Human Gene Therapy, Год журнала: 2024, Номер unknown
Опубликована: Дек. 26, 2024
Worldwide, thousands of male patients who carry ATP Binding Cassette Subfamily D Member 1 (
Язык: Английский
ACS Nano, Год журнала: 2025, Номер unknown
Опубликована: Янв. 21, 2025
Nucleic acid therapeutics represent a highly promising treatment approach in modern medicine, treating diseases at the genetic level. However, these face numerous challenges practical applications, particularly regarding their stability, effectiveness, cellular uptake efficiency, and limitations delivering them specifically to target tissues. To overcome obstacles, researchers have developed various innovative delivery systems, including viral vectors, lipid nanoparticles, polymer inorganic protein carriers, exosomes, antibody oligonucleotide conjugates, DNA nanostructure-based systems. These systems enhance therapeutic efficacy of nucleic drugs by improving targeting specificity, half-life vivo. In this review, we systematically discuss different types drugs, analyze major barriers encountered delivery, summarize current research progress emerging We also highlight latest advancements application for diseases, infectious cancer, brain wound healing. This review aims provide comprehensive overview drug systems' status future directions integrating nanotechnology, biomaterials science, gene editing technologies, emphasizing transformative potential precision medicine.
Язык: Английский
Процитировано
2
Journal of Neurorestoratology, Год журнала: 2025, Номер unknown, С. 100204 - 100204
Опубликована: Март 1, 2025
Язык: Английский
Процитировано
0
Molecular Therapy — Methods & Clinical Development, Год журнала: 2025, Номер 33(1), С. 101420 - 101420
Опубликована: Янв. 25, 2025
Язык: Английский
Процитировано
0
Advanced Biology, Год журнала: 2025, Номер unknown
Опубликована: Фев. 14, 2025
Abstract Diseases of the nervous system are recognized as second leading cause death worldwide. The global prevalence neurological diseases, such Huntington's disease, Alzheimer's and Parkinson's disease has seen a significant rise due to increasing proportion aging population. discovery clustered regularly interspaced short palindromic repeats (CRISPR) genome editing technique paved way for universal diseases treatment. However, finding safe effective method deliver CRISPR gene‐editing tools remains main challenge therapies in vivo. Adeno‐associated virus (AAV) is currently one most commonly used vector systems, but some issues remain unresolved, including capsid immunogenicity, off‐target mutations, potential genotoxicity. To address these concerns, researchers actively encouraging development new delivery like virus‐like particles nanoparticles. These novel systems have enhance targeting efficiency, thereby offering possible solutions current challenges. This article reviews vectors disorders treatment explores overcome limitations systems. Additionally, strategies highlighted valuable studying challenges opportunities that present.
Язык: Английский
Процитировано
0
Journal of Controlled Release, Год журнала: 2025, Номер unknown, С. 113641 - 113641
Опубликована: Март 1, 2025
Язык: Английский
Процитировано
0
Journal of Nanobiotechnology, Год журнала: 2025, Номер 23(1)
Опубликована: Апрель 1, 2025
Extracellular particles (EPs), including extracellular vesicles (EVs) and non-vesicular (NVEPs), are multimolecular biomaterials released by cells that play a crucial role in intercellular communication. Recently, new subtypes of EPs associated with central nervous system (CNS), such as exophers supermeres have been identified. These provide perspectives for understanding the pathological progression CNS disorders confer potential diagnostic value liquid biopsies neurodegenerative diseases (NDs). Moreover, emerged promising drug delivery vehicles targeted platforms CNS-specific therapies. In this review, we delineate landscape EP their roles pathophysiology diseases. We also review recent advances EP-based diagnosis NDs highlight importance analytical single-particle resolution exploitation biomarkers. Furthermore, summarize application engineered EVs treatment outline underexplored NVEPs novel therapeutic agents.
Язык: Английский
Процитировано
0
American Journal of Neuroradiology, Год журнала: 2025, Номер 46(4), С. 646 - 647
Опубликована: Апрель 1, 2025
Язык: Английский
Процитировано
0
Nanomedicine, Год журнала: 2025, Номер unknown, С. 1 - 12
Опубликована: Апрель 5, 2025
This study aimed to mitigate neuropathic pain behavior in a sciatic nerve transection (SNT)-induced mouse model by delivering anti-inflammatory cytokines - interleukin-4 (IL-4), interleukin-10 (IL-10), and transforming growth factor-beta 1 (TGF-β1) via poly(d,l-lactic-co-glycolic acid) (PLGA) nanoparticles (NPs). Upon gene delivery of IL-4, IL-10, TGF- β1, the effects induction microglia M2 polarization were evaluated. Plasmid (IL-4, TGF-β1)-encapsulated PLGA NPs (PLGA@IL-4, PLGA@IL-10, PLGA@TGF-β1) synthesized characterized for size, zeta potential, cellular toxicity, uptake. The analgesic effect using was then assessed pain. Gene TGF-β1 showed significant LPS-treated cells IL-4 strongly promoted vitro. successfully delivered cytokine-coding genes into spinal cord cells, specifically targeting microglia. PLGA@IL-4, PLGA@TGF-β1 produced SNT-induced model. Notably, PLGA@IL-4 demonstrated most effective remarkably long-lasting effect, enhancing therapy overexpression could be promising strategy treatment
Язык: Английский
Процитировано
0
Frontiers in Pharmacology, Год журнала: 2025, Номер 16
Опубликована: Апрель 7, 2025
Background Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease characterized by progressive degeneration of motor neurons, marked complex pathological mechanisms and lack effective treatments. Despite substantial global research efforts, no comprehensive bibliometric analysis has systematically mapped the evolution ALS biomarkers, therapeutic targets, pharmacological advancements. Methods This study, based on 4,250 publications retrieved from Web Science Core Collection (2005–2025), employs tools such as CiteSpace VOSviewer to conduct first multidimensional trends in drug research. Results The results revealed contributions 20,168 authors across 92 countries, with annual growing at an average rate 16.5%. United States dominated output, accounting for 34.07% (n=1,448, TLCS=7,100), while Kingdom achieved highest impact 68 citations per article. Leading institutions, including University Oxford Milan, consistently produced high-impact studies. Pioneering scholars Turner MR Kiernan MC made significant advancing targets discovery. interdisciplinary integration molecular biology genetics emerged core driver progress Neurofilament light chain (NfL), antisense oligonucleotide (ASO) drugs, transcranial magnetic stimulation (TMS), oxygen free radicals (oxidative stress), gene therapy have remained central focuses field. Looking ahead, stem cell therapy, blood-brain barrier (BBB) penetration technologies, skeletal muscle targeting are poised emerge prominent directions. Conclusion dominates productivity, whereas demonstrates superior citation influence. China’s publication volume, its limited underscores necessity enhanced methodological rigor strategic international collaboration. Current priorities encompass NfL, TMS, ASO therapies, emerging innovations BBB technologies showing promise. Future directions should prioritize biomarker standardization, optimization delivery systems, Clinical Translation.
Язык: Английский
Процитировано
0
Translational Neurodegeneration, Год журнала: 2025, Номер 14(1)
Опубликована: Апрель 16, 2025
Abstract Amyotrophic lateral sclerosis (ALS) is a fatal motor neuron disease that remains incurable. Although the etiologies of ALS are diverse and precise pathogenic mechanisms not fully understood, approximately 20% cases caused by genetic factors. Therefore, advancing targeted gene therapies holds significant promise, at least for patients with etiologies. In this review, we summarize main strategies techniques current based on risk genes, review recent findings from animal studies clinical trials. Additionally, highlight ALS-related genes well-understood potential numerous emerging gene-targeted therapeutic approaches ALS.
Язык: Английский
Процитировано
0