianxu; Cao, Xiaona; Ibnat, Mysha; Chen, Guojun

doi:10.1186/s12951-022-01570-y

Cited by 27 publications

(18 citation statements)

References 243 publications

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“…[86] Development of nanoformulations which are responsive to internal stimuli like pH, enzymes, adenosine triphosphate (ATP), glucose, oxygen may also enhance the target specific delivery of CRISPR/Cas9 tool. [87] Tan et al developed reactive oxygen species (ROS) responsive polypeptides to deliver CRISPR/Cas9-based gene therapy into the cells. They used boronate which is sensitive to ROS to decorate the polypeptides which formed uniform nanoparticles which were effective in delivering Cas9 ribonucleoprotein into the cells.…”

Section: Methods Of Preparation Of Lnpsmentioning

confidence: 99%

Section: Delivery Strategy Using Nonviral Nanocarriersmentioning

confidence: 99%

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CRISPR/Cas9 Genome Editing for Tissue‐Specific In Vivo Targeting: Nanomaterials and Translational Perspective

et al. 2023

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Clustered randomly interspaced short palindromic repeats (CRISPRs) and its associated endonuclease protein, i.e., Cas9, have been discovered as an immune system in bacteria and archaea; nevertheless, they are now being adopted as mainstream biotechnological/molecular scissors that can modulate ample genetic and nongenetic diseases via insertion/deletion, epigenome editing, messenger RNA editing, CRISPR interference, etc. Many Food and Drug Administration‐approved and ongoing clinical trials on CRISPR adopt ex vivo strategies, wherein the gene editing is performed ex vivo, followed by reimplantation to the patients. However, the in vivo delivery of the CRISPR components is still under preclinical surveillance. This review has summarized the nonviral nanodelivery strategies for gene editing using CRISPR/Cas9 and its recent advancements, strategic points of view, challenges, and future aspects for tissue‐specific in vivo delivery of CRISPR/Cas9 components using nanomaterials.

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Section: Methods Of Preparation Of Lnpsmentioning

confidence: 99%

Section: Delivery Strategy Using Nonviral Nanocarriersmentioning

confidence: 99%

CRISPR/Cas9 Genome Editing for Tissue‐Specific In Vivo Targeting: Nanomaterials and Translational Perspective

et al. 2023

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“…The MF is another commonly used exogenous stimulus for spatiotemporal control and noninvasive manipulation of drug/gene/protein delivery. External MF gradients enable delivery systems to be steered toward specific locations, allowing remote-controlled targeted drug delivery owing to their outstanding tissue penetrating properties [ 182 ]. MF-guiding of CRISPR-Cas9 in the field of genome editing can reduce the risk of undesirable off-target consequences, making precise genome editing achievable [ 183 ].…”

Section: Stimuli-responsive Gdncsmentioning

confidence: 99%

Stimuli-Responsive Gene Delivery Nanocarriers for Cancer Therapy

Zhang

Kuang

et al. 2023

Nano-Micro Lett.

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Gene therapy provides a promising approach in treating cancers with high efficacy and selectivity and few adverse effects. Currently, the development of functional vectors with safety and effectiveness is the intense focus for improving the delivery of nucleic acid drugs for gene therapy. For this purpose, stimuli-responsive nanocarriers displayed strong potential in improving the overall efficiencies of gene therapy and reducing adverse effects via effective protection, prolonged blood circulation, specific tumor accumulation, and controlled release profile of nucleic acid drugs. Besides, synergistic therapy could be achieved when combined with other therapeutic regimens. This review summarizes recent advances in various stimuli-responsive nanocarriers for gene delivery. Particularly, the nanocarriers responding to endogenous stimuli including pH, reactive oxygen species, glutathione, and enzyme, etc., and exogenous stimuli including light, thermo, ultrasound, magnetic field, etc., are introduced. Finally, the future challenges and prospects of stimuli-responsive gene delivery nanocarriers toward potential clinical translation are well discussed. The major objective of this review is to present the biomedical potential of stimuli-responsive gene delivery nanocarriers for cancer therapy and provide guidance for developing novel nanoplatforms that are clinically applicable.

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“…Some small-molecule inhibitors or activators can regulate CRISPR-based genome editing by time-based mechanisms to control the onset, duration, and intensity of CRISPR gene editing, with weaker control over the spatial dimension. However, bioresponsive delivery carriers and cell-specific promoters can achieve enhanced control of CRISPR genome editing in the spatial dimension [ 178 ].…”

Section: Difference Between Genetic Chemical and Physical Control For...mentioning

confidence: 99%

Recent Advances in Genome-Editing Technology with CRISPR/Cas9 Variants and Stimuli-Responsive Targeting Approaches within Tumor Cells: A Future Perspective of Cancer Management

Allemailem

Almatroodi

Almatroudi

et al. 2023

IJMS

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The innovative advances in transforming clustered regularly interspaced short palindromic repeats-associated protein 9 (CRISPR/Cas9) into different variants have taken the art of genome-editing specificity to new heights. Allosteric modulation of Cas9-targeting specificity by sgRNA sequence alterations and protospacer adjacent motif (PAM) modifications have been a good lesson to learn about specificity and activity scores in different Cas9 variants. Some of the high-fidelity Cas9 variants have been ranked as Sniper-Cas9, eSpCas9 (1.1), SpCas9-HF1, HypaCas9, xCas9, and evoCas9. However, the selection of an ideal Cas9 variant for a given target sequence remains a challenging task. A safe and efficient delivery system for the CRISPR/Cas9 complex at tumor target sites faces considerable challenges, and nanotechnology-based stimuli-responsive delivery approaches have significantly contributed to cancer management. Recent innovations in nanoformulation design, such as pH, glutathione (GSH), photo, thermal, and magnetic responsive systems, have modernized the art of CRISPR/Cas9 delivery approaches. These nanoformulations possess enhanced cellular internalization, endosomal membrane disruption/bypass, and controlled release. In this review, we aim to elaborate on different CRISPR/Cas9 variants and advances in stimuli-responsive nanoformulations for the specific delivery of this endonuclease system. Furthermore, the critical constraints of this endonuclease system on clinical translations towards the management of cancer and prospects are described.

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Stimuli-responsive nanoformulations for CRISPR-Cas9 genome editing

Cited by 27 publications

References 243 publications

CRISPR/Cas9 Genome Editing for Tissue‐Specific In Vivo Targeting: Nanomaterials and Translational Perspective

CRISPR/Cas9 Genome Editing for Tissue‐Specific In Vivo Targeting: Nanomaterials and Translational Perspective

Stimuli-Responsive Gene Delivery Nanocarriers for Cancer Therapy

Recent Advances in Genome-Editing Technology with CRISPR/Cas9 Variants and Stimuli-Responsive Targeting Approaches within Tumor Cells: A Future Perspective of Cancer Management

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