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"Gene therapy"

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https://www.readbyqxmd.com/read/29332089/gene-therapy-for-parkinson-s-disease
#1
Vivek Sudhakar, R Mark Richardson
Gene therapy is a clinical tool that may eventually provide therapeutic benefit to patients suffering from movement disorders through a few potential mechanisms: direct correction of the pathogenic mechanism, neuroprotection, neurorestoration or symptom control. The therapeutic mechanism is therefore dependent on knowledge of disease pathogenesis and the required temporal and spatial specificities of gene expression. An additional critical challenge is achieving the most complete transduction of the target structure while avoiding leakage into neighboring regions or perivascular spaces...
2018: Progress in Neurological Surgery
https://www.readbyqxmd.com/read/29331172/blood-phenylalanine-reduction-corrects-cns-dopamine-and-serotonin-deficiencies-and-partially-improves-behavioral-performance-in-adult-phenylketonuric-mice
#2
Shelley R Winn, Tanja Scherer, Beat Thöny, Ming Ying, Aurora Martinez, Sydney Weber, Jacob Raber, Cary O Harding
Central nervous system (CNS) deficiencies of the monoamine neurotransmitters dopamine and serotonin have been implicated in the pathophysiology of neuropsychiatric dysfunction in human phenylketonuria (PKU). In this study, we confirmed the occurrence of brain dopamine and serotonin deficiencies in association with severe behavioral alterations and cognitive impairments in hyperphenylalaninemic C57BL/6-Pahenu2/enu2 mice, a model of human PKU. Phenylalanine-reducing treatments, including either dietary phenylalanine restriction or liver-directed gene therapy, initiated during adulthood were associated with increased brain monoamine content along with improvements in nesting behavior but without a change in the severe cognitive deficits exhibited by these mice...
January 2018: Molecular Genetics and Metabolism
https://www.readbyqxmd.com/read/29327920/generation-of-optogenetically-modified-adenovirus-vector-for-spatiotemporally-controllable-gene-therapy
#3
Kazuo Takayama, Hiroyuki Mizuguchi
Gene therapy is expected to be utilized for the treatment of various diseases. However, the spatiotemporal resolution of current gene therapy technology is not high enough. In this study, we generated a new technology for spatiotemporally controllable gene therapy. We introduced optogenetic and CRISPR/Cas9 techniques into a recombinant adenovirus (Ad) vector, which is widely used in clinical trials and exhibits high gene transfer efficiency, to generate an illumination-dependent spatiotemporally controllable gene regulation system (designated the Opt/Cas-Ad system)...
January 12, 2018: ACS Chemical Biology
https://www.readbyqxmd.com/read/29327758/a-computational-analysis-of-pro-angiogenic-therapies-for-peripheral-artery-disease
#4
Lindsay E Clegg, Feilim Mac Gabhann
Inducing therapeutic angiogenesis to effectively form hierarchical, non-leaky networks of perfused vessels in tissue engineering applications and ischemic disease remains an unmet challenge, despite extensive research and multiple clinical trials. Here, we use a previously-developed, multi-scale, computational systems pharmacology model of human peripheral artery disease to screen a diverse array of promising pro-angiogenic strategies, including gene therapy, biomaterials, and antibodies. Our previously-validated model explicitly accounts for VEGF immobilization, Neuropilin-1 binding, and weak activation of VEGF receptor 2 (VEGFR2) by the "VEGFxxxb" isoforms...
January 12, 2018: Integrative Biology: Quantitative Biosciences From Nano to Macro
https://www.readbyqxmd.com/read/29327735/acquired-sensorineural-hearing-loss-in-children-current-research-and-therapeutic-perspectives
#5
M Ralli, R Rolesi, R Anzivino, R Turchetta, A R Fetoni
The knowledge of mechanisms responsible for acquired sensorineural hearing loss in children, such as viral and bacterial infections, noise exposure, aminoglycoside and cisplatin ototoxicity, is increasing and progressively changing the clinical management of affected patients. Viral infections are by far the most relevant cause of acquired hearing loss, followed by aminoglycoside and platinum derivative ototoxicity; moreover, cochlear damage induced by noise overexposure, mainly in adolescents, is an emerging topic...
December 2017: Acta Otorhinolaryngologica Italica
https://www.readbyqxmd.com/read/29326962/emerging-issues-in-aav-mediated-in%C3%A2-vivo-gene-therapy
#6
REVIEW
Pasqualina Colella, Giuseppe Ronzitti, Federico Mingozzi
In recent years, the number of clinical trials in which adeno-associated virus (AAV) vectors have been used for in vivo gene transfer has steadily increased. The excellent safety profile, together with the high efficiency of transduction of a broad range of target tissues, has established AAV vectors as the platform of choice for in vivo gene therapy. Successful application of the AAV technology has also been achieved in the clinic for a variety of conditions, including coagulation disorders, inherited blindness, and neurodegenerative diseases, among others...
March 16, 2018: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/29326851/recent-advancements-in-gene-therapy-for-hereditary-retinal-dystrophies
#7
REVIEW
Ayşe Öner
Hereditary retinal dystrophies (HRDs) are degenerative diseases of the retina which have marked clinical and genetic heterogeneity. Common presentations among these disorders include night or colour blindness, tunnel vision, and subsequent progression to complete blindness. The known causative disease genes have a variety of developmental and functional roles, with mutations in more than 120 genes shown to be responsible for the phenotypes. In addition, mutations within the same gene have been shown to cause different disease phenotypes, even amongst affected individuals within the same family, highlighting further levels of complexity...
December 2017: Turkish Journal of Ophthalmology
https://www.readbyqxmd.com/read/29326244/gene-therapy-comes-of-age
#8
REVIEW
Cynthia E Dunbar, Katherine A High, J Keith Joung, Donald B Kohn, Keiya Ozawa, Michel Sadelain
After almost 30 years of promise tempered by setbacks, gene therapies are rapidly becoming a critical component of the therapeutic armamentarium for a variety of inherited and acquired human diseases. Gene therapies for inherited immune disorders, hemophilia, eye and neurodegenerative disorders, and lymphoid cancers recently progressed to approved drug status in the United States and Europe, or are anticipated to receive approval in the near future. In this Review, we discuss milestones in the development of gene therapies, focusing on direct in vivo administration of viral vectors and adoptive transfer of genetically engineered T cells or hematopoietic stem cells...
January 12, 2018: Science
https://www.readbyqxmd.com/read/29325457/synthetic-adeno-associated-viral-vector-efficiently-targets-mouse-and-non-human-primate-retina-in-vivo
#9
Livia S Carvalho, Ru Xiao, Sarah Wassmer, Aliete Langsdorf, Eric Zinn, Simon Pacouret, Samiksha Shah, Jason I Comander, Leo Kim, Laurence Lim, Luk H Vandenberghe
Gene therapy is a promising approach in the treatment of inherited and common complex disorders of the retina. Preclinical and clinical studies have validated the use of adeno-associated viral vectors (AAV) as a safe and efficient delivery vehicle for gene transfer. RPE and rods, and to a lesser extent, cone photoreceptors can be efficiently targeted with AAV. Other retinal cell types however are more challenging targets. The aim of this study was to characterize the transduction profile and efficiency of in silico designed, synthetic Anc80 AAVs for retinal gene transfer...
January 12, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29325430/rapid-and-sensitive-assessment-of-globin-chains-for-gene-and-cell-therapy-of-haemoglobinopathies
#10
Constantinos Christos Loucari, Petros Patsali, Thamar B van Dijk, Coralea Stephanou, Panayiota Papasavva, Maria Zanti, Ryo Kurita, Yukio Nakamura, Soteroulla Christou, Maria Sitarou, Sjaak Philipsen, Carsten Werner Lederer, Marina Kleanthous
The β-haemoglobinopathies sickle cell anaemia and β-thalassaemia are the focus of many gene-therapy studies. A key disease parameter is the abundance of globin chains, because it indicates the level of anaemia, likely toxicity of excess or aberrant globins, and therapeutic potential of induced or exogenous β-like globins. Reversed-phase high-performance liquid chromatography (HPLC) allows versatile and inexpensive globin quantification, but commonly applied protocols suffer either from long run times, high sample requirements or inability to separate murine from human β-globin chains...
January 12, 2018: Human Gene Therapy Methods
https://www.readbyqxmd.com/read/29323644/vision-quest-gene-therapy-for-inherited-vision-loss
#11
EDITORIAL
(no author information available yet)
No abstract text is available yet for this article.
January 6, 2018: Lancet
https://www.readbyqxmd.com/read/29322065/modeling-anti-hiv-1-hspc-based-gene-therapy-in-humanized-mice-previously-infected-with-hiv-1
#12
Wannisa Khamaikawin, Saki Shimizu, Masakazu Kamata, Ruth Cortado, Yujin Jung, Jennifer Lam, Jing Wen, Patrick Kim, Yiming Xie, Sanggu Kim, Hubert Arokium, Angela P Presson, Irvin S Y Chen, Dong Sung An
Investigations of anti-HIV-1 human hematopoietic stem/progenitor cell (HSPC)-based gene therapy have been performed by HIV-1 challenge after the engraftment of gene-modified HSPCs in humanized mouse models. However, the clinical application of gene therapy is to treat HIV-1-infected patients. Here, we developed a new method to investigate an anti-HIV-1 HSPC-based gene therapy in humanized mice previously infected with HIV-1. First, humanized mice were infected with HIV-1. When plasma viremia reached >107 copies/mL 3 weeks after HIV-1 infection, the mice were myeloablated with busulfan and transplanted with anti-HIV-1 gene-modified CD34+ HSPCs transduced with a lentiviral vector expressing two short hairpin RNAs (shRNAs) against CCR5 and HIV-1 long terminal repeat (LTR), along with human thymus tissue under the kidney capsule...
June 15, 2018: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/29321088/upregulation-of-long-non-coding-rna-tug1-promotes-bladder-cancer-cell-5-proliferation-migration-and-invasion-by-inhibiting-mir-29c
#13
Peng Guo, Guohui Zhang, Jialin Meng, Qian He, Zhihui Li, Yawei Guan
Bladder cancer (BC) is one of the leading causes of cancer-related death in the word. Long non-coding RNA (lncRNA) taurine-upregulated gene 1 (TUG1) plays an important role in the development and progression of numerous cancers, including BC. However, the exact role of TUG1 in modulating BC progression is still poorly known. In this study, we found that TUG1 was upregulated and microRNA-29c (miR-29c) was downregulated in BC tissues and cell lines. Overexpression of TUG1 promoted the cell proliferation of T24 and EJ cells, whereas TUG1 knockdown had the opposite effect...
January 10, 2018: Oncology Research
https://www.readbyqxmd.com/read/29320893/the-current-status-of-non-viral-vectors-for-gene-therapy-in-china
#14
Li Liu, Jingyun Yang, Ke Men, Zhi-Yao He, Min Luo, Zhi-Yong Qian, Xiawei Wei
With the growing interest in the application of non-viral vectors for drug delivery, diagnosis and imaging, progress has been made in the field of non-viral vector gene therapy in China. Studies on nanobiotechnology are important in the development plan of China in 21st century research priorities. The National 973 Plan and "Strategic Priority Research Program" classify nanobiotechnology as a special project and give priority to supporting its development. From 2000 to 2017, many articles on non-viral vector gene therapy were published...
January 10, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29320887/transducing-airway-basal-cells-with-a-helper-dependent-adenoviral-vector-for-lung-gene-therapy
#15
Huibi Cao, Hong Ouyang, Hartmut Grasemann, Claire Bartlett, Kai Du, Rongqi Duan, Fushan Shi, Marvin Estrada, Kyle Seigel, Allan Coates, Herman Yeger, Christine Bear, Tanja Gonska, Theo Moraes, Jim Hu
A major challenge in developing gene-based therapies for airway diseases such as cystic fibrosis (CF) is sustaining therapeutic levels of transgene expression over time. This is largely due to airway epithelial cell turnover and the host immunogenicity to gene delivery vectors. Modern gene editing tools and delivery vehicles hold great potential for overcoming the challenge. There is currently not much known about how to deliver genes into airway stem cells, of which basal cells are the major type in human airways...
January 10, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29319697/fda-approves-hereditary-blindness-gene-therapy
#16
(no author information available yet)
No abstract text is available yet for this article.
January 10, 2018: Nature Biotechnology
https://www.readbyqxmd.com/read/29318258/clinical-advances-in-human-gene-therapies
#17
Jennifer Abbasi
No abstract text is available yet for this article.
January 9, 2018: JAMA: the Journal of the American Medical Association
https://www.readbyqxmd.com/read/29316814/biology-of-the-adrenal-gland-cortex-obviates-effective-use-of-adeno-associated-virus-vectors-to-treat-hereditary-adrenal-disorders
#18
Sandra Markmann, Bishnu P De, Jasmine Reid, Clarisse Jose, Jonathan B Rosenberg, Philip Leopold, Stephen M Kaminsky, Dolan Sondhi, Odelya E Pagovich, Ronald G Crystal
Congenital adrenal hyperplasia (CAH) is an autosomal recessive disorder occurring in 1:10,000-1:20,000 live births. In >95% of the cases, CAH results from mutations in the CYP21A2 gene, encoding the adrenal steroid enzyme 21-hydroxylase (21OH). Cardinal phenotypic features of CAH include genital ambiguity and sexual precocity, and in severe cases, neonatal salt loss and death. Current standard of care consists of life-long oral steroid replacement to reverse the cortisol deficiency. Although significant advances in the treatment of CAH have been made, the burden of a life-long therapeutic intervention is not ideal for quality of life...
January 9, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29316812/aavrh10-gene-therapy-ameliorates-central-and-peripheral-nervous-system-disease-in-canine-globoid-cell-leukodystrophy-krabbe-disease
#19
Allison M Bradbury, Mohammad A Rafi, Jessica Bagel, Becky K Brisson, Michael S Marshall, Jill Pesayco Salvador, Xuntian Jiang, Gary P Swain, Maria L Prociuk, Patricia O'Donnell, Caitlin Fitzgerald, Daniel S Ory, Ernesto R Bongarzone, G Diane Shelton, David A Wenger, Charles Vite
Globoid cell leukodystrophy (GLD), or Krabbe disease, is an inherited, neurologic disorder that results from deficiency of a lysosomal enzyme, galactosylceramidase (GALC). Most commonly, deficits of GALC result in widespread central and peripheral nervous system (CNS, PNS) demyelination and death in affected infants typically by 2 years of age. Hematopoietic stem cell transplantation is the current standard of care in children diagnosed prior to symptom onset; however, disease correction is incomplete. Herein we present the first adeno-associated virus (AAV) gene therapy experiments in a naturally occurring canine model of GLD that closely recapitulates the clinical disease progression, neuropathological alterations, and biochemical abnormalities observed in human patients...
January 9, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29316791/serine-integrases-advancing-synthetic-biology
#20
Christine Merrick, Jia Zhao, Susan Rosser
Serine integrases catalyze precise rearrangement of DNA through site-specific recombination of small sequences of DNA called attachment (att) sites. Unlike other site-specific recombinases, the recombination reaction driven by serine integrases is highly directional and can only be reversed in the presence of an accessory protein called a recombination directionality factor (RDF). The ability to control reaction directionality has led to the development of serine integrases as tools for controlled rearrangement and modification of DNA in synthetic biology, gene therapy, and biotechnology...
January 9, 2018: ACS Synthetic Biology
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