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Adeno associated

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https://www.readbyqxmd.com/read/28734478/ph-dependent-membrane-interactions-of-the-histidine-rich-cell-penetrating-peptide-lah4-l1
#1
Justine Wolf, Christopher Aisenbrey, Nicole Harmouche, Jesus Raya, Philippe Bertani, Natalia Voievoda, Regine Süss, Burkhard Bechinger
The histidine-rich designer peptide LAH4-L1 exhibits antimicrobial and potent cell-penetrating activities for a wide variety of cargo including nucleic acids, polypeptides, adeno-associated viruses, and nanodots. The non-covalent complexes formed between the peptide and cargo enter the cell via an endosomal pathway where the pH changes from neutral to acidic. Here, we investigated the membrane interactions of the peptide with phospholipid bilayers and its membrane topology using static solid-state NMR spectroscopy...
July 19, 2017: Biophysical Journal
https://www.readbyqxmd.com/read/28726562/gene-therapy-for-age-related-macular-degeneration
#2
Nicholas A Moore, Peter Bracha, Rehan M Hussain, Nuria Morral, Thomas A Ciulla
In neovascular age related macular degeneration (nAMD), gene therapy to chronically express anti-vascular endothelial growth factor (VEGF) proteins could ameliorate the treatment burden of chronic intravitreal therapy and improve limited visual outcomes associated with 'real world' undertreatment. Areas covered: In this review, the authors assess the evolution of gene therapy for AMD. Adeno-associated virus (AAV) vectors can transduce retinal pigment epithelium; one such early application was a phase I trial of AAV2-delivered pigment epithelium derived factor gene in advanced nAMD...
July 20, 2017: Expert Opinion on Biological Therapy
https://www.readbyqxmd.com/read/28726522/steerable-induction-of-the-thymosin-%C3%A3-4-mrtf-a-pathway-via-aav-based-overexpression-induces-therapeutic-neovascularization
#3
Tilman Ziegler, Markus Kraus, Wira Husada, Florian Gesenhues, Qui Jiang, Olaf Pinkenburg, Teresa Trenkwalder, Karl-Ludwig Laugwitz, Ferdinand le Noble, Christian Weber, Christian Kupatt, Rabea Hinkel
Viral vectors have been frequently used in a variety of preclinical animal models to deliver genetic constructs into tissues. Among the vectors used, adeno-associated viral vectors (AAVs) may be targeted to specific tissues, depending on the serotype used. Moreover, they show robust expression for prolonged periods of time and have a low immunogenic potential. Furthermore, AAVs, unlike other vector systems, only display a low rate of genomic integration. However, to ensure efficient transgene production, expression is typically driven by constitutively active promoters, such as the CMV promotor...
July 20, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28726496/a-preclinical-study-in-rhesus-macaques-for-cystic-fibrosis-to-assess-gene-transfer-and-transduction-by-aav1-and-aav5-with-a-dual-luciferase-reporter-system
#4
William Guggino, Janet Benson, JeanClare Seagrave, Ziying Yan, John F Engelhardt, Guangping Gao, Thomas J Conlon, Liudmila Cebotaru
Cystic fibrosis (CF) is an autosomal recessive disease that is potentially treatable by gene therapy. Since the identification of the gene encoding CFTR, a number of preclinical and clinical trials have been conducted using the first generation of adeno-associated virus, AAV2. All these studies showed that AAV gene therapy for CF is safe, but clinical benefit was not clearly demonstrated. Thus, a new generation of AAV Vectors based on other serotypes is needed to move the field forward. Here we have tested two AAV serotypes (AAV1 and AAV5), using a dual-luciferase reporter system with firefly and Renilla luciferase genes packaged into AAV1 or AAV5, respectively...
July 20, 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28726495/use-of-adeno-associated-virus-vector-for-cardiac-gene-delivery-in-large-animal-surgical-models-of-heart-failure
#5
Michael G Katz, Anthony S Fargnoli, Thomas Weber, Roger Hajjar, Charles Bridges
The advancement of gene-therapy-based approaches to treat heart disease represents a need for clinically-relevant animal models with characteristics equivalent to human pathologies. Rodent models of cardiac disease do not precisely reproduce heart failure phenotype and molecular defects. This has motivated researchers to use large animals whose heart size and physiological processes more similar and comparable to those of humans. Today, adeno-associated viruses (AAV)-based vectors are undoubtedly among the most promising DNA delivery vehicles...
July 20, 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28725658/mesenchymal-stem-cells-overexpressing-interleukin-10-promote-neuroprotection-in-experimental-acute-ischemic-stroke
#6
Masataka Nakajima, Chikako Nito, Kota Sowa, Satoshi Suda, Yasuhiro Nishiyama, Aki Nakamura-Takahashi, Yuko Nitahara-Kasahara, Kiwamu Imagawa, Tohru Hirato, Masayuki Ueda, Kazumi Kimura, Takashi Okada
Interleukin (IL)-10 is a contributing factor to neuroprotection of mesenchymal stem cell (MSC) transplantation after ischemic stroke. Our aim was to increase therapeutic effects by combining MSCs and ex vivo IL-10 gene transfer with an adeno-associated virus (AAV) vector using a rat transient middle cerebral artery occlusion (MCAO) model. Sprague-Dawley rats underwent 90 min MCAO followed by intravenous administration of MSCs alone or IL-10 gene-transferred MSCs (MSC/IL-10) at 0 or 3 hr after ischemia reperfusion...
September 15, 2017: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/28725275/ash-meeting-2016-developments-in-hemostaseology
#7
REVIEW
Clemens Feistritzer, Birgit Mosheimer
During the annual meeting of the American Society of Hematology (ASH) in San Diego/California, novel developments in the field of hemostaseology were presented. Alternative treatment strategies besides factor replacement were discussed for patients with hemophilia. One of the highlights of the meeting in this year's plenary session was the presentation of successful adeno-associated virus mediated gene transfer in patients with hemophilia B leading to sustained elevation of factor IX:C (FIX:c). Other alternative treatment approaches in patients with hemophilia A may include bispecific antibodies mimicking factor VIIIa (FVIIIa) activity or disrupting anticoagulant proteins...
2017: Memo
https://www.readbyqxmd.com/read/28723575/crispr-mediated-integration-of-large-gene-cassettes-using-aav-donor-vectors
#8
Rasmus O Bak, Matthew H Porteus
The CRISPR/Cas9 system has recently been shown to facilitate high levels of precise genome editing using adeno-associated viral (AAV) vectors to serve as donor template DNA during homologous recombination (HR). However, the maximum AAV packaging capacity of ∼4.5 kb limits the donor size. Here, we overcome this constraint by showing that two co-transduced AAV vectors can serve as donors during consecutive HR events for the integration of large transgenes. Importantly, the method involves a single-step procedure applicable to primary cells with relevance to therapeutic genome editing...
July 18, 2017: Cell Reports
https://www.readbyqxmd.com/read/28720467/intradermal-immunization-with-raav1-vector-induces-robust-memory-cd8-t-cell-responses-independently-of-transgene-expression-in-dcs
#9
Alexandre Ghenassia, David-Alexandre Gross, Stéphanie Lorain, Fabiola Tros, Dominique Urbain, Sofia Benkhelifa-Ziyyat, Alain Charbit, Jean Davoust, Pascal Chappert
Recombinant adeno-associated viral (rAAV) vectors exhibit interesting properties as vaccine carriers for their ability to induce long-lasting antibody responses. However, rAAV-based vaccines have been suggested to trigger functionally impaired long-term memory CD8(+) T cell responses, in part due to poor dendritic cell (DC) transduction. Such results, albeit limited to intramuscular immunization, undermined the use of rAAV as vaccine vehicles against intracellular pathogens. We report here that intradermal immunization with a model rAAV2/1-based vaccine drives the development of bona fide long-term memory CD8(+) T cell responses...
July 15, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/28715454/reduction-of-cav1-3-channels-in-dorsal-hippocampus-impairs-the-development-of-dentate-gyrus-newborn-neurons-and-hippocampal-dependent-memory-tasks
#10
Su-Hyun Kim, Ye-Ryoung Park, Boyoung Lee, Byungil Choi, Hyun Kim, Chong-Hyun Kim
Cav1.3 has been suggested to mediate hippocampal neurogenesis of adult mice and contribute to hippocampal-dependent learning and memory processes. However, the mechanism of Cav1.3 contribution in these processes is unclear. Here, roles of Cav1.3 of mouse dorsal hippocampus during newborn cell development were examined. We find that knock-out (KO) of Cav1.3 resulted in the reduction of survival of newborn neurons at 28 days old after mitosis. The retroviral eGFP expression showed that both dendritic complexity and the number and length of mossy fiber bouton (MFB) filopodia of newborn neurons at ≥ 14 days old were significantly reduced in KO mice...
2017: PloS One
https://www.readbyqxmd.com/read/28714989/correction-of-a-splicing-defect-in-a-mouse-model-of-congenital-muscular-dystrophy-type-1a-using-a-homology-directed-repair-independent-mechanism
#11
Dwi U Kemaladewi, Eleonora Maino, Elzbieta Hyatt, Huayun Hou, Maylynn Ding, Kara M Place, Xinyi Zhu, Prabhpreet Bassi, Zahra Baghestani, Amit G Deshwar, Daniele Merico, Hui Y Xiong, Brendan J Frey, Michael D Wilson, Evgueni A Ivakine, Ronald D Cohn
Splice-site defects account for about 10% of pathogenic mutations that cause Mendelian diseases. Prevalence is higher in neuromuscular disorders (NMDs), owing to the unusually large size and multi-exonic nature of genes encoding muscle structural proteins. Therapeutic genome editing to correct disease-causing splice-site mutations has been accomplished only through the homology-directed repair pathway, which is extremely inefficient in postmitotic tissues such as skeletal muscle. Here we describe a strategy using nonhomologous end-joining (NHEJ) to correct a pathogenic splice-site mutation...
July 17, 2017: Nature Medicine
https://www.readbyqxmd.com/read/28713896/an-optimized-gene-transfection-system-in-weri-rb1-cells
#12
Ying Liu, Zhigang Fan, Kang Li, Fei Deng, Yunfan Xiong, Meixin Liang, Jian Ge
The pathogenesis of Rb1 gene inactivation indicates that gene therapy could be a promising treatment for retinoblastoma. An appropriate gene transfer system is the basis for successful gene therapy; however, little attention has been given to an effective gene transfer system for retinoblastoma therapy in previous studies. This study was designed to provide an optimized transgene system for WERI‑Rb1 cells (W-RBCs). Green fluorescent protein (GFP) was adopted as a reporter. Four classic viral vectors based on retroviruses, recombinant adeno-associated viruses (rAAV2, rAAV2/1), lentiviruses (LVs) and a novel non-viral vector X-treme HP reagent were adopted for W-RBC gene transfection...
July 6, 2017: International Journal of Molecular Medicine
https://www.readbyqxmd.com/read/28710345/deletion-of-the-b-b-and-c-c-regions-of-inverted-terminal-repeats-reduces-raav-productivity-but-increases-transgene-expression
#13
Qingzhang Zhou, Wenhong Tian, Chunguo Liu, Zhonghui Lian, Xiaoyan Dong, Xiaobing Wu
Inverted terminal repeats (ITRs) of the adeno-associated virus (AAV) are essential for rescue, replication, packaging, and integration of the viral genome. While ITR mutations have been identified in previous reports, we designed a new truncated ITR lacking the B-B' and C-C' regions named as ITRΔBC and investigated its effects on viral genome replication, packaging, and expression of recombinant AAV (rAAV). The packaging ability was compared between ITRΔBC rAAV and wild-type (wt) ITR rAAV. Our results showed the productivity of ITRΔBC rAAV was reduced 4-fold, which is consistent with the 8-fold decrease in the replication of viral genomic DNA of ITRΔBC rAAV compared with wt ITR rAAV...
July 14, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28708260/effect-of-optogenetic-manipulation-of-accumbal-medium-spiny-neurons-expressing-dopamine-d2-receptors-in-cocaine-induced-behavioral-sensitization
#14
Byeong Jun Kang, Shelly Sooyun Song, Lei Wen, Ki-Pyo Hong, George J Augustine, Ja-Hyun Baik
Repetitive exposure to addictive drugs causes synaptic modification in the mesocorticolimbic dopamine (DA) system. Dopamine D1 receptors (D1R) or D2 receptors (D2R) expressed in the medium spiny neurons (MSNs) of the nucleus accumbens (NAc) play critical roles in the control of addictive behaviors. Optogenetic activation of D2R-expressing MSNs (D2R-MSNs) in the NAc previously demonstrated that these neurons play a key role in withdrawal-induced plasticity. Here, we examined the effect of optogenetic inhibition of D2R-MSNs in the NAc on cocaine-induced behavioral sensitization...
July 14, 2017: European Journal of Neuroscience
https://www.readbyqxmd.com/read/28707952/systemic-delivery-of-dysferlin-overlap-vectors-provides-long-term-functional-improvement-for-dysferlinopathy
#15
Rachael A Potter, Danielle A Griffin, Patricia C Sondergaard, Ryan W Johnson, Eric R Pozsgai, Kristin N Heller, Ellyn L Peterson, Kimmo K Lehtimaki, Hillarie P Windish, Plavi J Mittal, Doug E Albrecht, Jerry R Mendell, Louise R Rodino-Klapac
Dysferlinopathies comprise a family of disorders caused by mutations in the dysferlin (DYSF) gene leading to a progressive dystrophy characterized by chronic muscle fiber loss, fat replacement and fibrosis. To correct the underlying histopathology and function, expression of full-length DYSF is required. We have developed dual adeno-associated virus vectors defined by a region of homology to serve as a substrate for reconstitution of the full 6.5 kb dysferlin cDNA. Our previous work studied the efficacy of this treatment through intramuscular and regional delivery routes...
July 14, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28706756/impact-of-vital-dyes-on-cell-viability-and-transduction-efficiency-of-aav-vectors-used-in-retinal-gene-therapy-surgery-an-in-vitro-and-in-vivo-analysis
#16
Anna P Salvetti, Maria I Patrício, Alun R Barnard, Harry O Orlans, Doron G Hickey, Robert E MacLaren
PURPOSE: Treatment of inherited retinal degenerations using adeno-associated viral (AAV) vectors involves delivery by subretinal injection. In the latter stages, alteration of normal anatomy may cause difficulty in visualizing the retinotomy, retinal detachment extension, and vector diffusion. Vital dyes may be useful surgical adjuncts, but their safety and impact on AAV transduction are largely unknown. METHODS: The effects of Sodium Fluorescein (SF), Membrane Blue (MB), and Membrane Blue Dual (DB) at a range of dilutions were assessed on human embryonic kidney cells in vitro using an AAV2-green fluorescent protein (GFP) reporter at different multiplicities of infection...
July 2017: Translational Vision Science & Technology
https://www.readbyqxmd.com/read/28704696/the-function-of-dna-binding-protein-nucleophosmin-in-aav-replication
#17
Stifani Satkunanathan, Robin Thorpe, Yuan Zhao
Adeno-associated viruses (AAV) contain minimal viral proteins necessary for their replication. During virus assembly, AAV acquire, inherently and submissively, various cellular proteins. Our previous studies identified the association of AAV vectors with the DNA binding protein nucleophosmin (NPM1). Nucleophosmin has been reported to enhance AAV infection by mobilizing AAV capsids into and out of the nucleolus, indicating the importance of NPM1 in the AAV life cycle; however the role of NPM1 in AAV production remains unknown...
July 10, 2017: Virology
https://www.readbyqxmd.com/read/28702474/targeting-visceral-fat-by-intraperitoneal-delivery-of-novel-aav-serotype-vector-restricting-off-target-transduction-in-liver
#18
Wei Huang, Xianglan Liu, Nicholas J Queen, Lei Cao
It is challenging to genetically manipulate fat in adults. We demonstrate that intraperitoneal (i.p.) injection of an engineered adeno-associated virus (AAV) serotype Rec2 leads to high transduction of multiple visceral fat depots at a dose of 1 to 2 orders lower than commonly used doses for systemic gene delivery. To target adipose tissue, we develop a single AAV vector harboring two expression cassettes: one using the CBA promoter to drive transgene expression and one using the liver-specific albumin promoter to drive a microRNA-targeting WPRE sequence that only exists in this AAV vector...
September 15, 2017: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/28702323/aav-mediated-pancreatic-overexpression-of-igf1-counteracts-progression-to-autoimmune-diabetes-in-mice
#19
Cristina Mallol, Estefania Casana, Veronica Jimenez, Alba Casellas, Virginia Haurigot, Claudia Jambrina, Victor Sacristan, Meritxell Morró, Judith Agudo, Laia Vilà, Fatima Bosch
OBJECTIVE: Type 1 diabetes is characterized by autoimmune destruction of β-cells leading to severe insulin deficiency. Although many improvements have been made in recent years, exogenous insulin therapy is still imperfect; new therapeutic approaches, focusing on preserving/expanding β-cell mass and/or blocking the autoimmune process that destroys islets, should be developed. The main objective of this work was to test in non-obese diabetic (NOD) mice, which spontaneously develop autoimmune diabetes, the effects of local expression of Insulin-like growth factor 1 (IGF1), a potent mitogenic and pro-survival factor for β-cells with immunomodulatory properties...
July 2017: Molecular Metabolism
https://www.readbyqxmd.com/read/28700093/interleukin-10-release-from-astrocytes-suppresses-neuronal-apoptosis-via-the-tlr2-nf%C3%AE%C2%BAb-pathway-in-a-neonatal-rat-model-of-hypoxic-ischemic-brain-damage
#20
Mu Lan He, Ze Yu Lv, Xia Shi, Ting Yang, Yun Zhang, Ting-Yu Li, Jie Chen
The biological function of Interleukin-10 (IL-10) and the relationship between IL-10 secretion and the Toll-like receptor 2 (TLR2) expression levels in the central nervous system following hypoxic-ischemic brain damage (HIBD) are poorly understood. Here, we intend to elucidate the biological function and mechanism of IL-10 secretion following HIBD. In the current study, we used a neonatal rat model of HIBD and found that rats injected with adeno-associated virus (AAV)-IL-10-shRNA (short hairpin RNA) exhibited partially impaired learning and memory function compared to rats administered AAV-control-shRNA...
July 12, 2017: Journal of Neurochemistry
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