Viral and Non-Viral Systems to Deliver Gene Therapeutics to Clinical Targets

International Journal of Molecular Sciences, Journal Year: 2024, Volume and Issue: 25(13), P. 7333 - 7333

Published: July 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.

Language: Английский

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Emerging Wearable Acoustic Sensing Technologies

Advanced Science, Journal Year: 2025, Volume and Issue: unknown

Published: Jan. 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.

Language: Английский

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Adeno-associated virus vector delivery to the brain: Technology advancements and clinical applications

Advanced Drug Delivery Reviews, Journal Year: 2024, Volume and Issue: 211, P. 115363 - 115363

Published: June 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.

Language: Английский

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Cardiac gene delivery using ultrasound: State of the field

Molecular Therapy — Methods & Clinical Development, Journal Year: 2024, Volume and Issue: 32(3), P. 101277 - 101277

Published: June 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

Language: Английский

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The road ahead to successful BBB opening and drug-delivery with focused ultrasound

Journal of Controlled Release, Journal Year: 2024, Volume and Issue: 372, P. 901 - 913

Published: July 15, 2024

Language: Английский

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Focused ultrasound widely broadens AAV-delivered Cas9 distribution and activity

Gene Therapy, Journal Year: 2025, Volume and Issue: unknown

Published: Feb. 1, 2025

Language: Английский

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Focused-ultrasound-mediated CRISPR-Cas9 gene editing in human induced pluripotent stem cells

Molecular Therapy, Journal Year: 2025, Volume and Issue: 33(3), P. 831 - 832

Published: Feb. 20, 2025

Language: Английский

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Two-step ultrasonic cavitation controlled delivery of brain exogenous nucleic acids for ischemic stroke using acoustic-cationic-polymeric-nanodroplets

Drug Delivery and Translational Research, Journal Year: 2025, Volume and Issue: unknown

Published: March 6, 2025

Inefficient and low-precision delivery of exogenous nucleic acids (ENA) severely limits gene therapy on ischemic stroke (IS). Two problems need to be urgently addressed improve the efficacy therapy; first, blood brain barrier (BBB) should open promote accumulation ENA or genetic material carriers in parenchyma, second, efficient into cells. Previous studies applied ultrasonic cavitation either for opening BBB inducing sonoporation deliver materials However, effectiveness two-step remains unclear, let alone controllably delivered parenchyma IS. This study systematically explored by using artificial acoustic-cationic-polymeric-nanodroplets (ACPNs). The results demonstrated that first focused ultrasound (FUS), set at parameters 3.3 MPa, 20 Hz, 200 cycles 5 s, stimulating intravascular ACPNs effectively opened allow nonactivated ACPN extravasation parenchyma. Then, extravascular enhanced second noninvasively efficiently controlled cells through sonoporation, particularly applying 60 9 s control FAM-eNA delivery, 3.6 7 pEGFP-C1 delivery. Overall, represented a potential strategy IS-targeted

Language: Английский

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Lipid nanoparticle (LNP) mediated mRNA delivery in neurodegenerative diseases

Journal of Controlled Release, Journal Year: 2025, Volume and Issue: unknown, P. 113641 - 113641

Published: March 1, 2025

Language: Английский

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Recent progress and challenges in gene therapy for pharmacoresistant focal epilepsy

Revue Neurologique, Journal Year: 2025, Volume and Issue: unknown

Published: March 1, 2025

Language: Английский

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