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Material Type: Article
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The clinical landscape for AAV gene therapiesNature reviews. Drug discovery, 2021-03, Vol.20 (3), p.173-174 [Peer Reviewed Journal]COPYRIGHT 2021 Nature Publishing Group ;Nature 2021. ;ISSN: 1474-1776 ;EISSN: 1474-1784 ;DOI: 10.1038/d41573-021-00017-7 ;PMID: 33495615Full text available |
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2 |
Material Type: Article
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CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV VectorsCell, 2020-04, Vol.181 (1), p.136-150 [Peer Reviewed Journal]2020 Elsevier Inc. ;Copyright © 2020 Elsevier Inc. All rights reserved. ;ISSN: 0092-8674 ;EISSN: 1097-4172 ;DOI: 10.1016/j.cell.2020.03.023 ;PMID: 32243786Full text available |
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3 |
Material Type: Article
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Noncoding RNA therapeutics - challenges and potential solutionsNature reviews. Drug discovery, 2021-08, Vol.20 (8), p.629-651 [Peer Reviewed Journal]2021. Springer Nature Limited. ;COPYRIGHT 2021 Nature Publishing Group ;Springer Nature Limited 2021. ;Springer Nature Limited 2021 ;ISSN: 1474-1776 ;EISSN: 1474-1784 ;DOI: 10.1038/s41573-021-00219-z ;PMID: 34145432Full text available |
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4 |
Material Type: Article
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Therapeutic in vivo delivery of gene editing agentsCell, 2022-07, Vol.185 (15), p.2806-2827 [Peer Reviewed Journal]2022 ;Copyright © 2022. Published by Elsevier Inc. ;ISSN: 0092-8674 ;EISSN: 1097-4172 ;DOI: 10.1016/j.cell.2022.03.045 ;PMID: 35798006Full text available |
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5 |
Material Type: Article
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Adeno-associated virus vector as a platform for gene therapy deliveryNature reviews. Drug discovery, 2019-05, Vol.18 (5), p.358-378 [Peer Reviewed Journal]2019© Springer Nature Limited 2019 ;ISSN: 1474-1776 ;EISSN: 1474-1784 ;DOI: 10.1038/s41573-019-0012-9 ;PMID: 30710128Full text available |
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6 |
Material Type: Article
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MicroRNA therapeutics: towards a new era for the management of cancer and other diseasesNature reviews. Drug discovery, 2017-03, Vol.16 (3), p.203-222 [Peer Reviewed Journal]COPYRIGHT 2017 Nature Publishing Group ;Copyright Nature Publishing Group Mar 2017 ;ISSN: 1474-1776 ;EISSN: 1474-1784 ;DOI: 10.1038/nrd.2016.246 ;PMID: 28209991Full text available |
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7 |
Material Type: Article
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European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophyEuropean journal of paediatric neurology, 2020-09, Vol.28, p.38 [Peer Reviewed Journal]ISSN: 1532-2130 ;EISSN: 1532-2130 ;DOI: 10.1016/j.ejpn.2020.07.001Digital Resources/Online E-Resources |
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8 |
Material Type: Article
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TNF biology, pathogenic mechanisms and emerging therapeutic strategiesNature reviews. Rheumatology, 2016-01, Vol.12 (1), p.49-62 [Peer Reviewed Journal]Copyright Nature Publishing Group Jan 2016 ;ISSN: 1759-4790 ;EISSN: 1759-4804 ;DOI: 10.1038/nrrheum.2015.169 ;PMID: 26656660Full text available |
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9 |
Material Type: Article
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Current Clinical Applications of In Vivo Gene Therapy with AAVsMolecular therapy, 2021-02, Vol.29 (2), p.464-488 [Peer Reviewed Journal]2020 The Authors ;Copyright © 2020 The Authors. Published by Elsevier Inc. All rights reserved. ;2020 The Authors 2020 ;ISSN: 1525-0016 ;EISSN: 1525-0024 ;DOI: 10.1016/j.ymthe.2020.12.007 ;PMID: 33309881Full text available |
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10 |
Material Type: Article
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Delivery technologies for genome editingNature reviews. Drug discovery, 2017-06, Vol.16 (6), p.387-399 [Peer Reviewed Journal]COPYRIGHT 2017 Nature Publishing Group ;Copyright Nature Publishing Group Jun 2017 ;ISSN: 1474-1776 ;EISSN: 1474-1784 ;DOI: 10.1038/nrd.2016.280 ;PMID: 28337020Full text available |
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11 |
Material Type: Article
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Cellular uptake and trafficking of antisense oligonucleotidesNature biotechnology, 2017-03, Vol.35 (3), p.230-237 [Peer Reviewed Journal]COPYRIGHT 2017 Nature Publishing Group ;COPYRIGHT 2017 Nature Publishing Group ;Copyright Nature Publishing Group Mar 2017 ;ISSN: 1087-0156 ;EISSN: 1546-1696 ;DOI: 10.1038/nbt.3779 ;PMID: 28244996Full text available |
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12 |
Material Type: Article
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Clinical use of lentiviral vectorsLeukemia, 2018-07, Vol.32 (7), p.1529-1541 [Peer Reviewed Journal]COPYRIGHT 2018 Nature Publishing Group ;COPYRIGHT 2018 Nature Publishing Group ;2018. This work is published under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License. ;The Author(s) 2018 ;ISSN: 0887-6924 ;EISSN: 1476-5551 ;DOI: 10.1038/s41375-018-0106-0 ;PMID: 29654266Full text available |
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13 |
Material Type: Article
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Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX VariantThe New England journal of medicine, 2017-12, Vol.377 (23), p.2215-2227 [Peer Reviewed Journal]Copyright © 2017 Massachusetts Medical Society. All rights reserved. ;ISSN: 0028-4793 ;EISSN: 1533-4406 ;DOI: 10.1056/NEJMoa1708538 ;PMID: 29211678Full text available |
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14 |
Material Type: Article
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Non-viral Vectors in Gene Therapy: Recent Development, Challenges, and ProspectsThe AAPS journal, 2021-06, Vol.23 (4), p.78, Article 78 [Peer Reviewed Journal]American Association of Pharmaceutical Scientists 2021 ;ISSN: 1550-7416 ;EISSN: 1550-7416 ;DOI: 10.1208/s12248-021-00608-7 ;PMID: 34076797Full text available |
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15 |
Material Type: Article
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Delivering the Messenger: Advances in Technologies for Therapeutic mRNA DeliveryMolecular therapy, 2019-04, Vol.27 (4), p.710-728 [Peer Reviewed Journal]2019 ;Copyright © 2019. Published by Elsevier Inc. ;Copyright Elsevier Limited Apr 10, 2019 ;2019. 2019 ;ISSN: 1525-0016 ;EISSN: 1525-0024 ;DOI: 10.1016/j.ymthe.2019.02.012 ;PMID: 30846391Full text available |
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16 |
Material Type: Article
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Genome editing with Cas9 in adult mice corrects a disease mutation and phenotypeNature biotechnology, 2014-06, Vol.32 (6), p.551-553 [Peer Reviewed Journal]COPYRIGHT 2014 Nature Publishing Group ;COPYRIGHT 2014 Nature Publishing Group ;Copyright Nature Publishing Group Jun 2014 ;2014 Nature America, Inc. All rights reserved. 2014 ;ISSN: 1087-0156 ;EISSN: 1546-1696 ;DOI: 10.1038/nbt.2884 ;PMID: 24681508Full text available |
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17 |
Material Type: Article
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Genome-editing Technologies for Gene and Cell TherapyMolecular therapy, 2016-03, Vol.24 (3), p.430-446 [Peer Reviewed Journal]2016 Official journal of the American Society of Gene & Cell Therapy ;Copyright Nature Publishing Group Mar 2016 ;Copyright © 2016 Official journal of the American Society of Gene & Cell Therapy 2016 Official journal of the American Society of Gene & Cell Therapy ;ISSN: 1525-0016 ;EISSN: 1525-0024 ;DOI: 10.1038/mt.2016.10 ;PMID: 26755333Full text available |
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18 |
Material Type: Article
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Cystic fibrosis genetics: from molecular understanding to clinical applicationNature reviews. Genetics, 2015-01, Vol.16 (1), p.45-56 [Peer Reviewed Journal]COPYRIGHT 2015 Nature Publishing Group ;COPYRIGHT 2015 Nature Publishing Group ;Copyright Nature Publishing Group Jan 2015 ;2014 Macmillan Publishers Limited. All rights reserved 2014 ;ISSN: 1471-0056 ;EISSN: 1471-0064 ;DOI: 10.1038/nrg3849 ;PMID: 25404111Full text available |
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19 |
Material Type: Article
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Delivering CRISPR: a review of the challenges and approachesDrug delivery, 2018, Vol.25 (1), p.1234-1257 [Peer Reviewed Journal]2018 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group. 2018 ;2018 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group. This work is licensed under the Creative Commons Attribution License http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License. ;2018 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group. 2018 The Author(s) ;ISSN: 1071-7544 ;EISSN: 1521-0464 ;DOI: 10.1080/10717544.2018.1474964 ;PMID: 29801422Full text available |
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20 |
Material Type: Article
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Gene therapy returns to centre stageNature (London), 2015-10, Vol.526 (7573), p.351-360 [Peer Reviewed Journal]COPYRIGHT 2015 Nature Publishing Group ;COPYRIGHT 2015 Nature Publishing Group ;Copyright Nature Publishing Group Oct 15, 2015 ;ISSN: 0028-0836 ;EISSN: 1476-4687 ;DOI: 10.1038/nature15818 ;PMID: 26469046 ;CODEN: NATUASFull text available |