Extracellular Vesicle, Год журнала: 2024, Номер 4, С. 100057 - 100057
Опубликована: Ноя. 26, 2024
Язык: Английский
International Journal of Molecular Sciences, Год журнала: 2024, Номер 25(13), С. 7333 - 7333
Опубликована: Июль 4, 2024
Clustered regularly interspersed short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) technology has revolutionized the field of gene therapy as it enabled precise genome editing with unprecedented accuracy and efficiency, paving way for clinical applications to treat otherwise incurable genetic disorders. Typically, requires delivery multiple components target cells that, depending on platform used, may include messenger RNA (mRNA), complexes, DNA fragments. For purposes, these have be efficiently delivered into transplantable cells, such primary T lymphocytes or hematopoietic stem progenitor that are typically sensitive exogenous substances. This challenge limited broad applicability those strategies which efficient methods available. Electroporation-based methodologies been generally applied applications, but procedure-associated toxicity represented a major burden. With advent novel less disruptive deliver cargo is now possible safely editing, thus expanding strategies. In this review, we describe different systems available components, including viral non-viral systems, highlighting their advantages, limitations, recent applications. Recent improvements achieve cell specificity represent critical development enable in vivo targeting future will certainly play pivotal role field.
Язык: Английский
Процитировано
28
Advanced Science, Год журнала: 2025, Номер unknown
Опубликована: Янв. 3, 2025
Sound signals not only serve as the primary communication medium but also find application in fields such medical diagnosis and fault detection. With public healthcare resources increasingly under pressure, challenges faced by disabled individuals on a daily basis, solutions that facilitate low-cost private hold considerable promise. Acoustic methods have been widely studied because of their lower technical complexity compared to other solutions, well high safety threshold human body acoustic energy. Furthermore, with recent development artificial intelligence technology applied speech recognition, recognition devices, systems capable assisting interacting scenes are constantly being updated. This review meticulously summarizes sensing mechanisms, materials, structural design, multidisciplinary applications wearable devices health human-computer interaction. Further, advantages disadvantages different approaches used flexible various examined. Finally, current roadmap for future research analyzed based existing progress achieve more comprehensive personalized healthcare.
Язык: Английский
Процитировано
6
Advanced Drug Delivery Reviews, Год журнала: 2024, Номер 211, С. 115363 - 115363
Опубликована: Июнь 19, 2024
Adeno-associated virus (AAV) vectors have emerged as a promising tool in the development of gene therapies for various neurological diseases, including Alzheimer's disease and Parkinson's disease. However, blood–brain barrier (BBB) poses significant challenge to successfully delivering AAV brain. Strategies that can overcome BBB improve delivery efficiency brain are essential successful brain-targeted therapy. This review provides an overview existing strategies employed brain, direct intraparenchymal injection, intra-cerebral spinal fluid intranasal delivery, intravenous injection BBB-permeable AAVs. Focused ultrasound has technology noninvasive spatially targeted administered by injection. also summarizes each strategy's current preclinical clinical applications treating diseases. Moreover, this includes detailed discussion recent advances emerging focused ultrasound-mediated delivery. Understanding state-of-the-art these approaches is critical future fulfill great promise treatment.
Язык: Английский
Процитировано
10
Journal of Nanotheranostics, Год журнала: 2025, Номер 6(2), С. 10 - 10
Опубликована: Апрель 9, 2025
Nanotheranostics—where nanoscale materials serve both diagnostic and therapeutic functions—are rapidly transforming gene therapy by tackling critical delivery challenges. This review explores the design engineering of various nanoparticle systems (lipid-based, polymeric, inorganic, hybrid) to enhance stability, targeting, endosomal escape genetic payloads. We discuss how real-time imaging capabilities integrated into these platforms enable precise localization controlled release genes, improving treatment efficacy while reducing off-target effects. Key strategies overcome barriers (such as proton sponge effect photothermal disruption) achieve nuclear are highlighted, along with recent advances in stimuli-responsive that facilitate spatiotemporal control expression. Clinical trials preclinical studies demonstrate expanding role nanotheranostics managing cancer, inherited disorders, cardiovascular neurological diseases. further address regulatory manufacturing hurdles must be for widespread clinical adoption nanoparticle-based therapies. By synthesizing progress ongoing challenges, this underscores transformative potential effective, targeted, image-guided delivery.
Язык: Английский
Процитировано
1
Journal of Controlled Release, Год журнала: 2024, Номер 372, С. 901 - 913
Опубликована: Июль 15, 2024
Язык: Английский
Процитировано
8
Molecular Therapy — Methods & Clinical Development, Год журнала: 2024, Номер 32(3), С. 101277 - 101277
Опубликована: Июнь 5, 2024
Over the past two decades, there has been tremendous and exciting progress toward extending use of medical ultrasound beyond a traditional imaging tool. Ultrasound contrast agents, typically used for improved visualization blood flow, have explored as novel non-viral gene delivery vectors cardiovascular therapy. Given this adaptation to contrast-enhancing presents an image-guided site-specific technique with potential multi-gene repeatable protocols—overcoming some limitations alternative therapy approaches. In review, we provide overview studies date that employ cardiac using disease animal models summarize their key findings.Graphical abstract
Язык: Английский
Процитировано
6
Magnetic Resonance Imaging Clinics of North America, Год журнала: 2024, Номер 32(4), С. 681 - 698
Опубликована: Июль 26, 2024
Язык: Английский
Процитировано
3
Science Bulletin, Год журнала: 2024, Номер unknown
Опубликована: Сен. 1, 2024
Язык: Английский
Процитировано
3
International Journal of Molecular Sciences, Год журнала: 2024, Номер 25(23), С. 12485 - 12485
Опубликована: Ноя. 21, 2024
This review explores recent advancements in gene therapy as a potential treatment for neurodegenerative diseases, focusing on intervention mechanisms, administration routes, and associated limitations. Following the PRISMA procedure guidelines, we systematically analyzed studies published since 2020 using PICO framework to derive reliable conclusions. The efficacy of various therapies was evaluated Parkinson's disease (n = 12), spinal muscular atrophy 8), Huntington's 3), Alzheimer's amyotrophic lateral sclerosis 6). For each condition, assessed therapeutic approach, curative or disease-modifying potential, delivery methods, advantages, drawbacks, side effects. Results indicate that targeting specific genes are particularly effective monogenic disorders, with promising clinical outcomes expected near future. In contrast, polygenic primarily aim promote cell survival. A major challenge remains: translation animal model success human application. Additionally, while intracerebral methods enhance efficacy, they highly invasive. Despite these hurdles, represents frontier underscoring need continued research refine personalize treatments condition.
Язык: Английский
Процитировано
3
Gene Therapy, Год журнала: 2025, Номер unknown
Опубликована: Фев. 1, 2025
Язык: Английский
Процитировано
0