The delivery of transgenes to the central nervous system (CNS) can be a valuable tool to treat CNS diseases. Various systems for the delivery to the CNS have been developed; vascular delivery of viral vectors being most recent. Here, we investigated gene transfer to the CNS by intravenous injection of recombinant adenoviral vectors, containing green fluorescence protein (GFP) as a reporter gene. Expression of GFP was first observed 6 days after the gene transfer, peaked at 14 days, and almost diminished after 28 days. The observed expression of GFP in the CNS was highly localized to hippocampal CA regions of cerebral neocortex, inferior colliculus of midbrain, and granular cell and Purkinje cell layers of cerebellum. It is concluded that intravenous delivery of adenoviral vectors can be used for gene delivery to the CNS, and hence the technique could be beneficial to gene therapy.
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http://dx.doi.org/10.3346/jkms.2003.18.1.108 | DOI Listing |
CNS Neurosci Ther
December 2024
Central Laboratory of The Lishui Hospital of Wenzhou Medical University, The First Affiliated Hospital of Lishui University, Lishui People's Hospital, Lishui, Zhejiang, China.
Ther Deliv
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Department of Pharmaceutics, University Institute of Pharma Sciences, Chandigarh University, Mohali, India.
Ovarian cancer remains one of the main causes of human mortality, accounting for millions of deaths every year. Despite of several clinical options such as chemotherapy, photodynamic therapy (PDT), hormonal treatment, radiation therapy, and surgery to manage this disease, the mortality rate is still very high. This alarming statistic highlights the urgent need for innovative approaches to improve both diagnosis and treatment.
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December 2024
Gulbenkian Institute for Molecular Medicine, Faculdade de Medicina da Universidade de Lisboa, Av. Prof. Egas Moniz, Lisbon, 1649-028, Portugal.
Brain metastases (BM) are frequently found in cancer patients and, though their precise incidence is difficult to estimate, there is evidence for a correlation between BM and specific primary cancers, such as lung, breast, and skin (melanoma). Among all these, breast cancer is the most frequently diagnosed among women and, in this case, BM cause a critical reduction of the overall survival (OS), especially in triple negative breast cancer (TNBC) patients. The main challenge of BM treatment is the impermeable nature of the blood-brain barrier (BBB), which shields the central nervous systems (CNS) from chemotherapeutic drugs.
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December 2024
Preclinical Safety (PCS), Novartis Biomedical Research, Cambridge, MA, USA.
Administration of AAV-based gene therapies into the intra-cerebrospinal fluid (CSF) compartments via routes such as lumbar puncture (LP) has been implemented as an alternative to intravenous dosing to target the CNS regions. This route enables lower doses, decreases systemic toxicity, and circumvents intravascular pre-existing anti-AAV antibodies. In this study, AAV9-GFP vectors were administered via LP to juvenile cynomolgus macaques with and without pre-existing serum anti-AAV9 antibodies at a 5.
View Article and Find Full Text PDFInt J Biol Macromol
December 2024
Department of Biotechnology, Faculty of Science, Mahidol University, Bangkok 10400, Thailand. Electronic address:
Delivering double-stranded RNA (dsRNA) in shrimp is challenging due to the lack of an effective carrier system. This study optimized chitosan nanoparticles (CNs) from two sources-α-chitosan from shrimp and β-chitosan from squid-to encapsulate antiviral dsRNA for oral administration via shrimp feed. Using response surface methodology (RSM), formulations were refined for encapsulation efficiency, particle size, polydispersity index, and zeta potential.
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