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Neuroprotective therapies in glaucoma: II. Genetic nanotechnology tools

Identifieur interne : 001D61 ( Ncbi/Merge ); précédent : 001D60; suivant : 001D62

Neuroprotective therapies in glaucoma: II. Genetic nanotechnology tools

Auteurs : Nafiseh Nafissi ; Marianna Foldvari

Source :

RBID : PMC:4604245

Abstract

Neurotrophic factor genome engineering could have many potential applications not only in the deeper understanding of neurodegenerative disorders but also in improved therapeutics. The fields of nanomedicine, regenerative medicine, and gene/cell-based therapy have been revolutionized by the development of safer and efficient non-viral technologies for gene delivery and genome editing with modern techniques for insertion of the neurotrophic factors into clinically relevant cells for a more sustained pharmaceutical effect. It has been suggested that the long-term expression of neurotrophic factors is the ultimate approach to prevent and/or treat neurodegenerative disorders such as glaucoma in patients who do not respond to available treatments or are at the progressive stage of the disease. Recent preclinical research suggests that novel neuroprotective gene and cell therapeutics could be promising approaches for both non-invasive neuroprotection and regenerative functions in the eye. Several progenitor and retinal cell types have been investigated as potential candidates for glaucoma neurotrophin therapy either as targets for gene therapy, options for cell replacement therapy, or as vehicles for gene delivery. Therefore, in parallel with deeper understanding of the specific protective effects of different neurotrophic factors and the potential therapeutic cell candidates for glaucoma neuroprotection, the development of non-invasive and highly specific gene delivery methods with safe and effective technologies to modify cell candidates for life-long neuroprotection in the eye is essential before investing in this field.


Url:
DOI: 10.3389/fnins.2015.00355
PubMed: 26528114
PubMed Central: 4604245

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PMC:4604245

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<p>Neurotrophic factor genome engineering could have many potential applications not only in the deeper understanding of neurodegenerative disorders but also in improved therapeutics. The fields of nanomedicine, regenerative medicine, and gene/cell-based therapy have been revolutionized by the development of safer and efficient non-viral technologies for gene delivery and genome editing with modern techniques for insertion of the neurotrophic factors into clinically relevant cells for a more sustained pharmaceutical effect. It has been suggested that the long-term expression of neurotrophic factors is the ultimate approach to prevent and/or treat neurodegenerative disorders such as glaucoma in patients who do not respond to available treatments or are at the progressive stage of the disease. Recent preclinical research suggests that novel neuroprotective gene and cell therapeutics could be promising approaches for both non-invasive neuroprotection and regenerative functions in the eye. Several progenitor and retinal cell types have been investigated as potential candidates for glaucoma neurotrophin therapy either as targets for gene therapy, options for cell replacement therapy, or as vehicles for gene delivery. Therefore, in parallel with deeper understanding of the specific protective effects of different neurotrophic factors and the potential therapeutic cell candidates for glaucoma neuroprotection, the development of non-invasive and highly specific gene delivery methods with safe and effective technologies to modify cell candidates for life-long neuroprotection in the eye is essential before investing in this field.</p>
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<author>
<name sortKey="Rad, R" uniqKey="Rad R">R. Rad</name>
</author>
<author>
<name sortKey="Takeda, J" uniqKey="Takeda J">J. Takeda</name>
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<author>
<name sortKey="Bradley, A" uniqKey="Bradley A">A. Bradley</name>
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<name sortKey="Zhu, W" uniqKey="Zhu W">W. Zhu</name>
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<name sortKey="Mao, Y" uniqKey="Mao Y">Y. Mao</name>
</author>
<author>
<name sortKey="Zhou, L F" uniqKey="Zhou L">L. F. Zhou</name>
</author>
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<biblStruct>
<analytic>
<author>
<name sortKey="Zou, J" uniqKey="Zou J">J. Zou</name>
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<author>
<name sortKey="Mali, P" uniqKey="Mali P">P. Mali</name>
</author>
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<name sortKey="Huang, X" uniqKey="Huang X">X. Huang</name>
</author>
<author>
<name sortKey="Dowey, S N" uniqKey="Dowey S">S. N. Dowey</name>
</author>
<author>
<name sortKey="Cheng, L" uniqKey="Cheng L">L. Cheng</name>
</author>
</analytic>
</biblStruct>
</listBibl>
</div1>
</back>
</TEI>
<pmc article-type="review-article">
<pmc-dir>properties open_access</pmc-dir>
<front>
<journal-meta>
<journal-id journal-id-type="nlm-ta">Front Neurosci</journal-id>
<journal-id journal-id-type="iso-abbrev">Front Neurosci</journal-id>
<journal-id journal-id-type="publisher-id">Front. Neurosci.</journal-id>
<journal-title-group>
<journal-title>Frontiers in Neuroscience</journal-title>
</journal-title-group>
<issn pub-type="ppub">1662-4548</issn>
<issn pub-type="epub">1662-453X</issn>
<publisher>
<publisher-name>Frontiers Media S.A.</publisher-name>
</publisher>
</journal-meta>
<article-meta>
<article-id pub-id-type="pmid">26528114</article-id>
<article-id pub-id-type="pmc">4604245</article-id>
<article-id pub-id-type="doi">10.3389/fnins.2015.00355</article-id>
<article-categories>
<subj-group subj-group-type="heading">
<subject>Neuroscience</subject>
<subj-group>
<subject>Review</subject>
</subj-group>
</subj-group>
</article-categories>
<title-group>
<article-title>Neuroprotective therapies in glaucoma: II. Genetic nanotechnology tools</article-title>
</title-group>
<contrib-group>
<contrib contrib-type="author">
<name>
<surname>Nafissi</surname>
<given-names>Nafiseh</given-names>
</name>
<uri xlink:type="simple" xlink:href="http://loop.frontiersin.org/people/210314/overview"></uri>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Foldvari</surname>
<given-names>Marianna</given-names>
</name>
<xref ref-type="author-notes" rid="fn001">
<sup>*</sup>
</xref>
<uri xlink:type="simple" xlink:href="http://loop.frontiersin.org/people/123919/overview"></uri>
</contrib>
</contrib-group>
<aff>
<institution>School of Pharmacy and Waterloo Institute of Nanotechnology, University of Waterloo</institution>
<country>Waterloo, ON, Canada</country>
</aff>
<author-notes>
<fn fn-type="edited-by">
<p>Edited by: Ioan Opris, Wake Forest University School of Medicine, USA</p>
</fn>
<fn fn-type="edited-by">
<p>Reviewed by: Hun-Kuk Park, Kyung Hee University, South Korea; Hari S. Sharma, Uppsala University, Sweden</p>
</fn>
<corresp id="fn001">*Correspondence: Marianna Foldvari, School of Pharmacy and Waterloo Institute of Nanotechnology, University of Waterloo, 200 University Avenue West, Waterloo, ON N2L 3G1, Canada
<email xlink:type="simple">foldvari@uwaterloo.ca</email>
</corresp>
<fn fn-type="other" id="fn002">
<p>This article was submitted to Neural Technology, a section of the journal Frontiers in Neuroscience</p>
</fn>
</author-notes>
<pub-date pub-type="epub">
<day>14</day>
<month>10</month>
<year>2015</year>
</pub-date>
<pub-date pub-type="collection">
<year>2015</year>
</pub-date>
<volume>9</volume>
<elocation-id>355</elocation-id>
<history>
<date date-type="received">
<day>29</day>
<month>6</month>
<year>2015</year>
</date>
<date date-type="accepted">
<day>17</day>
<month>9</month>
<year>2015</year>
</date>
</history>
<permissions>
<copyright-statement>Copyright © 2015 Nafissi and Foldvari.</copyright-statement>
<copyright-year>2015</copyright-year>
<copyright-holder>Nafissi and Foldvari</copyright-holder>
<license xlink:href="http://creativecommons.org/licenses/by/4.0/">
<license-p>This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.</license-p>
</license>
</permissions>
<abstract>
<p>Neurotrophic factor genome engineering could have many potential applications not only in the deeper understanding of neurodegenerative disorders but also in improved therapeutics. The fields of nanomedicine, regenerative medicine, and gene/cell-based therapy have been revolutionized by the development of safer and efficient non-viral technologies for gene delivery and genome editing with modern techniques for insertion of the neurotrophic factors into clinically relevant cells for a more sustained pharmaceutical effect. It has been suggested that the long-term expression of neurotrophic factors is the ultimate approach to prevent and/or treat neurodegenerative disorders such as glaucoma in patients who do not respond to available treatments or are at the progressive stage of the disease. Recent preclinical research suggests that novel neuroprotective gene and cell therapeutics could be promising approaches for both non-invasive neuroprotection and regenerative functions in the eye. Several progenitor and retinal cell types have been investigated as potential candidates for glaucoma neurotrophin therapy either as targets for gene therapy, options for cell replacement therapy, or as vehicles for gene delivery. Therefore, in parallel with deeper understanding of the specific protective effects of different neurotrophic factors and the potential therapeutic cell candidates for glaucoma neuroprotection, the development of non-invasive and highly specific gene delivery methods with safe and effective technologies to modify cell candidates for life-long neuroprotection in the eye is essential before investing in this field.</p>
</abstract>
<kwd-group>
<kwd>neurodegenerative disease</kwd>
<kwd>glaucoma</kwd>
<kwd>neurotrophic factor therapy</kwd>
<kwd>gene and cell therapy</kwd>
<kwd>nanotechnology</kwd>
<kwd>minicircle DNA vectors</kwd>
<kwd>transposon systems</kwd>
<kwd>ZFN- TALEN-CRISPR/Cas</kwd>
</kwd-group>
<counts>
<fig-count count="5"></fig-count>
<table-count count="2"></table-count>
<equation-count count="0"></equation-count>
<ref-count count="127"></ref-count>
<page-count count="16"></page-count>
<word-count count="13118"></word-count>
</counts>
</article-meta>
</front>
</pmc>
<affiliations>
<list></list>
<tree>
<noCountry>
<name sortKey="Foldvari, Marianna" sort="Foldvari, Marianna" uniqKey="Foldvari M" first="Marianna" last="Foldvari">Marianna Foldvari</name>
<name sortKey="Nafissi, Nafiseh" sort="Nafissi, Nafiseh" uniqKey="Nafissi N" first="Nafiseh" last="Nafissi">Nafiseh Nafissi</name>
</noCountry>
</tree>
</affiliations>
</record>

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