In Situ Gene Engineering Approach to Overcome Tumor Resistance and Enhance T Cell-Mediated Cancer Immunotherapy DOI
Di Zhang, Wenjuan Wang, Mingtan Tang

и другие.

Nano Letters, Год журнала: 2025, Номер unknown

Опубликована: Апрель 3, 2025

T cell-mediated cancer immunotherapy harnesses the power of cytotoxic lymphocytes (CTLs) to target and eradicate tumor cells. However, cells often evade immune attack through membrane repair mechanisms involving endosomal sorting complexes required for transport (ESCRT) suppression within microenvironment. Here, we developed a robust TMV@PpCHIL nanomedicine address these issues by reprogramming via in situ gene editing. Using CRISPR/Cas9, disrupted Chmp4b gene, key component ESCRT machinery, preventing from repairing CTL-induced damage. Simultaneously, genetically engineered produce interleukin-12 (IL-12), cytokine that enhances CTL activation. The nanomedicine, designed coating vesicles (TMVs) onto polyamidoamine (PAMAM) dendrimer-condensed plasmid complexes, ensured efficient CRISPR/Cas9-based editing sustained IL-12 production. This approach significantly enhanced CTL-mediated cell cytotoxicity, suppressed growth, reduced metastasis, prolonged survival, providing promising strategy durable treatment.

Язык: Английский

Biologics, theranostics, and personalized medicine in drug delivery systems DOI Creative Commons
Matteo Puccetti, Marilena Pariano, Aurélie Schoubben

и другие.

Pharmacological Research, Год журнала: 2024, Номер 201, С. 107086 - 107086

Опубликована: Янв. 29, 2024

The progress in human disease treatment can be greatly advanced through the implementation of nanomedicine. This approach involves targeted and cell-specific therapy, controlled drug release, personalized dosage forms, wearable delivery, companion diagnostics. By integrating cutting-edge technologies with delivery systems, greater precision achieved at tissue cellular levels use stimuli-responsive nanoparticles, development electrochemical sensor systems. targeting – by virtue nanotechnology allows for therapy to directed specifically affected tissues while reducing side effects on healthy tissues. As such, nanomedicine has potential transform conditions such as cancer, genetic diseases, chronic illnesses facilitating precise delivery. Additionally, forms devices offer ability tailor unique needs each patient, thereby increasing therapeutic effectiveness compliance. Companion diagnostics further enable efficient monitoring response, enabling customized adjustments plan. question whether all approaches outlined here are viable alternatives current treatments is also discussed. In general, application field biomedicine may provide a strong alternative existing several reasons. this review, we aim present evidence that, although early stages, fully merging technology innovative shows promise successful across various areas, including cancer or diseases.

Язык: Английский

Процитировано

79
CRISPR-Cas9 Gene Therapy: Non-Viral Delivery and Stimuli-Responsive Nanoformulations DOI Creative Commons
Hyun-Woo Lee, Won‐Yeop Rho,

Yoon-Hee Kim

и другие.

Molecules, Год журнала: 2025, Номер 30(3), С. 542 - 542

Опубликована: Янв. 24, 2025

The CRISPR-Cas9 technology, one of the groundbreaking genome editing methods for addressing genetic disorders, has emerged as a powerful, precise, and efficient tool. However, its clinical translation remains hindered by challenges in delivery efficiency targeting specificity. This review provides comprehensive analysis structural features, advantages, potential applications various non-viral stimuli-responsive systems, examining recent progress to emphasize address these limitations advance therapeutics. We describe how reports that nonviral vectors, including lipid-based nanoparticles, extracellular vesicles, polymeric gold mesoporous silica can offer diverse advantages enhance stability, cellular uptake, biocompatibility, based on their structures physio-chemical stability. also summarize nanoformulations, type vector, introduce precision control delivery. Stimuli-responsive nanoformulations are designed respond pH, redox states, external triggers, facilitate controlled targeted delivery, minimize off-target effects. insights our suggest future gene therapy technologies highlight systems CRISPR-Cas9’s efficacy, positioning them pivotal tools gene-editing therapies.

Язык: Английский

Процитировано

6
Innovations in RNA therapeutics: a review of recent advances and emerging technologies DOI
Tuward J. Dweh,

Glay Jr Eric Wulu,

John Kessellie Jallah

и другие.

Nucleosides Nucleotides & Nucleic Acids, Год журнала: 2025, Номер unknown, С. 1 - 25

Опубликована: Янв. 13, 2025

The field of biomedical science has witnessed another milestone with the advent RNA-based therapeutics. This review explores three major RNA molecules, namely: messenger (mRNA), interference technology (RNAi), and Antisense Oligonucleotide (ASO), analyses U.S. Food Drug Administration drugs from 14 pharmaceutical companies in terms targeted genes, diseases types, clinical trials status, mode delivery, year production. Many such are clinically approved or pending approval by (FDA) alongside other leading agencies. Regarding diseases, this article emphasizes cancer therapy, genetic viral infections, two categories drug delivery systems include vectors nanoparticles. Despite tremendous progress made, key issues associated these stability, off-target activities payloads, efficiency cellular uptake, innovative need for engineering techniques modifications. transformative potential therapeutics role technologies addressing needs, paving way a new era precision medicine.

Язык: Английский

Процитировано

3
Robust genome editing activity and the applications of enhanced miniature CRISPR-Cas12f1 DOI Creative Commons
Soo-Ji Park,

Sungjin Ju,

Won Jun Jung

и другие.

Nature Communications, Год журнала: 2025, Номер 16(1)

Опубликована: Янв. 15, 2025

Abstract With recent advancements in gene editing technology using the CRISPR/Cas system, there is a demand for more effective editors. A key factor facilitating efficient CRISPR delivery into cells, which known to be associated with size of system. Accordingly, compact CRISPR-Cas systems derived from various strains are discovered, among Un1Cas12f1 2.6 times smaller than SpCas9, providing advantages therapy research. Despite extensive engineering efforts improve Un1Cas12f1, efficiency still shown low depending on target site. To overcome this limitation, we develop enhanced Cas12f1 (eCas12f1), exhibits activity similar SpCas9 and AsCpf1, even targets where previously improved variants showed efficiency. Furthermore, demonstrate that eCas12f1 efficiently induces apoptosis cancer cells compatible base regulation expression, verifying its high utility applicability

Язык: Английский

Процитировано

3
The role of nanoparticles in transforming plant genetic engineering: advancements, challenges and future prospects DOI

Neelam Rani,

Kusum Kumari, Vinita Hooda

и другие.

Functional & Integrative Genomics, Год журнала: 2025, Номер 25(1)

Опубликована: Янв. 22, 2025

Язык: Английский

Процитировано

3
Application and perspective of CRISPR/Cas9 genome editing technology in human diseases modeling and gene therapy DOI Creative Commons
Manling Zhang, Hongbin Li, Yong Jin

и другие.

Frontiers in Genetics, Год журнала: 2024, Номер 15

Опубликована: Апрель 11, 2024

The Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) mediated Cas9 nuclease system has been extensively used for genome editing and gene modification in eukaryotic cells. CRISPR/Cas9 technology holds great potential various applications, including the correction of genetic defects or mutations within human genome. application disease research is anticipated to solve a multitude intricate molecular biology challenges encountered life science research. Here, we review fundamental principles underlying its recent neurodegenerative diseases, cardiovascular autoimmune related cancer, focusing on modeling therapy these diseases. Finally, provide an overview limitations future prospects associated with employing diseases study treatment.

Язык: Английский

Процитировано

15
Optimizing mesenchymal stem cell extracellular vesicles for chronic wound healing: Bioengineering, standardization, and safety DOI Creative Commons
Yusuke Shimizu, Edward Hosea Ntege, Yoshikazu Inoue

и другие.

Regenerative Therapy, Год журнала: 2024, Номер 26, С. 260 - 274

Опубликована: Июнь 1, 2024

Chronic wounds represent a significant global burden, afflicting millions with debilitating complications. Despite standard care, impaired healing persists due to factors like persistent inflammation and tissue regeneration. Mesenchymal stem cell (MSC)-derived extracellular vesicles (EVs) offer an innovative regenerative medicine approach, delivering cell-derived therapeutic cargo in engineered nanoscale delivery systems. This review examines pioneering bioengineering strategies engineer MSC-EVs into precision nanotherapeutics for chronic wounds. Emerging technologies CRISPR gene editing, microfluidic manufacturing, biomimetic systems are highlighted their potential enhance MSC-EV targeting, optimize enrichment, ensure consistent clinical-grade production. However, key hurdles remain, including batch variability, rigorous safety assessment tumorigenicity, immunogenicity, biodistribution profiling. Crucially, collaborative frameworks harmonizing regulatory science patient advocacy hold the expediting clinical translation. By overcoming these challenges, could catalyze new era of off-the-shelf therapies, restoring hope afflicted by non-healing

Язык: Английский

Процитировано

11
Core-shell vector-mediated co-delivery of CRISPR/Cas9 system and hydrophobic drugs against triple-negative breast cancer stem cells DOI
Tong Wang, Tianyi Chen, Dazhao Li

и другие.

Journal of Controlled Release, Год журнала: 2025, Номер 378, С. 1080 - 1091

Опубликована: Янв. 5, 2025

Язык: Английский

Процитировано

2
Synthetic Polymers for Drug, Gene, and Vaccine Delivery DOI Creative Commons
Yajun Xu, Jie Chen, Jianxun Ding

и другие.

Polymer science & technology., Год журнала: 2025, Номер unknown

Опубликована: Апрель 25, 2025

Язык: Английский

Процитировано

2
Emerging technology has a brilliant future: the CRISPR-Cas system for senescence, inflammation, and cartilage repair in osteoarthritis DOI Creative Commons
Shicheng Jia,

Rongji Liang,

Jiayou Chen

и другие.

Cellular & Molecular Biology Letters, Год журнала: 2024, Номер 29(1)

Опубликована: Май 2, 2024

Abstract Osteoarthritis (OA), known as one of the most common types aseptic inflammation musculoskeletal system, is characterized by chronic pain and whole-joint lesions. With cellular molecular changes including senescence, inflammatory alterations, subsequent cartilage defects, OA eventually leads to a series adverse outcomes such disability. CRISPR-Cas-related technology has been proposed explored gene therapy, offering potential gene-editing tools that are in spotlight. Considering genetic multigene regulatory mechanisms OA, we systematically review current studies on CRISPR-Cas for improving terms inflammation, damage summarize various strategies delivering CRISPR products, hoping provide new perspective treatment taking advantage technology.

Язык: Английский

Процитировано

7