دورية أكاديمية
أعرض في EDS

Therapeutic potential of combined viral transduction and CRISPR/Cas9 gene editing in treating neurodegenerative diseases.

التفاصيل البيبلوغرافية
العنوان: Therapeutic potential of combined viral transduction and CRISPR/Cas9 gene editing in treating neurodegenerative diseases.
المؤلفون: Kuruvilla J; Division of Applied Biomedical Sciences and Biotechnology, School of Health Sciences, International Medical University, 126 Jalan Jalil Perkasa 19, Bukit Jalil, 57000, Kuala Lumpur, Malaysia., Sasmita AO; Division of Applied Biomedical Sciences and Biotechnology, School of Health Sciences, International Medical University, 126 Jalan Jalil Perkasa 19, Bukit Jalil, 57000, Kuala Lumpur, Malaysia., Ling APK; Division of Applied Biomedical Sciences and Biotechnology, School of Health Sciences, International Medical University, 126 Jalan Jalil Perkasa 19, Bukit Jalil, 57000, Kuala Lumpur, Malaysia. anna_ling@imu.edu.my.
المصدر: Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology [Neurol Sci] 2018 Nov; Vol. 39 (11), pp. 1827-1835. Date of Electronic Publication: 2018 Aug 03.
نوع المنشور: Journal Article; Review
اللغة: English
بيانات الدورية: Publisher: Springer-Verlag Italia Country of Publication: Italy NLM ID: 100959175 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1590-3478 (Electronic) Linking ISSN: 15901874 NLM ISO Abbreviation: Neurol Sci Subsets: MEDLINE
أسماء مطبوعة: Original Publication: Milano, Italy : Springer-Verlag Italia, c2000-
مواضيع طبية MeSH: CRISPR-Cas Systems/*physiology , Gene Editing/*methods , Genetic Therapy/*methods , Neurodegenerative Diseases/*genetics , Neurodegenerative Diseases/*therapy, Animals ; Genetic Vectors ; Humans
مستخلص: Background and Purpose: The central nervous system (CNS) faces unique difficulties in attaining permanent therapy for neurodegenerative disorder (ND). Genomic level forms of therapy have garnered interest in the recent decade, with the novel CRISPR/Cas9 gene editing tool continuing to be explored due to its efficiency, safety, and adaptability to varying conditions. With the aid of viral vectors as transport vectors, the gene editing tool has produced promising in vitro and in vivo findings in study models. Thus, this review focuses on the recent advancements and update of CRISPR/Cas9 to combat neurodegenerative diseases.
Methods: Articles detailing potential applications of CRISPR/Cas9 in neurodegenerative settings were retrieved from PubMed and Google Scholar with the keywords "CRISPR," "gene editing," and "neurodegenerative diseases." Relevant information was collected and critically reviewed.
Results: The utility of CRISPR/Cas9 coupled with viral transduction ranges from the disruption of amyloid precursor protein (APP) production at a genomic level in Alzheimer's disease (AD) to the deletion of varying exon portions of the Dmd gene in Duchenne muscular dystrophy (DMD) which would increase dystrophin expression. This usage of CRISPR/Cas9 also extends to experimentally ameliorate the neurodegenerative effects caused by viral infections.
Conclusion: The CRISPR/Cas9 gene editing tool is a powerful arsenal in the field of gene therapy and molecular medicine; hence, more research should be called to focus on the ample potential this tool has to offer in the field of neurodegenerative diseases.
References: Hum Mol Genet. 2011 Dec 1;20(23):4530-9. (PMID: 21900357)
Exp Neurol. 2000 Jul;164(1):2-14. (PMID: 10877910)
Mol Neurobiol. 2018 Jan;55(1):682-695. (PMID: 27995572)
Science. 2016 Jan 22;351(6271):403-7. (PMID: 26721684)
Mol Ther. 2009 Aug;17(8):1316-32. (PMID: 19491821)
Stem Cell Res. 2016 Sep;17(2):285-288. (PMID: 27879212)
J Neuropsychiatry Clin Neurosci. 2005 Summer;17(3):310-23. (PMID: 16179652)
Mol Ther. 2015 Mar;23(3):477-87. (PMID: 25358252)
Int J Neurosci. 2018 May 4;:1-17. (PMID: 29667473)
J Clin Oncol. 2003 Sep 1;21(17):3255-61. (PMID: 12947060)
Eur Radiol. 2017 Oct;27(10):4218-4236. (PMID: 28293774)
Lancet. 1996 Sep 21;348(9030):795-9. (PMID: 8813989)
Hum Gene Ther. 2013 Feb;24(2):132-42. (PMID: 23311447)
Mov Disord. 2014 Sep 15;29(11):1455-61. (PMID: 25164989)
Lancet Neurol. 2010 Dec;9(12):1164-1172. (PMID: 20970382)
JAMA Neurol. 2016 Nov 1;73(11):1349-1355. (PMID: 27668807)
Sci Adv. 2017 Dec 20;3(12):eaar3952. (PMID: 29279867)
Neuroscientist. 2015 Feb;21(1):84-98. (PMID: 24557878)
Drug Des Devel Ther. 2015 Jan 07;9:305-12. (PMID: 25609916)
Nat Med. 2014 May;20(5):542-7. (PMID: 24705334)
Neuropathology. 2015 Oct;35(5):487-96. (PMID: 25946231)
Curr Gene Ther. 2017;17(4):309-319. (PMID: 29173170)
Nat Commun. 2015 Feb 18;6:6244. (PMID: 25692716)
Neuron. 2012 Jun 21;74(6):1031-44. (PMID: 22726834)
Nat Biotechnol. 2015 May;33(5):538-42. (PMID: 25798939)
Proc Natl Acad Sci U S A. 2014 Aug 5;111(31):11461-6. (PMID: 25049410)
Hum Gene Ther Clin Dev. 2014 Sep;25(3):164-77. (PMID: 25144894)
Hum Gene Ther. 2005 Jun;16(6):741-51. (PMID: 15960605)
Cell. 2013 Dec 5;155(6):1351-64. (PMID: 24290359)
Nat Biotechnol. 2015 Jan;33(1):102-6. (PMID: 25326897)
Nat Med. 2004 Jul;10 Suppl:S10-7. (PMID: 15272267)
Clin Sci (Lond). 2006 Apr;110(4):393-407. (PMID: 16526945)
J Clin Invest. 2017 Jun 30;127(7):2719-2724. (PMID: 28628038)
Gene Ther. 2014 Mar;21(3):233-41. (PMID: 24401836)
Sci Rep. 2016 Nov 14;6:36921. (PMID: 27841295)
Nature. 2013 Aug 22;500(7463):472-476. (PMID: 23877069)
Cell Stem Cell. 2016 Jun 2;18(6):817-26. (PMID: 27133795)
Mol Cell Neurosci. 2016 Dec;77:76-86. (PMID: 27989734)
Mol Ther. 2017 Jan 4;25(1):12-23. (PMID: 28129107)
Korean J Intern Med. 2017 Jan;32(1):42-61. (PMID: 28049282)
Mol Ther. 2014 Jul;22(7):1299-1309. (PMID: 24781136)
Adv Biomed Res. 2012;1:27. (PMID: 23210086)
Mol Ther Nucleic Acids. 2018 Jun 1;11:429-440. (PMID: 29858078)
Nature. 2008 Jun 12;453(7197):921-4. (PMID: 18488016)
Curr Opin Pharmacol. 2011 Oct;11(5):540-8. (PMID: 21737346)
PLoS Pathog. 2016 Jun 30;12(6):e1005701. (PMID: 27362483)
Sci Rep. 2013;3:2510. (PMID: 23974631)
Nucleic Acids Res. 2016 May 19;44(9):e85. (PMID: 26850641)
Curr Neuropharmacol. 2010 Sep;8(3):305-15. (PMID: 21358979)
Mol Ther Nucleic Acids. 2015 May 12;4:e241. (PMID: 25965551)
Sci Rep. 2017 Jan 09;7:40336. (PMID: 28067312)
Biol Proced Online. 2016 Jul 11;18:14. (PMID: 27403084)
Science. 2016 Jan 22;351(6271):400-3. (PMID: 26721683)
Nat Commun. 2015 Mar 10;6:6413. (PMID: 25752527)
Cell. 2014 Jun 5;157(6):1262-78. (PMID: 24906146)
Mol Ther. 2016 Mar;24(3):447-57. (PMID: 26336974)
Mol Ther Nucleic Acids. 2016 Jul 19;5(7):e338. (PMID: 27434683)
Br J Pharmacol. 2009 May;157(2):153-65. (PMID: 18776913)
Mol Ther. 2011 Mar;19(3):526-35. (PMID: 21179009)
Science. 2013 Feb 15;339(6121):819-23. (PMID: 23287718)
Mol Ther. 2012 Apr;20(4):699-708. (PMID: 22273577)
Mol Brain. 2015 Feb 24;8:12. (PMID: 25887710)
Gene Ther. 2002 Jun;9(11):670-3. (PMID: 12032684)
Acta Neuropathol Commun. 2017 Oct 27;5(1):77. (PMID: 29078805)
N Engl J Med. 2017 Nov 2;377(18):1713-1722. (PMID: 29091557)
Nat Commun. 2017 Feb 14;8:14454. (PMID: 28195574)
Neuron. 2013 Oct 30;80(3):624-32. (PMID: 24183015)
Nat Med. 2001 Jan;7(1):33-40. (PMID: 11135613)
Cell. 2014 Oct 9;159(2):440-55. (PMID: 25263330)
Science. 2016 Jan 22;351(6271):407-411. (PMID: 26721686)
PLoS One. 2015 Sep 11;10(9):e0136046. (PMID: 26360417)
Hum Genomics. 2011 Oct;5(6):623-90. (PMID: 22155606)
فهرسة مساهمة: Keywords: CRISPR/Cas9; Demyelination; Gene editing; Molecular neuroscience; Neurodegeneration
تواريخ الأحداث: Date Created: 20180805 Date Completed: 20181116 Latest Revision: 20181116
رمز التحديث: 20221213
DOI: 10.1007/s10072-018-3521-0
PMID: 30076486
قاعدة البيانات: MEDLINE
الوصف
تدمد:1590-3478
DOI:10.1007/s10072-018-3521-0