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https://www.readbyqxmd.com/read/30295175/considerations-for-preclinical-safety-assessment-of-adeno-associated-virus-gene-therapy-products
#1
Basel T Assaf, Laurence O Whiteley
Progress in understanding the molecular bases of human health and disease in recent decades has flourished making it possible for the field of gene therapy (GT) to offer new possibilities for treating, and even curing, a plethora of medical conditions such as monogenic disorders and metabolic diseases. GT is a therapeutic intervention to genetically alter or modify living cells by means of gene delivery achieved using either viral vectors or nonviral vectors, with adeno-associated virus (AAV) vectors constituting market-share majority...
October 7, 2018: Toxicologic Pathology
https://www.readbyqxmd.com/read/30293334/-the-clinical-significance-of-circulating-follicular-helper-t-cells-in-patients-with-anti-neutrophil-cytoplasmic-myeloperoxidase-antibody-associated-vasculitis
#2
S Wang, M J Zheng, X L Zhou, Y Q Liu, Z W Shuai
Objective: To investigate the change of circulating follicular helper T cells (cTfh) in patients with anti-neutrophil cytoplasmic myeloperoxidase antibody-associated vasculitis (MPO-AAV), and to analyze the relationship between cTfh and disease activity. Methods: Thirty-eight untreated MPO-AAV patients (patient group) and thirty-eight healthy volunteers (control group) were enrolled in this study. cTfh and membrane expression of inducible co-stimulator (ICOS) and programmed cell death protein 1 (PD-1) were detected by flow cytometry (FCM)...
October 1, 2018: Zhonghua Nei Ke za Zhi [Chinese Journal of Internal Medicine]
https://www.readbyqxmd.com/read/30280954/toxicology-and-pharmacology-of-an-aav-vector-expressing-codon-optimized-rpgr-in-rpgr-deficient-rd9-mice
#3
Chunjuan Song, Thomas J Conlon, Wen-Tao Deng, Kirsten E Coleman, Ping Zhu, Cayrn Plummer, Savitri Mandapati, Mailin Van Hoosear, Kari B Green, Peter Sonnentag, Alok K Sharma, Adrian Timmers, Paulette Robinson, David R Knop, William W Hauswirth, Jeffrey David Chulay, Mark S Shearman, Guo-Jie Ye
AGTC is developing a recombinant adeno-associated virus (rAAV) vector AGTC-501, also designated AAV2tYF-GRK1-RPGRco, to treat retinitis pigmentosa (RP) in patients with mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. The vector contains a codon-optimized human RPGR cDNA (RPGRco) driven by a photoreceptor-specific promoter (G protein-coupled receptor kinase 1, GRK1) and is packaged in an AAV2 capsid with three surface tyrosine residues changed to phenylalanine (AAV2tYF). We conducted a safety and potency study of this vector administered by subretinal injection in the naturally occurring RPGR-deficient Rd9 mouse model...
October 3, 2018: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/30274824/endogenous-il-10-maintains-immune-tolerance-but-il-10-gene-transfer-exacerbates-autoimmune-cholangitis
#4
Yu-Hsin Hsueh, Hung-Wen Chen, Bi-Jhen Syu, Chia-I Lin, Patrick S C Leung, M Eric Gershwin, Ya-Hui Chuang
The immunomodulatory effect of IL-10 as an immunosuppressive and anti-inflammatory cytokine is well known. Taking advantage of our established mouse model of autoimmune cholangitis using 2-octynoic acid conjugated ovalbumin (2-OA-OVA) induction, we compared liver pathology, immune cell populations and antimitochondrial antibodies between IL-10 knockout and wild type mice immunized with 2-OA-OVA. At 10 weeks post immunization, portal inflammation and fibrosis were more severe in 2-OA-OVA immunized IL-10 knockout mice than in wild type mice...
September 28, 2018: Journal of Autoimmunity
https://www.readbyqxmd.com/read/30262824/ginsenoside-rg1-protects-against-ischemic-reperfusion-induced-neuronal-injury-through-mir-144-nrf2-are-pathway
#5
Shi-Feng Chu, Zhao Zhang, Xin Zhou, Wen-Bin He, Chen Chen, Piao Luo, Dan-Dan Liu, Qi-di Ai, Hai-Fan Gong, Zhen-Zhen Wang, Hong-Shuo Sun, Zhong-Ping Feng, Nai-Hong Chen
Ginsenoside Rg1 (Rg1), a saponin extracted from Panax ginseng, has been well documented to be effective against ischemic/reperfusion (I/R) neuronal injury. However, the underlying mechanisms remain obscure. In the present study, we investigated the roles of Nrf2 and miR-144 in the protective effects of Rg1 against I/R-induced neuronal injury. In OGD/R-treated PC12 cells, Rg1 (0.01-1 μmol/L) dose-dependently attenuated the cell injury accompanied by prolonging nuclear accumulation of Nrf2, enhancing the transcriptional activity of Nrf2, as well as promoting the expression of ARE-target genes...
September 27, 2018: Acta Pharmacologica Sinica
https://www.readbyqxmd.com/read/30258218/cell-type-specific-activation-of-mitogen-activated-protein-kinase-in-d1-receptor-expressing-neurons-of-the-nucleus-accumbens-potentiates-stimulus-reward-learning-in-mice
#6
Md Ali Bin Saifullah, Taku Nagai, Keisuke Kuroda, Bolati Wulaer, Toshitaka Nabeshima, Kozo Kaibuchi, Kiyofumi Yamada
Medium spiny neurons (MSN) in the nucleus accumbens (NAc) are a fundamental component of various aspects of motivated behavior. Although mitogen-activated protein kinase (MAPK) signaling plays a crucial role in several types of learning, the cell type-specific role of MAPK pathway in stimulus-reward learning and motivation remains unclear. We herein investigated the role of MAPK in accumbal MSNs in reward-associated learning and memory. During the acquisition of Pavlovian conditioning, the number of phosphorylated MAPK1/3-positive cells was increased significantly and exclusively in the NAc core by 7-days of extensive training...
September 26, 2018: Scientific Reports
https://www.readbyqxmd.com/read/30258047/neuroprotection-of-retinal-ganglion-cells-by-a-novel-gene-therapy-construct-that-achieves-sustained-enhancement-of-brain-derived-neurotrophic-factor-tropomyosin-related-kinase-receptor-b-signaling
#7
Andrew Osborne, Tasneem Z Khatib, Lalana Songra, Amanda C Barber, Katie Hall, George Y X Kong, Peter S Widdowson, Keith R Martin
Previous studies have demonstrated that intravitreal delivery of brain-derived neurotrophic factor (BDNF) by injection of recombinant protein or by gene therapy can alleviate retinal ganglion cell (RGC) loss after optic nerve injury. BDNF gene therapy can improve RGC survival in experimental models of glaucoma, the leading cause of irreversible blindness worldwide. However, the therapeutic efficacy of BDNF supplementation alone is time limited at least in part due to BDNF receptor downregulation. Tropomyosin-related receptor kinase-B (TrkB) downregulation has been reported in many neurological diseases including glaucoma, potentially limiting the effect of sustained or repeated BDNF delivery...
September 26, 2018: Cell Death & Disease
https://www.readbyqxmd.com/read/30249384/evaluation-of-the-activity-levels-of-rat-fviii-and-human-fviii-delivered-by-adeno-associated-viral-vectors-both-in-vitro-and-in-vivo
#8
Wei Zhang, Jianhua Mao, Yan Shen, Guowei Zhang, Yanyan Shao, Zheng Ruan, Yun Wang, Wenman Wu, Xuefeng Wang, Jiang Zhu, Saijuan Chen, Weidong Xiao, Xiaodong Xi
The development of a novel coagulation factor VIII (FVIII) expression cassette with an enhanced activity for gene therapy of hemophilia A (HA) is essential. The biological properties of several non-human FVIII sequences, such as porcine and canine, have been evaluated. Here, we compared the activity level of rat FVIII (rFVIII) and human FVIII (hFVIII) by using single-chain and dual-chain strategies in 293 T cells and the HA mice. In both in vitro and hydrodynamic injection studies, the activity of rFVIII detected by the activated partial thromboplastin time assay was higher than that of hFVIII both by single-chain (~2...
September 20, 2018: Blood Cells, Molecules & Diseases
https://www.readbyqxmd.com/read/30245710/cortical-aav-cntf-gene-therapy-combined-with-intraspinal-mesenchymal-precursor-cell-transplantation-promotes-functional-and-morphological-outcomes-after-spinal-cord-injury-in-adult-rats
#9
Stuart I Hodgetts, Jun Han Yoon, Alysia Fogliani, Emmanuel A Akinpelu, Danii Baron-Heeris, Imke G J Houwers, Lachlan P G Wheeler, Bernadette T Majda, Sreya Santhakumar, Sarah J Lovett, Emma Duce, Margaret A Pollett, Tylie M Wiseman, Brooke Fehily, Alan R Harvey
Ciliary neurotrophic factor (CNTF) promotes survival and enhances long-distance regeneration of injured axons in parts of the adult CNS. Here we tested whether CNTF gene therapy targeting corticospinal neurons (CSN) in motor-related regions of the cerebral cortex promotes plasticity and regrowth of axons projecting into the female adult F344 rat spinal cord after moderate thoracic (T10) contusion injury (SCI). Cortical neurons were transduced with a bi cis tronic adeno-associated viral vector (AAV1) expressing a secretory form of CNTF coupled to mCHERRY (AAV-CNTFmCherry ) or with control AAV only (AAV-GFP) two weeks prior to SCI...
2018: Neural Plasticity
https://www.readbyqxmd.com/read/30245625/p58-ipk-is-an-endogenous-neuroprotectant-for-retinal-ganglion-cells
#10
Todd McLaughlin, Narayan Dhimal, Junhua Li, Joshua Jianxin Wang, Sarah Xin Zhang
p58IPK is an endoplasmic reticulum (ER)-resident chaperone playing a critical role in facilitating protein folding and protein homeostasis. Previously, we have demonstrated that p58IPK is expressed broadly in retinal neurons including retinal ganglion cells (RGCs) and loss of p58IPK results in age-related RGC degeneration. In the present study, we investigate the role of p58IPK in neuroprotection by in vitro and in vivo studies using primary RGC culture and two well-established disease-relevant RGC injury models: retinal ischemia/reperfusion (I/R) and microbead-induced ocular hypertension...
2018: Frontiers in Aging Neuroscience
https://www.readbyqxmd.com/read/30245471/tissue-dependent-expression-and-translation-of-circular-rnas-with-recombinant-aav-vectors-in-vivo
#11
Rita M Meganck, Erin K Borchardt, Ruth M Castellanos Rivera, Miranda L Scalabrino, Jeremy E Wilusz, William F Marzluff, Aravind Asokan
Circular RNAs (circRNAs) are long-lived, covalently closed RNAs that are abundantly expressed and evolutionarily conserved across eukaryotes. Possible functions ranging from microRNA (miRNA) and RNA binding protein sponges to regulators of transcription and translation have been proposed. Here we describe the design and characterization of recombinant adeno-associated viral (AAV) vectors packaging transgene cassettes containing intronic sequences that promote backsplicing to generate circularized RNA transcripts...
August 18, 2018: Molecular Therapy. Nucleic Acids
https://www.readbyqxmd.com/read/30243083/co-expression-of-akt1-and-wnt11-promotes-the-proliferation-and-cardiac-differentiation-of-mesenchymal-stem-cells-and-attenuates-hypoxia-reoxygenation-induced-cardiomyocyte-apoptosis
#12
Bangdang Chen, Xiaocui Chen, Cheng Liu, Jihong Li, Fen Liu, Ying Huang
Mesenchymal stem cells (MSCs) transplantation has emerged as a promising therapeutic strategy for acute myocardial infarction. However, there are still limitations for this therapy, such as low survival rate and poor cardiac differentiation potential of MSCs. In this study, we genetically engineered MSCs using ex vivo adeno-associated virus (AAV) transduction to overexpress Akt1 and Wnt11, which are well-characterized genes involved in MSC proliferation and cardiac differentiation. Our results showed that infection with AAV-Akt1-Wnt11 significantly upregulated the growth and proliferation of MSCs, as compared with those infected with AAV-Akt1 or AAV-Wnt11...
September 19, 2018: Biomedicine & Pharmacotherapy, Biomédecine & Pharmacothérapie
https://www.readbyqxmd.com/read/30242692/scalable-production-and-purification-of-adeno-associated-viral-vectors-aav
#13
Daniel Blessing, Nicole Déglon, Bernard L Schneider
Here we describe methods for the production of adeno-associated viral (AAV) vectors by transient transfection of HEK293 cells grown in serum-free medium in orbital shaken bioreactors and the subsequent purification of vector particles. The protocol for expression of AAV components is based on polyethyleneimine (PEI) mediated transfection of a 2-plasmid system and is specified for production in milliliter to liter scales. After PEI and plasmid DNA (pDNA) complex formation the diluted cell culture is transfected without a prior concentration step or medium exchange...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/30210317/optogenetic-stimulation-of-gabaergic-neurons-in-the-globus-pallidus-produces-hyperkinesia
#14
Jun Tian, Yaping Yan, Wang Xi, Rui Zhou, Huifang Lou, Shumin Duan, Jiang Fan Chen, Baorong Zhang
The globus pallidus (GP) is emerging as a critical locus of basal ganglia control of motor activity, but the exact role of GABAergic GP neurons remain to be defined. By targeted expression of channelrhodopsin 2 (ChR2) in GABAergic neurons using the VGAT-ChR2-EYFP transgenic mice, we showed that optogenetic stimulation of GABAergic neurons in the right GP produced hyperkinesia. Optogenetic stimulation of GABAergic GP neurons increased c-Fos-positive cells in GP, M1 cortex, and caudate-putamen (CPu), and decreased c-Fos-positive cells in entopeduncular nucleus (EPN), compared to the contralateral hemisphere...
2018: Frontiers in Behavioral Neuroscience
https://www.readbyqxmd.com/read/30199099/reverse-transduction-can-improve-efficiency-of-aav-vectors-in-transduction-resistant-cells
#15
Esther J Lee, Tawana M Robinson, Jeffrey J Tabor, Antonios G Mikos, Junghae Suh
Reverse transduction, also known as substrate-mediated gene delivery, is a strategy in which viral vectors are first coated onto a surface that subsequently comes into contact with mammalian cells. The cells internalize the surface-attached vectors, resulting in transgene expression. We hypothesized that forcing the interaction between cells and adeno-associated virus (AAV) vectors through a reverse transduction format would increase in vitro gene delivery efficiencies of the vectors in transduction-resistant cells...
September 10, 2018: Biotechnology and Bioengineering
https://www.readbyqxmd.com/read/30195768/spliceosome-mediated-pre-mrna-trans-splicing-can-repair-cep290-mrna
#16
Scott J Dooley, Devin S McDougald, Krishna J Fisher, Jeanette L Bennicelli, Lloyd G Mitchell, Jean Bennett
Ocular gene therapy with recombinant adeno-associated virus (AAV) has shown vector-mediated gene augmentation to be safe and efficacious in the retina in one set of diseases (retinitis pigmentosa and Leber congenital amaurosis (LCA) caused by RPE65 deficiency), with excellent safety profiles to date and potential for efficacy in several additional diseases. However, size constraints imposed by the packaging capacity of the AAV genome restrict application to diseases with coding sequence lengths that are less than 5,000 nt...
September 7, 2018: Molecular Therapy. Nucleic Acids
https://www.readbyqxmd.com/read/30195763/one-vector-system-for-multiplexed-crispr-cas9-against-hepatitis-b-virus-cccdna-utilizing-high-capacity-adenoviral-vectors
#17
Maren Schiwon, Eric Ehrke-Schulz, Andreas Oswald, Thorsten Bergmann, Thomas Michler, Ulrike Protzer, Anja Ehrhardt
High-capacity adenoviral vectors (HCAdVs) devoid of all coding genes are powerful tools to deliver large DNA cargos into cells. Here HCAdVs were designed to deliver a multiplexed complete CRISPR/Cas9 nuclease system or a complete pair of transcription activator-like effector nucleases (TALENs) directed against the hepatitis B virus (HBV) genome. HBV, which remains a serious global health burden, forms covalently closed circular DNA (cccDNA) as a persistent DNA species in infected cells. This cccDNA promotes the chronic carrier status, and it represents a major hurdle in the treatment of chronic HBV infection...
September 7, 2018: Molecular Therapy. Nucleic Acids
https://www.readbyqxmd.com/read/30194601/gene-delivery-of-alpha-1-antitrypsin-using-recombinant-adeno-associated-virus-raav
#18
Sihong Song, Yuanqing Lu
The challenge for alpha-1-antitrypsin (AAT also known as SERPINA1) gene therapy is to achieve long term and high levels of AAT production. Recombinant adeno-associated virus (rAAV) vector has several advantages for AAT gene delivery including no viral genes in the vector, no requirement of integration for long-term transgene expression, low immunogenicity, and wide tropism. AAV-mediated AAT gene therapy has been developed and tested in animal models for AAT deficiency, type 1 diabetes, rheumatoid arthritis, and osteoporosis...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/30183384/virally-mediated-overexpression-of-glial-derived-neurotrophic-factor-elicits-age-and-dose-dependent-neuronal-toxicity-and-hearing-loss
#19
Omar Akil, Bas Blits, Lawrence R Lustig, Patricia A Leake
Contemporary cochlear implants (CI) are generally very effective for remediation of severe to profound sensorineural hearing loss, but outcomes are still highly variable. Auditory nerve survival is likely one of the major factors underlying this variability. Neurotrophin therapy therefore has been proposed for CI recipients, with the goal of improving outcomes by promoting improved survival of cochlear spiral ganglion neurons (SGN) and/or residual hair cells. Previous studies have shown that glial-derived neurotrophic factor (GDNF), brain-derived neurotrophic factor, and neurotrophin-3 can rescue SGNs following insult...
September 5, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/30180762/a-new-cancer-immunotherapy-based-on-a-cancer-cell-specific-nf-%C3%AE%C2%BAb-activating-gene-expression-technique
#20
Danyang Wang, Wei Dai, Jinke Wang
In recent years, the immunotherapy greatly contributes to the cancer treatment. However, the current cancer immunotherapies are still seriously prevented by the limited cancer cell-specific antigens. Therefore, scientists have to look for neoantigens by tumor sequencing and bioinformatics prediction for developing personalized cancer vaccines. Anyway, this is still an expensive, challenging and long-term exploration. Here we testified a new immunotherapy for cancers by developing a cancer cell-specific gene expression technique that employed a NF-κB-activating gene expression vector to express a protein or peptide on cancer cell surface...
September 5, 2018: Human Gene Therapy
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