CRISPR/Cas9 therapeutics: progress and prospects

Signal Transduction and Targeted Therapy, Journal Year: 2023, Volume and Issue: 8(1)

Published: Jan. 16, 2023

Abstract Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) gene-editing technology is the ideal tool of future for treating diseases by permanently correcting deleterious base mutations or disrupting disease-causing genes with great precision and efficiency. A variety efficient Cas9 variants derivatives have been developed to cope complex genomic changes that occur during diseases. However, strategies effectively deliver CRISPR system diseased cells in vivo are currently lacking, nonviral vectors target recognition functions may be focus research. Pathological physiological resulting from disease onset expected serve as identifying factors targeted delivery targets gene editing. Diseases both varied complex, choice appropriate methods different important. Meanwhile, there still many potential challenges identified when targeting CRISPR/Cas9 treatment. This paper reviews current developments three aspects, namely, type, vector, characteristics. Additionally, this summarizes successful examples clinical trials finally describes possible problems associated applications.

Language: Английский

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Current applications and future perspective of CRISPR/Cas9 gene editing in cancer

Molecular Cancer, Journal Year: 2022, Volume and Issue: 21(1)

Published: Feb. 21, 2022

Abstract Clustered regularly interspaced short palindromic repeats (CRISPR) system provides adaptive immunity against plasmids and phages in prokaryotes. This inspires the development of a powerful genome engineering tool, CRISPR/CRISPR-associated nuclease 9 (CRISPR/Cas9) editing system. Due to its high efficiency precision, CRISPR/Cas9 technique has been employed explore functions cancer-related genes, establish tumor-bearing animal models probe drug targets, vastly increasing our understanding cancer genomics. Here, we review current status gene technology oncological research. We first explain basic principles introduce several new CRISPR-based modes. next detail rapid progress CRISPR screening revealing tumorigenesis, metastasis, resistance mechanisms. In addition, delivery vectors finally demonstrate potential enhance effect adoptive T cell therapy (ACT) reduce adverse reactions.

Language: Английский

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Nanoparticles in Clinical Translation for Cancer Therapy

International Journal of Molecular Sciences, Journal Year: 2022, Volume and Issue: 23(3), P. 1685 - 1685

Published: Feb. 1, 2022

The advent of cancer therapeutics brought a paradigm shift from conventional therapy to precision medicine. new therapeutic modalities accomplished through the properties nanomaterials have extended their scope in beyond drug delivery. Nanoparticles can be channeled encapsulate active pharmaceutical ingredients and deliver them tumor site more efficient manner. This review enumerates various types nanoparticles that entered clinical trials for treatment. obstacles journey nanodrug clinic market are reviewed. Furthermore, latest developments using also highlighted.

Language: Английский

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Cancer nanomedicine toward clinical translation: Obstacles, opportunities, and future prospects

Med, Journal Year: 2022, Volume and Issue: 4(3), P. 147 - 167

Published: Dec. 21, 2022

Language: Английский

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Therapeutic DNAzymes: From Structure Design to Clinical Applications

Advanced Materials, Journal Year: 2023, Volume and Issue: 35(30)

Published: Feb. 5, 2023

Therapeutic DNAzymes have unceasingly intrigued the scientific community owing to their prosperous gene regulation capability. The efficacy of against many types diseases has been extensively studied for over two decades. However, high expectations are still not translated clinic because low effectiveness in vivo. Over last five years, several aspects considered optimize DNAzyme-integrated therapeutics, including structural stability, mechanism exploration, cell internalization rate, cofactor activation, and off-target effects. Hence, this review first discusses early monotherapy design DNAzymes. Subsequently, latest modes action reviewed, followed by an elaboration on stabilization strategies considering catalytic core substrate-binding arms. DNAzyme-based synergistic therapy is then examined, highlighting responsive carrier construction, effects, discovered advanced functions, concerns. Beyond this, key clinical advances elucidated showcasing progress. Finally, future trends development challenges DNAzyme-powered therapeutics coming years discussed detail.

Language: Английский

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Principles of CRISPR-Cas9 technology: Advancements in genome editing and emerging trends in drug delivery

Journal of Drug Delivery Science and Technology, Journal Year: 2024, Volume and Issue: 92, P. 105338 - 105338

Published: Jan. 6, 2024

The rapid advancement of CRISPR-Cas9 technology has instigated a profound transformation in genome editing with significant implications for fields like health, agriculture, and biotechnology. This abstract provides an overview the historical significance fundamental components CRISPR-Cas9, notably Cas9 protein guide RNA, underscoring its pivotal role genetic manipulation. It emphasizes CRISPR-Cas9's preeminence domain precise editing, driving breakthroughs personalized medicine, gene therapy, agriculture. Of paramount importance is integration nanomaterials, encompassing lipid-based polymeric nanoparticles, alongside viral vectors, serving as potent vehicles augmenting delivery efficiency precision. We explore strategies aimed at enhancing through while also addressing ethical regulatory considerations. In expert opinion section, we offer nuanced perspective on present state field, highlighting potential transformative progress research therapy. stands brink unlocking new possibilities providing innovative solutions to address pressing global challenges.

Language: Английский

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Bacterial protoplast-derived nanovesicles carrying CRISPR-Cas9 tools re-educate tumor-associated macrophages for enhanced cancer immunotherapy

Nature Communications, Journal Year: 2024, Volume and Issue: 15(1)

Published: Jan. 31, 2024

Abstract The CRISPR-Cas9 system offers substantial potential for cancer therapy by enabling precise manipulation of key genes involved in tumorigenesis and immune response. Despite its promise, the faces critical challenges, including preservation cell viability post-editing ensuring safe vivo delivery. To address these issues, this study develops an targeting tumor-associated macrophages (TAMs). We employ bacterial protoplast-derived nanovesicles (NVs) modified with pH-responsive PEG-conjugated phospholipid derivatives galactosamine-conjugated tailored TAM targeting. Utilizing plasmid-transformed E. coli protoplasts as production platforms, we successfully load NVs two components: a Cas9-sgRNA ribonucleoprotein Pik3cg , pivotal molecular switch macrophage polarization, CpG-rich DNA fragments, acting potent TLR9 ligands. This NV-based, self-assembly approach shows promise scalable clinical production. Our strategy remodels tumor microenvironment stabilizing M1-like phenotype TAMs, thus inhibiting growth female mice. technology opens avenues immunotherapy, overcoming challenges related to safe,

Language: Английский

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Therapeutic Applications of Nanomedicine: Recent Developments and Future Perspectives

Molecules, Journal Year: 2024, Volume and Issue: 29(9), P. 2073 - 2073

Published: April 30, 2024

The concept of nanomedicine has evolved significantly in recent decades, leveraging the unique phenomenon known as enhanced permeability and retention (EPR) effect. This facilitated major advancements targeted drug delivery, imaging, individualized therapy through integration nanotechnology principles into medicine. Numerous nanomedicines have been developed applied for disease treatment, with a particular focus on cancer therapy. Recently, utilized various advanced fields, including diagnosis, vaccines, immunotherapy, gene tissue engineering. Multifunctional facilitate concurrent medication therapeutic monitoring, allowing immediate responses personalized treatment plans. review concerns advancement nanomaterials their potential applications biological medical fields. Along this, we also mention clinical translations challenges that is currently facing to overcome translation barrier.

Language: Английский

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Blood–Brain Barrier-Targeting Nanoparticles: Biomaterial Properties and Biomedical Applications in Translational Neuroscience

Pharmaceuticals, Journal Year: 2024, Volume and Issue: 17(5), P. 612 - 612

Published: May 10, 2024

Overcoming the blood–brain barrier (BBB) remains a significant hurdle in effective drug delivery to brain. While BBB serves as crucial protective barrier, it poses challenges delivering therapeutic agents their intended targets within brain parenchyma. To enhance for treatment of neurological diseases, several technologies circumvent have been developed last few years. Among them, nanoparticles (NPs) are one most versatile and promising tools. Here, we summarize characteristics NPs that facilitate penetration, including size, shape, chemical composition, surface charge, importantly, conjugation with various biological or synthetic molecules such glucose, transferrin, insulin, polyethylene glycol, peptides, aptamers. Additionally, discuss coating surfactants. A comprehensive overview common vitro vivo models NP penetration studies is also provided. The discussion extends discussing impairment under pathological conditions leveraging alterations delivery. Emphasizing need future uncover inherent properties NPs, review advocates role beyond systems calls efforts translating clinic therapeutics. Overall, stand out highly strategy precise targeting disorders.

Language: Английский

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Designing bioactive micro-/nanomotors for engineered regeneration

Engineered Regeneration, Journal Year: 2021, Volume and Issue: 2, P. 109 - 115

Published: Jan. 1, 2021

Micro/nanoscale motors(MNMs) have been regarded as promising tools in the field of engineered regeneration due to unique property autonomous motion. Herein, a review on advancements MNMs area is presented, covering aspects from their propulsion mechanisms frontiers regeneration, listing revolutionary applications biosensing, medical imaging, drug delivery and tissue engineering. Finally, challenges future directions are finally discussed basis achievements.

Language: Английский

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