Article
Biochemistry & Molecular Biology
Ana Sofia Moreira, Sandra Bezemer, Tiago Q. Faria, Frank Detmers, Pim Hermans, Laurens Sierkstra, Ana Sofia Coroadinha, Cristina Peixoto
Summary: The use of affinity chromatography can simplify the purification process of viral vectors, resulting in high purity clinical-grade products. This technology contributes to increasing downstream process efficiency, reducing time-to-market.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Clinical Neurology
Guillem Modol-Caballero, Belen Garcia-Lareu, Mireia Herrando-Grabulosa, Sergi Verdes, Ruben Lopez-Vales, Gemma Pages, Miguel Chillon, Xavier Navarro, Assumpcio Bosch
Summary: Targeted expression of GDNF in muscle can delay disease progression in a mouse model of ALS by protecting neuromuscular function, increasing innervated neuromuscular junctions, preserving spinal motoneurons, and reducing glial reactivity. These beneficial effects are attributed to a paracrine protective mechanism from the muscle to the motoneurons mediated by GDNF, with no adverse secondary effects detected, highlighting the potential for ALS therapy.
Article
Multidisciplinary Sciences
Agata Antepowicz, Omar Habib, Freja Kirsebom, Cecilia Johansson, Deborah R. Gill, Stephen C. Hyde
Summary: In this study, gene delivery approaches using recombinant adeno-associated virus and simian immunodeficiency virus vectors were utilized to achieve sustained in vivo production of Palivizumab in a murine model. Pre-treatment with Palivizumab-expressing vectors provided complete protection against RSV-induced weight loss. This approach offers prophylaxis against RSV infection, potentially reducing treatment costs in vulnerable populations.
SCIENTIFIC REPORTS
(2021)
Review
Pharmacology & Pharmacy
Yvan Arsenijevic, Adeline Berger, Florian Udry, Corinne Kostic
Summary: This review provides an overview of lentiviral vector technologies, their application in ophthalmology, and their potential for gene therapy.
Article
Medicine, Research & Experimental
Lin Wu, Shuangxi Chen, Bing He, Guijuan Zhou, Yan Xu, Guanghua Zhu, Juan Xie, Limin Deng, Xuanwei Wen, Sijing Li, Zijian Xiao
Summary: This study focused on the influence of NAG on motor and sensory recovery after BPRA in rats and the underlying mechanisms. The results showed that NAG significantly accelerated the recovery of motor function, enhanced motoneuronal survival, inhibited the expression of inflammatory cytokines and pyroptosis pathway factors, facilitated axonal remyelination, and alleviated muscle atrophy. Additionally, NAG attenuated neuropathic pain following BPRA. In conclusion, NAG promotes functional motor recovery and alleviates neuropathic pain by enhancing motoneuronal survival and axonal remyelination and inhibiting the pyroptosis pathway after BPRA in rats.
JOURNAL OF TRANSLATIONAL MEDICINE
(2023)
Article
Pharmacology & Pharmacy
Alexandra McCarron, Nigel Farrow, Patricia Cmielewski, Emma Knight, Martin Donnelley, David Parsons
Summary: The lungs have evolved complex defenses to prevent foreign materials from entering epithelial cells, which can also affect gene transfer agents. Disrupting the integrity of the epithelium prior to gene delivery can enhance gene expression levels.
FRONTIERS IN PHARMACOLOGY
(2021)
Review
Biology
Xiaoyu Wang, Cuicui Ma, Roberto Rodriguez Labrada, Zhou Qin, Ting Xu, Zhiyao He, Yuquan Wei
Summary: Lentiviral vectors, derived from HIV, have been widely used in research and clinical trials for modifying eukaryotic cell genes, showing promising results in treating diseases such as leukemia, beta-thalassemia, and immunodeficiency disorders. Their safety and efficiency make them a valuable tool for human gene therapy.
SCIENCE CHINA-LIFE SCIENCES
(2021)
Review
Cell Biology
Ciara Shortiss, Linda Howard, Siobhan S. McMahon
Summary: Spinal cord injury is a devastating trauma that can lead to permanent disability and chronic issues, presenting a significant socioeconomic burden. Regenerative medicine aims to overcome injury and restore function through gene therapy and tissue engineering. Combining biomaterials and lentiviral vectors has been shown to be more effective in promoting repair and function restoration post SCI.
Article
Pharmacology & Pharmacy
Nicole Reyne, Patricia Cmielewski, Alexandra McCarron, Juliette Delhove, David Parsons, Martin Donnelley
Summary: The study evaluated the effect of lentiviral-mediated CFTR airway gene delivery on nasal PD in a CFTR knockout rat model, showing that the gene therapy resulted in a mean correction of 46% towards wild-type chloride response in treated CF rats.
FRONTIERS IN PHARMACOLOGY
(2021)
Article
Multidisciplinary Sciences
Chee-Hong Takahiro Yew, Narmatha Gurumoorthy, Fazlina Nordin, Gee Jun Tye, Wan Safwani Wan Kamarul Zaman, Jun Jie Tan, Min Hwei Ng
Summary: HIV-1-derived lentiviral vector is an efficient transporter for delivering desired genetic materials into targeted cells. Integrase-deficient lentiviral vector, with reduced integration rate, offers better safety and wider clinical applications.
Article
Genetics & Heredity
Maria Alejandra Puentes-Tellez, Oscar F. Sanchez, Felipe Rojas-Rodriguez, Eliana Benincore-Flore, Hector Barbosa, Carlos J. Almeciga Diaz
Summary: "Mucopolysaccharidosis type IVA (MPS IVA) is a lysosomal storage disease caused by deficiency of the GALNS enzyme, resulting in GAGs accumulation. This study demonstrates that transduction of GALNS and SUMF1 genes into MPS IVA fibroblasts led to increased GALNS enzyme activity and reduced lysosomal mass. Patient-specific differences in response were associated with individual mutations. Additionally, evidence of autophagy impairment in MPS IVA fibroblasts was presented."
Article
Polymer Science
Paula Regina Gelinski Kempe, Mateus Vidigal de Castro, Victor Campos Khuriyeh, Benedito Barraviera, Rui Seabra Ferreira Jr, Alexandre Leite Rodrigues de Oliveira
Summary: Spinal cord injury leads to severe loss of motor and sensory function. Our study aimed to test the combined treatment of surgical repair with fibrin biopolymer and pharmacological treatment with dimethyl fumarate. Results showed that this combination can protect neurons and promote motor neuron regeneration.
Article
Biochemistry & Molecular Biology
Jie Gong, Rui Yang, Min Zhou, Lung-Ji Chang
Summary: This study developed advanced lentiviral vectors for targeted expression of factor VIII (FVIII) and conducted functional experiments in human endothelial and megakaryocytic cell lines. In a mouse model, one of the vectors showed excellent packaging and delivery efficiencies, with endothelial specificity and low immunogenicity, suggesting its potential for clinical applications.
MOLECULAR MEDICINE
(2023)
Article
Biochemistry & Molecular Biology
Fatai Lu, Guodong Zhang, Yingkang Zhu, Zunpeng Liu
Summary: This study found that EGCG has a neuroprotective effect against cell death caused by brachial plexus root avulsion, and this effect may be correlated with the inhibition of c-Jun phosphorylation.
CURRENT MEDICINAL CHEMISTRY
(2022)
Article
Pharmacology & Pharmacy
P. Cmielewski, J. Delhove, M. Donnelley, D. Parsons
Summary: The study established a rapid nasal PD technique for assessing the efficacy of CF therapies, showing that LV-CFTR gene therapy can improve chloride transport. This technique enables improved functional assessment of the bioelectrical ion transport defect for both current and potential CF therapies.
FRONTIERS IN PHARMACOLOGY
(2021)
Article
Biotechnology & Applied Microbiology
Arjanneke F. van de Merbel, Geertje van der Horst, Maaike H. van der Mark, Selas T. F. Bots, Diana J. M. van den Wollenberg, Corrina M. A. de Ridder, Debra Stuurman, Tilly Aalders, Sigrun Erkens-Schulz, Nadine van Montfoort, Wouter R. Karthaus, Niven Mehra, Minke Smits, Jack A. Schalken, Wytske M. van Weerden, Rob C. Hoeben, Gabri van der Pluijm
Summary: The study evaluated the oncolytic efficacy of the bioselected oncolytic reovirus mutant jin-3 in multiple human prostate cancer models, showing tumor tropism and potent anti-cancer immune responses.
CANCER GENE THERAPY
(2022)
Editorial Material
Immunology
Sally C. Kent, Arnaud Zaldumbide
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Biotechnology & Applied Microbiology
Tom J. Harryvan, Matteo Golo, Nicole Dam, Mark J. A. Schoonderwoerd, Elham Aida Farshadi, Marten Hornsveld, Rob C. Hoeben, Lukas J. A. C. Hawinkels, Vera Kemp
Summary: Gastrointestinal (GI) cancers are characterized by extensive tumor stroma. In this study, we investigated the interaction between commonly studied oncolytic viruses (adenovirus and reovirus) and the tumor stroma of GI cancers. We found that the reovirus can interact with the tumor stroma and induce apoptosis.
CANCER GENE THERAPY
(2022)
Article
Clinical Neurology
Kevin R. Keene, Johan M. de Nie, Mechteld J. Brink, Irene C. Notting, Jan J. G. M. Verschuuren, Hermien E. Kan, Jan-Willem M. Beenakker, Martijn R. Tannemaat
Summary: This study aimed to explore whether orthoptic measures, including an adapted Hess chart examination, can aid in diagnosing ocular myasthenia gravis (MG). The results showed that orthoptic measurements can be used to diagnose MG by quantifying extraocular muscle (EOM) weakness and fatiguability. Drift during persistent gaze on a Hess chart was found to be specific for MG, and could be used for diagnostic purposes.
JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY
(2023)
Article
Biochemistry & Molecular Biology
Diana J. M. van den Wollenberg, Vera Kemp, Martijn J. W. E. Rabelink, Rob C. Hoeben
Summary: This study demonstrates that reoviruses efficiently induce necroptosis in mouse cells but not in human cells, likely due to the RIPK3 expression defect in human cell lines.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Clinical Neurology
Esli D. Steenbeek, Willem Pondaag, Martijn R. Tannemaat, Erik W. Van Zwet, Martijn J. A. Malessy, Justus L. Groen
Summary: The value of needle electromyography (NEMG) in diagnosing the severity of traumatic radial nerve injury (RNI) can help determine the need for early surgical reconstruction.
Article
Virology
Selas T. F. Bots, Sanne L. L. Landman, Martijn J. W. E. Rabelink, Diana J. M. van den Wollenberg, Rob C. C. Hoeben
Summary: Pancreatic ductal adenocarcinoma (PDAC) is a highly aggressive malignancy with resistance to treatment. Oncolytic viruses have shown promise as a new treatment option by killing cancer cells. GoraVir, derived from a gorilla adenovirus, has demonstrated excellent activity against PDAC. It induces immunogenic cell death independent of antiviral responses, making it a potential candidate for oncolytic virotherapy.
Article
Endocrinology & Metabolism
Rene van Tienhoven, Maria J. L. Kracht, Arno R. van der Slik, Sofia Thomaidou, Anouk H. G. Wolters, Ben N. G. Giepmans, Juan Pablo Romero Riojas, Michael S. Nelson, Francoise Carlotti, Eelco J. P. de Koning, Rob C. Hoeben, Arnaud Zaldumbide, Bart O. Roep
Summary: Transcriptome analysis identified insulin-gene-derived transcripts in non-beta endocrine islet cells. Researchers studied alternative splicing of human INS mRNA in pancreatic islets and found an alternatively spliced INS product that is expressed in delta cells but not in beta cells. This variant encodes the complete insulin signal peptide and B chain, and its presence in delta cells may play a role in islet autoimmunity and pathology.
Article
Medicine, General & Internal
Tomi Suomi, Inna Starskaia, Ubaid Ullah Kalim, Omid Rasool, Maria K. Jaakkola, Toni Gronroos, Tommi Valikangas, Caroline Brorsson, Gianluca Mazzoni, Sylvaine Bruggraber, Lut Overbergh, David Dunger, Mark Peakman, Piotr Chmura, Seren Brunak, Anke M. Schulte, Chantal Mathieu, Mikael Knip, Riitta Lahesmaa, Laura L. Elo
Summary: This study aimed to identify transcriptional changes associated with disease progression in patients with recent-onset type 1 diabetes. They found that genes and pathways related to innate immunity were downregulated during the first year after diagnosis. Associations between gene expression changes and ZnT8A autoantibody positivity were also observed. Additionally, changes in the expression of 16 genes were found to predict the decline in C-peptide at 24 months, and increased B cell levels and decreased neutrophil levels were associated with rapid progression, consistent with previous reports.
Article
Clinical Neurology
Kevin R. Keene, Irene C. Notting, Jan J. G. M. Verschuuren, N. Voermans, Ronald. O. B. de Keizer, Jan-Willem M. Beenakker, Martijn R. Tannemaat, Hermien E. Kan
Summary: Quantitative MRI was used to study the extra-ocular muscles (EOM) in patients with myasthenia gravis (MG). The study found that chronic MG patients showed small increases in EOM volume and muscle fat fraction (FF), while patients with chronic progressive external ophthalmoplegia (CPEO) showed decreased volume and patients with Graves' ophthalmopathy (GO) showed increased volume. The severity of ophthalmoplegia in MG was not correlated with EOM volume, but was correlated in CPEO and oculopharyngeal muscular dystrophy (OPMD).
JOURNAL OF NEUROMUSCULAR DISEASES
(2023)
Article
Clinical Neurology
Deborah Hubers, Wouter Potters, Olivier Paalvast, Sterre de Jonge, Brian Doelkahar, Martijn Tannemaat, Luuk Wieske, Camiel Verhamme
Summary: This study developed a two-step artificial neural network model that can classify rest, muscle contraction, and artifacts in real-world n-EMG recordings with high accuracy. The classification of MUAP duration has moderate accuracy.
CLINICAL NEUROPHYSIOLOGY
(2023)
Letter
Neurosciences
Oystein Dunker, Marie U. Lie, Aristomo Andries, Kristian Bernhard Nilsen, Petter Omland, Andrew Reiner, Martijn R. Tannemaat, Robert H. Reijntjes, Joe F. Jabre
CLINICAL NEUROPHYSIOLOGY PRACTICE
(2023)
Article
Biotechnology & Applied Microbiology
Willemijn van der Wulp, Dennis F. G. Remst, Michel G. D. Kester, Renate S. Hagedoorn, Paul W. H. I. Parren, Sander I. van Kasteren, Janine Schuurman, Rob C. Hoeben, Maaike E. Ressing, Boris Bleijlevens, Mirjam H. M. Heemskerk
Summary: The antibody-epitope conjugates (AECs) are a promising new therapeutic strategy that redirects virus-specific T-cells towards cancer cells. This study demonstrates that AECs with epitopes fused to the heavy chain of antibodies can result in higher levels of T-cell activation compared to AECs with epitopes fused to the light chain. Furthermore, these AECs can efficiently deliver the epitope to different cancer cell lines and reduce tumor burden in vivo, especially when combined with immune checkpoint blockade.
CANCER GENE THERAPY
(2023)
Meeting Abstract
Biotechnology & Applied Microbiology
Selas T. F. Bots, Tom J. Harrijvan, Christianne Groeneveldt, Priscilla Kinderman, Vera Kemp, Nadine van Montfoort, Rob C. Hoeben