Magnetic Resonance Imaging Clinics of North America, Journal Year: 2024, Volume and Issue: 32(4), P. 681 - 698
Published: July 26, 2024
Language: Английский
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
Citations
27
Trends in Molecular Medicine, Journal Year: 2024, Volume and Issue: 30(3), P. 263 - 277
Published: Jan. 11, 2024
Language: Английский
Citations
20
Molecular Therapy — Nucleic Acids, Journal Year: 2025, Volume and Issue: 36(1), P. 102457 - 102457
Published: Jan. 18, 2025
Language: Английский
Citations
2
Nature Communications, Journal Year: 2024, Volume and Issue: 15(1)
Published: June 10, 2024
Language: Английский
Citations
14
Advanced Drug Delivery Reviews, Journal Year: 2024, Volume and Issue: 208, P. 115274 - 115274
Published: March 6, 2024
Ultrasound enhances drug delivery into the central nervous system (CNS) by opening barriers between blood and CNS triggering release of drugs from carriers. A key challenge in translating setups vitro to vivo settings is achieving equivalent acoustic energy delivery. Multiple devices have now been demonstrated focus ultrasound brain, with concepts emerging also target spinal cord. Clinical trials date used facilitate blood–brain barrier. While most focused on feasibility safety considerations, therapeutic benefits are beginning emerge. To advance translation these technologies for applications, researchers should standardise exposure protocol fine-tune parameters. Computational modelling be increasingly as a core component develop both delivering accurate reproducible CNS. This field holds promise transformative advancements management pharmacological treatment complex challenging disorders.
Language: Английский
Citations
13
Proceedings of the National Academy of Sciences, Journal Year: 2024, Volume and Issue: 121(26)
Published: June 17, 2024
Advancing our understanding of brain function and developing treatments for neurological diseases hinge on the ability to modulate neuronal groups in specific areas without invasive techniques. Here, we introduce Airy-beam holographic sonogenetics (AhSonogenetics) as an implant-free, cell type–specific, spatially precise, flexible neuromodulation approach freely moving mice. AhSonogenetics utilizes wearable ultrasound devices manufactured using 3D-printed metasurfaces. These are designed manipulate neurons genetically engineered express ultrasound-sensitive ion channels, enabling precise modulation populations. By dynamically steering focus Airy beams through frequency tuning, is capable modulating populations within subregions striatum. One notable feature its flexibly stimulate either left or right striatum a single mouse. This flexibility achieved by simply switching acoustic metasurface device, eliminating need multiple implants interventions. AhSonogentocs also integrates seamlessly with vivo calcium recording via fiber photometry, showcasing compatibility optical modalities cross talk. Moreover, can generate double foci bilateral stimulation alleviate motor deficits Parkinson’s disease advancement significant since many disorders, including disease, involve dysfunction regions. type–specific procedures, provides powerful tool investigating intact neural circuits offers promising interventions disorders.
Language: Английский
Citations
12
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
Citations
10
Parkinsonism & Related Disorders, Journal Year: 2024, Volume and Issue: 122, P. 106040 - 106040
Published: Feb. 12, 2024
Language: Английский
Citations
9
Advanced Drug Delivery Reviews, Journal Year: 2024, Volume and Issue: 205, P. 115176 - 115176
Published: Jan. 9, 2024
Language: Английский
Citations
7
International Journal of Pharmaceutics, Journal Year: 2024, Volume and Issue: 666, P. 124800 - 124800
Published: Oct. 5, 2024
Language: Английский
Citations
7