Ultrasound-activated particles as CRISPR/Cas9 delivery system for androgenic alopecia therapy

Ryu, Jee-Yeon; Won, Eun-Jeong; Lee, Han A Reum; Kim, Jin Hyun; Hui, Emmanuel; Kim, Hong Pyo; Yoon, Tae‐Jong

doi:10.1016/j.biomaterials.2019.119736

Cited by 81 publications

(79 citation statements)

References 33 publications

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“…The production of the protein in the bacterial hosts might be expensive and the endotoxin contamination could be considered as an additional obstacle for their large-scale production. Various non-viral carriers have been used to transfer such platforms into the target cells including gold NPs, graphene oxide, carboxylated branched poly (β-amino ester) NPs, β-cyclodextrin-conjugated low-molecular-weight PEI, microbubble-nanoliposomal particles, pH-responsive silica-metal-organic framework (SMOF) hybrid NPs consisting of both silica and zeolitic imidazole framework (ZIF) as well as cell-penetrating peptides and DNA nanoclews [319][320][321][322][323][324][325][326][327][328][329][330][331].…”

Section: Future Perspectivesmentioning

confidence: 99%

In vivo gene delivery mediated by non-viral vectors for cancer therapy

Mohammadinejad

Dehshahri

Madamsetty

et al. 2020

Journal of Controlled Release

177

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Elsevier has created a COVID-19 resource centre with free information in English and Mandarin on the novel coronavirus COVID-19. The COVID-19 resource centre is hosted on Elsevier Connect, the company's public news and information website. Elsevier hereby grants permission to make all its COVID-19-related research that is available on the COVID-19 resource centre-including this research content-immediately available in PubMed Central and other publicly funded repositories, such as the WHO COVID database with rights for unrestricted research re-use and analyses in any form or by any means with acknowledgement of the original source. These permissions are granted for free by Elsevier for as long as the COVID-19 resource centre remains active.

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Section: Future Perspectivesmentioning

confidence: 99%

In vivo gene delivery mediated by non-viral vectors for cancer therapy

Mohammadinejad

Dehshahri

Madamsetty

et al. 2020

Journal of Controlled Release

177

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“…The targeted 3-oxo-5-alpha-steroid 4-dehydrogenase 2 (SRD5A20) gene, which is responsible for the pathogenesis of male-pattern baldness, was specifically recognized and efficiently edited in vivo, thereby inhibiting the production of the SRD5A2 protein. [192] The major challenge of effective gene therapy for central nervous system diseases is the presence of the blood-brain barrier (BBB). Conventional intracranial injection produces poor gene expression and may cause nerve damage.…”

Section: Other Environmentally Responsive Nanocarrier Schemesmentioning

confidence: 99%

“…The targeted 3‐oxo‐5‐alpha‐steroid 4‐dehydrogenase 2 ( SRD5A20 ) gene, which is responsible for the pathogenesis of male‐pattern baldness, was specifically recognized and efficiently edited in vivo, thereby inhibiting the production of the SRD5A2 protein. [ 192 ]…”

Section: Stimulus‐responsive Nanocarrier Delivery Systemsmentioning

confidence: 99%

Recent Advances in Stimulus‐Responsive Nanocarriers for Gene Therapy

Cheng

et al. 2021

Advanced Science

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Gene therapy provides a promising strategy for curing monogenetic disorders and complex diseases. However, there are challenges associated with the use of viral delivery vectors. The advent of nanomedicine represents a quantum leap in the application of gene therapy. Recent advances in stimulus-responsive nonviral nanocarriers indicate that they are efficient delivery systems for loading and unloading of therapeutic nucleic acids. Some nanocarriers are responsive to cues derived from the internal environment, such as changes in pH, redox potential, enzyme activity, reactive oxygen species, adenosine triphosphate, and hypoxia. Others are responsive to external stimulations, including temperature gradients, light irradiation, ultrasonic energy, and magnetic field. Multiple stimuli-responsive strategies have also been investigated recently for experimental gene therapy.

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“…Enzyme-responsive gene delivery systems furthermore allowed temporal control of DNA release and were fabricated by incorporating the matrix metalloproteinase (MMP) susceptible peptides GPQGIWGQ [ 116 ] and the GCRD-GPQGIWGQDRCG [ 117 , 118 ] into an Au surface [ 116 ], into a breath figure porous structures [ 117 ] or in PEG hydrogels [ 118 ]. By using similar approaches, pH-sensitive [ 119 , 120 ], thermosensitive [ 120 , 121 ] or even ultrasound-activated [ 122 , 123 ], visible-light assisted [ 124 ], photothermal-assisted [ 125 ] gene delivery systems were developed.…”

Section: Main Textmentioning

confidence: 99%

Physical and mechanical cues affecting biomaterial-mediated plasmid DNA delivery: insights into non-viral delivery systems

Graceffa

2021

Journal of Genetic Engineering and Biotechnology

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Background Whilst traditional strategies to increase transfection efficiency of non-viral systems aimed at modifying the vector or the polyplexes/lipoplexes, biomaterial-mediated gene delivery has recently sparked increased interest. This review aims at discussing biomaterial properties and unravelling underlying mechanisms of action, for biomaterial-mediated gene delivery. DNA internalisation and cytoplasmic transport are initially discussed. DNA immobilisation, encapsulation and surface-mediated gene delivery (SMD), the role of extracellular matrix (ECM) and topographical cues, biomaterial stiffness and mechanical stimulation are finally outlined. Main text Endocytic pathways and mechanisms to escape the lysosomal network are highly variable. They depend on cell and DNA complex types but can be diverted using appropriate biomaterials. 3D scaffolds are generally fabricated via DNA immobilisation or encapsulation. Degradation rate and interaction with the vector affect temporal patterns of DNA release and transgene expression. In SMD, DNA is instead coated on 2D surfaces. SMD allows the incorporation of topographical cues, which, by inducing cytoskeletal re-arrangements, modulate DNA endocytosis. Incorporation of ECM mimetics allows cell type-specific transfection, whereas in spite of discordances in terms of optimal loading regimens, it is recognised that mechanical loading facilitates gene transfection. Finally, stiffer 2D substrates enhance DNA internalisation, whereas in 3D scaffolds, the role of stiffness is still dubious. Conclusion Although it is recognised that biomaterials allow the creation of tailored non-viral gene delivery systems, there still are many outstanding questions. A better characterisation of endocytic pathways would allow the diversion of cell adhesion processes and cytoskeletal dynamics, in order to increase cellular transfection. Further research on optimal biomaterial mechanical properties, cell ligand density and loading regimens is limited by the fact that such parameters influence a plethora of other different processes (e.g. cellular adhesion, spreading, migration, infiltration, and proliferation, DNA diffusion and release) which may in turn modulate gene delivery. Only a better understanding of these processes may allow the creation of novel robust engineered systems, potentially opening up a whole new area of biomaterial-guided gene delivery for non-viral systems.

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Ultrasound-activated particles as CRISPR/Cas9 delivery system for androgenic alopecia therapy

Cited by 81 publications

References 33 publications

In vivo gene delivery mediated by non-viral vectors for cancer therapy

In vivo gene delivery mediated by non-viral vectors for cancer therapy

Recent Advances in Stimulus‐Responsive Nanocarriers for Gene Therapy

Physical and mechanical cues affecting biomaterial-mediated plasmid DNA delivery: insights into non-viral delivery systems

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