Antibody-Mediated Enzyme Therapeutics and Applications in Glycogen Storage Diseases.
Pompe disease
antibody-directed therapies, Lafora disease
antibody–drug conjugate
antibody–enzyme fusion
glycogen
Journal
Trends in molecular medicine
ISSN: 1471-499X
Titre abrégé: Trends Mol Med
Pays: England
ID NLM: 100966035
Informations de publication
Date de publication:
12 2019
12 2019
Historique:
received:
27
05
2019
revised:
07
08
2019
accepted:
12
08
2019
pubmed:
17
9
2019
medline:
22
7
2020
entrez:
17
9
2019
Statut:
ppublish
Résumé
The use of antibodies as targeting molecules or cell-penetrating tools has emerged at the forefront of pharmaceutical research. Antibody-directed therapies in the form of antibody-drug conjugates, immune modulators, and antibody-directed enzyme prodrugs have been most extensively utilized as hematological, rheumatological, and oncological therapies, but recent developments are identifying additional applications of antibody-mediated delivery systems. A novel application of this technology is for the treatment of glycogen storage disorders (GSDs) via an antibody-enzyme fusion (AEF) platform to penetrate cells and deliver an enzyme to the cytoplasm, nucleus, and/or other organelles. Exciting developments are currently underway for AEFs in the treatment of the GSDs Pompe disease and Lafora disease (LD). Antibody-based therapies are quickly becoming an integral part of modern disease therapeutics.
Identifiants
pubmed: 31522955
pii: S1471-4914(19)30226-6
doi: 10.1016/j.molmed.2019.08.005
pmc: PMC6889062
mid: NIHMS1537636
pii:
doi:
Substances chimiques
Antibodies
0
Immunoconjugates
0
Recombinant Fusion Proteins
0
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
1094-1109Subventions
Organisme : NINDS NIH HHS
ID : P01 NS097197
Pays : United States
Organisme : NINDS NIH HHS
ID : R01 NS070899
Pays : United States
Organisme : NCATS NIH HHS
ID : TL1 TR001997
Pays : United States
Organisme : NCATS NIH HHS
ID : UL1 TR001998
Pays : United States
Informations de copyright
Copyright © 2019 Elsevier Ltd. All rights reserved.
Références
Hum Mol Genet. 2013 Apr 15;22(8):1525-38
pubmed: 23307925
J Physiol. 2018 Feb 1;596(3):445-475
pubmed: 29023798
J Biol Chem. 2018 May 11;293(19):7117-7125
pubmed: 29483193
Inflamm Allergy Drug Targets. 2011 Dec;10(6):497-508
pubmed: 21999177
J Neurosci Res. 2019 Aug;97(8):923-932
pubmed: 30675919
Oncotarget. 2016 Dec 27;7(52):87016-87030
pubmed: 27894092
J Drug Target. 2005 Feb;13(2):81-7
pubmed: 15823959
Eur J Hum Genet. 1999 Sep;7(6):713-6
pubmed: 10482961
Arthritis Rheum. 1987 Jun;30(6):669-78
pubmed: 3606686
Blood. 2013 Jun 13;121(24):4854-60
pubmed: 23591789
Biochem J. 2001 Jun 15;356(Pt 3):867-73
pubmed: 11389696
Sci Rep. 2015 Jul 09;5:12022
pubmed: 26156563
Expert Opin Biol Ther. 2008 Apr;8(4):491-502
pubmed: 18352852
Cancer Res. 2007 Feb 15;67(4):1769-74
pubmed: 17308119
Bioconjug Chem. 2018 Dec 19;29(12):3937-3966
pubmed: 30265523
Adv Drug Deliv Rev. 2017 Dec 1;122:2-19
pubmed: 27916504
Int J Oncol. 2009 Jul;35(1):167-73
pubmed: 19513564
J Biol Chem. 2007 Jul 20;282(29):20790-3
pubmed: 17525162
Br J Haematol. 2018 Oct;183(2):313-317
pubmed: 29048126
Sci Transl Med. 2011 May 25;3(84):84ra44
pubmed: 21613623
Cancer Res. 2003 Mar 15;63(6):1288-96
pubmed: 12649189
Hum Mol Genet. 1999 Feb;8(2):345-52
pubmed: 9931343
Curr Drug Targets. 2016;17(10):1172-88
pubmed: 26648073
PLoS Genet. 2011 Apr;7(4):e1002037
pubmed: 21552327
Neurology. 1991 Nov;41(11):1757-64
pubmed: 1944905
J Histochem Cytochem. 2009 Jan;57(1):1-5
pubmed: 18854594
J Immunol. 2004 Jul 1;173(1):61-7
pubmed: 15210759
Cancer Invest. 2009 Aug;27(7):774-80
pubmed: 19488908
BioDrugs. 2006;20(3):151-60
pubmed: 16724863
Drug Des Devel Ther. 2017 Aug 23;11:2467-2480
pubmed: 28860717
Protein Eng Des Sel. 2006 Jan;19(1):37-45
pubmed: 16243897
Int J Mol Sci. 2017 Aug 11;18(8):
pubmed: 28800070
FEBS Lett. 2002 Apr 10;516(1-3):208-12
pubmed: 11959134
Mol Pharm. 2019 Sep 3;16(9):3791-3801
pubmed: 31329461
Bioconjug Chem. 2010 Jan;21(1):151-6
pubmed: 20000684
Oncotarget. 2016 Sep 13;7(37):59965-59975
pubmed: 27494868
Mol Cancer Ther. 2016 May;15(5):971-84
pubmed: 26826117
J Control Release. 2015 Jan 10;197:78-86
pubmed: 25449807
Biotechnol Bioeng. 2014 Nov;111(11):2317-25
pubmed: 24889100
Br J Haematol. 2006 Feb;132(4):398-409
pubmed: 16412015
Nat Biotechnol. 2005 Sep;23(9):1126-36
pubmed: 16151406
J Clin Endocrinol Metab. 1992 Aug;75(2):424-31
pubmed: 1379254
Ann N Y Acad Sci. 2011 Mar;1222:55-63
pubmed: 21434943
Mol Genet Metab. 2018 Jul;124(3):184-188
pubmed: 29793829
Genet Med. 2009 Mar;11(3):210-9
pubmed: 19287243
Orphanet J Rare Dis. 2018 Jul 5;13(1):110
pubmed: 29976218
Glia. 2018 Oct;66(10):2094-2107
pubmed: 30152044
Int J Cancer. 2016 Jan 1;138(1):182-6
pubmed: 26174762
IUBMB Life. 2012 Oct;64(10):801-8
pubmed: 22815132
Ann Neurol. 2004 Apr;55(4):495-502
pubmed: 15048888
Curr Pharm Biotechnol. 2008 Jun;9(3):180-4
pubmed: 18673283
MAbs. 2014 Jan-Feb;6(1):34-45
pubmed: 24423619
J Biol Chem. 1998 Feb 27;273(9):5288-93
pubmed: 9478986
Neurology. 2007 Jan 9;68(2):99-109
pubmed: 17151339
Sci Transl Med. 2012 Oct 24;4(157):157ra142
pubmed: 23100628
MAbs. 2019 Feb/Mar;11(2):219-238
pubmed: 30516432
Cancer Res. 2008 Nov 15;68(22):9280-90
pubmed: 19010901
Biochimie. 2008 May;90(5):717-25
pubmed: 18291113
Clin Cancer Res. 2001 Apr;7(4):1061-72
pubmed: 11309358
Mol Pharm. 2016 Apr 4;13(4):1385-92
pubmed: 26910785
Adv Drug Deliv Rev. 2013 Oct;65(10):1357-69
pubmed: 23026637
Immunotherapy. 2011 Feb;3(2):193-211
pubmed: 21322759
JCI Insight. 2019 Mar 7;4(5):
pubmed: 30843882
N Engl J Med. 2005 Sep 15;353(11):1114-23
pubmed: 16162882
Clin Cancer Res. 2006 Nov 1;12(21):6509-16
pubmed: 17085666
Hum Mol Genet. 2008 Dec 15;17(24):3897-908
pubmed: 18782848
Drugs Today (Barc). 2009 Jun;45(6):423-30
pubmed: 19649332
Hum Mol Genet. 2010 Feb 15;19(4):684-96
pubmed: 19959526
Adv Drug Deliv Rev. 2017 Sep 1;118:2-7
pubmed: 28916498
Leuk Lymphoma. 2007 Jun;48(6):1179-86
pubmed: 17577782
J Clin Oncol. 2010 Nov 20;28(33):4969-75
pubmed: 20921469
Pediatr Endocrinol Rev. 2014 Sep;12 Suppl 1:107-13
pubmed: 25345092
Bioconjug Chem. 2004 Jan-Feb;15(1):16-26
pubmed: 14733579
Haematologica. 2019 Jan;104(1):7-9
pubmed: 30598494
Folia Neuropathol. 2008;46(2):123-33
pubmed: 18587706
Community Genet. 1999;2(2-3):91-6
pubmed: 15181339
Cancer Res. 2015 Jun 1;75(11):2285-91
pubmed: 25832653
Immunotechnology. 1997 Jun;3(2):127-36
pubmed: 9237097
Clin Cancer Res. 2011 Apr 1;17(7):1998-2005
pubmed: 21447719
Epilepsy Behav. 2019 Jan;90:284-286
pubmed: 30528121
PLoS One. 2008 Jun 18;3(6):e2434
pubmed: 18560598
Clin Immunol. 2018 Dec;197:68-76
pubmed: 30205177
Ann Neurol. 2014 Mar;75(3):442-6
pubmed: 24419970
Microbiol Mol Biol Rev. 2012 Mar;76(1):16-32
pubmed: 22390970
Nature. 1978 Jan 5;271(5640):67-9
pubmed: 625325
J Autoimmun. 1998 Oct;11(5):539-46
pubmed: 9802941
Genet Med. 2012 Sep;14(9):800-10
pubmed: 22538254
Nat Biotechnol. 2012 Jul 10;30(7):631-7
pubmed: 22781692
Proc Natl Acad Sci U S A. 1996 Jan 9;93(1):65-70
pubmed: 8552676
PLoS One. 2012;7(7):e40776
pubmed: 22815812
Neuron. 2016 Jan 6;89(1):70-82
pubmed: 26687840
Hum Mol Genet. 2018 Apr 1;27(7):1290-1300
pubmed: 29408991
Bioconjug Chem. 2008 Jul;19(7):1327-38
pubmed: 18547095
Int J Oncol. 2004 Dec;25(6):1867-73
pubmed: 15547728
Mol Cancer Ther. 2012 Oct;11(10):2169-73
pubmed: 22863609
J Clin Endocrinol Metab. 2003 Jun;88(6):2900-7
pubmed: 12788904
Cancer Immunol Immunother. 2012 Dec;61(12):2261-71
pubmed: 22678096
Genet Med. 2016 Jan;18(1):73-81
pubmed: 25834948
Int J Mol Sci. 2016 Apr 14;17(4):561
pubmed: 27089329
Clin Exp Immunol. 1979 Mar;35(3):364-75
pubmed: 378481
Mol Pharm. 2011 Feb 7;8(1):280-5
pubmed: 21141969
Biotechnol Bioeng. 2011 Aug;108(8):1954-64
pubmed: 21351076
Cancer Lett. 2016 May 1;374(2):229-40
pubmed: 26912070
Cell Metab. 2019 Oct 1;30(4):689-705.e6
pubmed: 31353261
Brain Res. 2010 Sep 17;1352:208-13
pubmed: 20599807
Orphanet J Rare Dis. 2013 Jun 20;8:90
pubmed: 23787031
Protein Eng Des Sel. 2014 Mar;27(3):83-8
pubmed: 24421342
J Immunol. 1996 Sep 1;157(5):2082-8
pubmed: 8757330
Target Oncol. 2018 Aug;13(4):525-532
pubmed: 30090971
Blood Cancer J. 2013 Mar 22;3:e106
pubmed: 23524591
Ann Transl Med. 2018 Aug;6(15):313
pubmed: 30211201
J Immunol. 2000 Jun 1;164(11):6020-6
pubmed: 10820286
Mol Ther Nucleic Acids. 2014 Oct 28;3:e206
pubmed: 25350581
Lancet. 2008 Oct 11;372(9646):1342-53
pubmed: 18929906
J Immunol. 1990 Apr 1;144(7):2653-8
pubmed: 2319132
J Immunol Res. 2015;2015:561814
pubmed: 26605343
ScientificWorldJournal. 2005 Sep 16;5:782-8
pubmed: 16170440
J Cancer Res Clin Oncol. 2013 Mar;139(3):447-55
pubmed: 23160853
Mol Ther. 2018 May 2;26(5):1366-1374
pubmed: 29606503
Virchows Arch B Cell Pathol Incl Mol Pathol. 1984;45(1):23-36
pubmed: 6199885
Dermatol Ther. 2007 Jul-Aug;20(4):270-6
pubmed: 17970892
Am J Med Genet. 1998 Aug 27;79(1):69-72
pubmed: 9738873
Nat Genet. 2003 Oct;35(2):125-7
pubmed: 12958597
Oncotarget. 2019 Feb 12;10(13):1272-1283
pubmed: 30863489
Hum Mol Genet. 2014 Jun 15;23(12):3147-56
pubmed: 24452334
Mol Pharm. 2019 Mar 4;16(3):1412-1420
pubmed: 30714739
Nucleic Acids Res. 2017 Nov 16;45(20):11782-11799
pubmed: 29036688
Science. 1957 Jul 26;126(3265):162-3
pubmed: 13442661
Sci Transl Med. 2017 Nov 29;9(418):
pubmed: 29187643
MAbs. 2017 Feb/Mar;9(2):182-212
pubmed: 28071970
MAbs. 2013 Jan-Feb;5(1):13-21
pubmed: 23221464
J Mol Med (Berl). 2017 May;95(5):513-521
pubmed: 28154884
MAbs. 2016 May-Jun;8(4):659-71
pubmed: 27045800
Brain Res. 2006 May 9;1088(1):187-96
pubmed: 16630585
Mol Genet Metab. 2019 Feb;126(2):83-97
pubmed: 30528228
Lab Invest. 2006 Dec;86(12):1208-20
pubmed: 17075580
Lancet Oncol. 2014 Aug;15(9):986-96
pubmed: 25008258
J Pharmacol Exp Ther. 2010 Jun;333(3):961-9
pubmed: 20233799