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Relevant bibliographies by topics / Mucopolysaccharidosis Gene therapy / Dissertations / Theses

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Dissertations / Theses on the topic 'Mucopolysaccharidosis Gene therapy'

Author: Grafiati

Published: 4 June 2021

Last updated: 31 January 2023

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1

Lutzko, Carolyn Mary. "Gene therapy for canine mucopolysaccharidosis type I." Thesis, National Library of Canada = Bibliothèque nationale du Canada, 1999. http://www.collectionscanada.ca/obj/s4/f2/dsk1/tape7/PQDD_0006/NQ41221.pdf.

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2

Langford-Smith, Alexander William Walker. "Lentiviral vector mediated haematopoietic stem cell gene therapy for mucopolysaccharidosis type IIIA." Thesis, University of Manchester, 2012. https://www.research.manchester.ac.uk/portal/en/theses/lentiviral-vector-mediated-haematopoietic-stem-cell-gene-therapy-for-mucopolysaccharidosis-type-iiia(89f8e108-58f3-42bb-8b80-0e0a1fe45fd7).html.

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Mucopolysaccharidosis type III (Sanfilippo) is comprised of four phenotypically similar lysosomal storage disorders (MPS IIIA-D) caused by the deficiency of enzymes that catabolise heparan sulphate (HS). Progressive accumulation of HS results in abnormal behaviour, progressive cognitive and motor impairment and death in mid-teens. There are currently no treatments for MPS III. To assess the effect of novel therapeutics in the mouse models of MPS III it is necessary to examine the effect on primary storage of HS, secondary storage and behaviour. The reported behaviour of MPS IIIA and B mice is

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3

Yogalingam, Gouri. "Molecular characterisation of feline MPS VI and evaluation of gene therapy /." Title page, contents and abstract only, 1998. http://web4.library.adelaide.edu.au/theses/09PH/09phy54.pdf.

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4

Gliddon, Briony Lee. "Enzyme replacement therapy in a murine model of mucopolysaccharidosis type IIIA /." Title page, contents and abstract only, 2002. http://web4.library.adelaide.edu.au/theses/09PH/09phg5595.pdf.

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5

Crawley, Allison Catherine. "Enzyme replacement therapy in a feline model of mucopolysaccharidosis type VI /." Title page, contents and abstract only, 1998. http://web4.library.adelaide.edu.au/theses/09PH/09phc9107.pdf.

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6

Motas, Mallol Sandra. "Gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (hunter syndrome)." Doctoral thesis, Universitat Autònoma de Barcelona, 2016. http://hdl.handle.net/10803/390961.

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La Mucopolisacaridosis tipus II (MPSII), o síndrome de Hunter, és una malaltia d’acumulació lisosòmica d’herència recessiva lligada al cromosoma X i està causada per la deficiència de l’Iduronat-2-sulfatasa (IDS), enzim que actua en la via de degradació dels glicosaminoglicans (GAGs) heparan sulfat (HS) i dermatan sulfat (DS). Aquests GAGs no degradats s’acumulen als lisosomes de manera patològica, causant disfunció cel·lular. La forma més severa i també més prevalent de la MPSII es caracteritza per una neurodegeneració crònica i progressiva del sistema nerviós central (SNC) acompanyada de dis

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7

Sorrentino, Nicolina Cristina. "Systemic AAV-mediated gene therapy approach to treat CNS pathology in Mucopolysaccharidosis type IIIA." Thesis, Open University, 2012. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.594745.

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Mucopolysaccharidosis type IIIA (MPS-IIIA) is a severe neurodegenerative lysosomal storage disorder (LSD) inherited as an autosomal recessive trait and caused by sulfamidase deficiency. Using somatic gene transfer, we demonstrated therapeutic efficacy of a novel low-invasive gene therapy approach to treat the brain pathology in MPS-IIIA. The therapeutic strategy is based on a chimeric sulfamidase engineered with both the signal peptide (sp) from the highly secreted iduronate-2-sulfatase (IDS) linked to its N-terminal end and the blood-brain barrier (BBB)-binding domain of apolipoproteinB (ApoB

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8

Sergijenko, Ana. "Improved lentiviral vectors for haematopoietic stem cell gene therapy of Mucopolysaccaridosis type IIIA." Thesis, University of Manchester, 2012. http://www.manchester.ac.uk/escholar/uk-ac-man-scw:176449.

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Mucopolysaccharidosis type IIIA (MPS IIIA) is caused by mutations in the N-sulphoglucosamine sulphohydrolase (SGSH) gene, leading to cellular accumulation of heparan sulphate and progressive neurodegeneration in patients. One of the proposed treatment methods is haematopoietic stem cell (HSC) gene therapy, which should result in an excess of SGSH produced in the peripheral organs and brain. The pre-clinical feasibility of this approach was demonstrated by our group in a mouse model of MPS IIIA. However, the overall efficiency of this method was limited and a number of approaches to solving the

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9

Schuh, Roselena Silvestri. "Desenvolvimento de vetores nanotecnológicos lipídicos do sistema CRISPR/Cas9 visando à terapia gênica para Mucopolissacaridose tipo I." reponame:Biblioteca Digital de Teses e Dissertações da UFRGS, 2017. http://hdl.handle.net/10183/175139.

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A mucopolissacaridose tipo I (MPS I) é causada pela deficiência de alfa-L-iduronidase (IDUA), responsável pelo catabolismo de glicosaminoglicanos (GAGs), levando ao acúmulo multissistêmico de sulfato de heparano e dermatano. Este estudo tem por objetivo avaliar o potencial de sistemas lipídicos nanoestruturados como carreadores do plasmídeo do sistema CRISPR/Cas9 e um vetor doador da sequência do gene IDUA/Idua para edição gênica em fibroblastos de pacientes e em modelo murino de MPS I. Foram produzidos lipossomas (DOTAP, DOPE e DSPE-PEG) e nanoemulsões (e TCM) por homogeneização à alta pressã

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10

Pérez, Castro Jennifer Ana. "Estrategia de terapia génica para el tratamiento de las alteraciones auditivas y visuales de la mucopolisacaridosis tipo IIIB." Doctoral thesis, Universitat Autònoma de Barcelona, 2021. http://hdl.handle.net/10803/671992.

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La Mucopolisacaridosi tipus IIIB (MPSIIIB) és una malaltia minoritària d’acumulació lisosòmica, d’herència autosòmica recessiva, causada per la deficiència de l’enzim lisosòmic α-N-acetilglucosaminidasa (NAGLU), la qual cosa provoca l’acumulació de formes parcialment degradades del glicosaminoglicà (GAG) heparan sulfat (HS) a l’interior dels lisosomes. Aquesta acumulació sostinguda en el temps genera disfunció cel·lular i la posterior mort de les cèl·lules. La MPSIIIB presenta una profunda afectació progressiva del Sistema Nerviós Central (SNC) caracteritzada per neurodegeneració i neuroinflam

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11

Ogez, Brittney Dawn. "Efficacy of AAV2 and AAV8 to cross the blood brain barrier in the MPS IIIA mouse model." Oklahoma City : [s.n.], 2009.

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12

Lau, Adeline Allison. "Gene transfer in murine MPS IIIA using canine adenoviral vectors." 2008. http://digital.library.adelaide.edu.au/dspace/handle/2440/49475.

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Thesis (Ph.D.) -- University of Adelaide, School of Paediatrics and Reproductive Health, Discipline of Paediatrics, 2007.<br>"June 2007" Includes Addenda attached to back page. Bibliography: leaves 215-274. Also available in print form.

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13

Crawley, Allison Catherine. "Enzyme replacement therapy in a feline model of mucopolysaccharidosis type VI / Allison Catherine Crawley." Thesis, 1998. http://hdl.handle.net/2440/19171.

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Bibliography: leaves 269-297.<br>xvii, 297, [10] leaves, [31] leaves of plates : ill. (some col.) ; 30 cm.<br>Evaluates the efficacy of enzyme replacement therapy (ERT) with artifically produced recombinant human 4S (rh4S) in feline mucopolysaccharidosis Type VI (MPS VI) and tests the hypothesis that this form of therapy would reverse or alter the disease course, particularly the bone dysplasia and connective tissue pathologics.<br>Thesis (Ph.D.)--University of Adelaide, Dept. of Paediatrics, 1998

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14

Lau, Adeline Allison. "Gene transfer in murine MPS IIIA using canine adenoviral vectors." 2007. http://hdl.handle.net/2440/49475.

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Mucopolysaccharidosis type IIIA (MPS IIIA) is an autosomal-recessively inherited disorder caused by the deficiency of lysosomal sulphamidase (NS) enzyme activity, resulting in the accumulation of the glycosaminoglycan (GAG) heparan sulphate (HS). MPS IIIA patients experience progressive and severe neurological deterioration with death usually occurring in the mid-late teenage years. A naturally-occurring mouse model of MPS IIIA has been characterised and the biochemical, histological and behavioural changes closely parallel the human condition. In order to treat the neurological effects of MPS

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15

Lau, Adeline Allison. "Gene transfer in murine MPS IIIA using canine adenoviral vectors." Thesis, 2007. http://hdl.handle.net/2440/49475.

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Abstract:

Mucopolysaccharidosis type IIIA (MPS IIIA) is an autosomal-recessively inherited disorder caused by the deficiency of lysosomal sulphamidase (NS) enzyme activity, resulting in the accumulation of the glycosaminoglycan (GAG) heparan sulphate (HS). MPS IIIA patients experience progressive and severe neurological deterioration with death usually occurring in the mid-late teenage years. A naturally-occurring mouse model of MPS IIIA has been characterised and the biochemical, histological and behavioural changes closely parallel the human condition. In order to treat the neurological effects of MPS

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16

Gliddon, Briony Lee. "Enzyme replacement therapy in a murine model of mucopolysaccharidosis type IIIA / by Briony Lee Gliddon." Thesis, 2002. http://hdl.handle.net/2440/21922.

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Addenda page on inside back cover.<br>Bibliography: leaves 153-176.<br>xiii, 176 leaves ; ill. (some col.) ; 30 cm<br>Mucopolysaccharideosis type IIIA (MPS IIIA, Sanfilippo A syndrome) is an autosomal recessive lysosomal storage disease, with a prevalence in Australia of 1 in 114,000. MPS IIIA is caused by a deficiency of the lysosomal enzyme sulphamidase which is needed together with other exohydrolases and a N-acetyltransferase to break down the glycosaminoglycan heparan sulphate to sulphate and monosaccharides. Patients are characterised by severe central nervous systems degeneration togeth

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17

Jackson, Matilda. "Alpha-L-iduronidase transduced mesenchymal stem cells as a therapy for the treatment of CNS degeneration in mucopolysaccharidosis type I mice." Thesis, 2015. http://hdl.handle.net/2440/103497.

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Mucopolysaccharidosis type I (MPS I) is an autosomal recessive disorder that is characterised by a deficiency in the α-L-iduronidase (IDUA) enzyme, resulting in the accumulation of undegraded heparan sulphate and dermatan sulphate glycosaminoglycans (gags) within the lysosome of nearly every cell. MPS I is a multi-tissue and organ disease, presenting with profound mental retardation and skeletal abnormalities. Haematopoietic stem cell (HSC) transplant and enzyme replacement therapy, two clinically available forms of treatment, are able to correct the soft tissue aspects of MPS disease, but hav

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