Combination of Genetics and Nanotechnology for Down Syndrome Modification: A Potential Hypothesis and Review of the Literature.
CRISPR/Cas9
Chromosome 21 inactivation
Designed DNA construct
Down syndrome
Nano-carrier
Poly D L-lactide-co-glycolide (PLGA)
Journal
Iranian journal of public health
ISSN: 2251-6085
Titre abrégé: Iran J Public Health
Pays: Iran
ID NLM: 7505531
Informations de publication
Date de publication:
Mar 2019
Mar 2019
Historique:
entrez:
22
6
2019
pubmed:
22
6
2019
medline:
22
6
2019
Statut:
ppublish
Résumé
Down syndrome (DS) is one of the most prevalent genetic disorders in humans. The use of new approaches in genetic engineering and nanotechnology methods in combination with natural cellular phenomenon can modify the disease in affected people. We consider two CRISPR/Cas9 systems to cut a specific region from short arm of the chromosome 21 (Chr21) and replace it with a novel designed DNA construct, containing the essential genes in chromatin remodeling for inactivating of an extra Chr21. This requires mimicking of the natural cellular pattern for inactivation of the extra X chromosome in females. By means of controlled dosage of an appropriate Nano-carrier (a surface engineered Poly D, L-lactide-co-glycolide (PLGA) for integrating the relevant construct in Trisomy21 brain cell culture media and then in DS mouse model, we would be able to evaluate the modification and the reduction of the active extra Chr21 and in turn reduce substantial adverse effects of the disease, like intellectual disabilities. The hypothesis and study seek new insights in Down syndrome modification.
Types de publication
Journal Article
Langues
eng
Pagination
371-378Déclaration de conflit d'intérêts
Conflict of interest The authors declare that there is no conflict of interest.
Références
Res Dev Disabil. 1999 Jul-Aug;20(4):297-303
pubmed: 10425657
Lancet. 2003 Mar 8;361(9360):835-6
pubmed: 12642052
Crit Rev Ther Drug Carrier Syst. 2004;21(5):387-422
pubmed: 15719481
J Control Release. 2005 Jul 20;105(3):185-98
pubmed: 15921775
Curr Opin Genet Dev. 2005 Oct;15(5):482-9
pubmed: 16107314
Zhonghua Yi Xue Za Zhi. 2007 Oct 23;87(39):2759-63
pubmed: 18167266
Genome. 2008 Jan;51(1):19-29
pubmed: 18356936
ChemMedChem. 2008 Sep;3(9):1395-403
pubmed: 18613205
Nat Genet. 2009 Apr;41(4):488-93
pubmed: 19305411
Exp Mol Med. 2009 Jul 31;41(7):471-7
pubmed: 19322033
Genome Res. 2010 May;20(5):614-22
pubmed: 20363980
Genome Biol. 2010;11(6):213
pubmed: 20573260
BMC Genomics. 2010 Nov 03;11:614
pubmed: 21047393
J Neurochem. 2011 May;117(4):613-22
pubmed: 21388375
J Neurosci. 2011 Apr 13;31(15):5764-76
pubmed: 21490218
Hum Genet. 2011 Aug;130(2):237-45
pubmed: 21614513
Chromosoma. 2012 Feb;121(1):71-8
pubmed: 21947602
Genet Med. 2011 Nov;13(11):913-20
pubmed: 22005709
Adv Pharmacol Sci. 2011;2011:153218
pubmed: 22028705
Dis Markers. 2011;31(5):247-57
pubmed: 22048266
Genet Med. 2012 Mar;14(3):296-305
pubmed: 22281937
Science. 2013 Feb 15;339(6121):819-23
pubmed: 23287718
Science. 2013 Feb 15;339(6121):823-6
pubmed: 23287722
Elife. 2013 Jan 29;2:e00471
pubmed: 23386978
Cell. 2013 Feb 28;152(5):1173-83
pubmed: 23452860
Congenit Anom (Kyoto). 2013 Mar;53(1):1-2
pubmed: 23480351
Methods Mol Biol. 2013;1078:1-8
pubmed: 23975816
Transgenic Res. 2014 Apr;23(2):317-29
pubmed: 24293126
Nat Methods. 2014 Jul;11(7):723-6
pubmed: 24797424
Prog Neuropsychopharmacol Biol Psychiatry. 2014 Oct 3;54:140-8
pubmed: 24842803
Nat Commun. 2014 Jun 26;5:4240
pubmed: 24967838
Mol Brain. 2014 Sep 16;7:63
pubmed: 25223359
Stem Cells Dev. 2015 May 1;24(9):1053-65
pubmed: 25517294
Cell. 2015 Jan 15;160(1-2):339-50
pubmed: 25533786
Nat Biotechnol. 2015 Apr;33(4):390-394
pubmed: 25690852
PLoS Genet. 2015 Mar 18;11(3):e1005079
pubmed: 25785854
Angew Chem Int Ed Engl. 2015 Oct 5;54(41):12029-33
pubmed: 26310292
Int J Clin Exp Pathol. 2015 Jul 01;8(7):8385-93
pubmed: 26339408
Transl Res. 2016 Feb;168:15-21
pubmed: 26470680
Science. 2016 Jan 1;351(6268):84-8
pubmed: 26628643
Nature. 2016 Jan 28;529(7587):490-5
pubmed: 26735016
Hum Mol Genet. 2016 Jun 15;25(12):2525-2538
pubmed: 27106104
Int Heart J. 2016 Jul 27;57(4):490-5
pubmed: 27396555
Biomater Res. 2016 Oct 31;20:34
pubmed: 27807476
Cell Res. 2017 Mar;27(3):440-443
pubmed: 28117345
ACS Nano. 2017 Mar 28;11(3):2452-2458
pubmed: 28129503
Proc Natl Acad Sci U S A. 2017 Apr 4;114(14):E2882-E2890
pubmed: 28320934
Cell. 2017 Apr 20;169(3):559
pubmed: 28431253
Sci China Life Sci. 2017 May;60(5):447-457
pubmed: 28534256
Gene. 1997 Dec 31;205(1-2):39-46
pubmed: 9461378