Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies.
CAR NK cell
CAR T cell
gene therapy
hematopoietic stem and progenitor cell
mesenchymal stromal cell
somatic cell therapy
tissue-engineered medicinal product
Journal
Pharmaceutics
ISSN: 1999-4923
Titre abrégé: Pharmaceutics
Pays: Switzerland
ID NLM: 101534003
Informations de publication
Date de publication:
05 Apr 2022
05 Apr 2022
Historique:
received:
28
02
2022
revised:
29
03
2022
accepted:
01
04
2022
entrez:
23
4
2022
pubmed:
24
4
2022
medline:
24
4
2022
Statut:
epublish
Résumé
Advanced therapy medicinal products (ATMPs) are medicines for human use based on genes, cells or tissue engineering. After clear successes in adults, the nascent technology now sees increasing pediatric application. For many still untreatable disorders with pre- or perinatal onset, timely intervention is simply indispensable; thus, prenatal and pediatric applications of ATMPs hold great promise for curative treatments. Moreover, for most inherited disorders, early ATMP application may substantially improve efficiency, economy and accessibility compared with application in adults. Vindicating this notion, initial data for cell-based ATMPs show better cell yields, success rates and corrections of disease parameters for younger patients, in addition to reduced overall cell and vector requirements, illustrating that early application may resolve key obstacles to the widespread application of ATMPs for inherited disorders. Here, we provide a selective review of the latest ATMP developments for prenatal, perinatal and pediatric use, with special emphasis on its comparison with ATMPs for adults. Taken together, we provide a perspective on the enormous potential and key framework parameters of clinical prenatal and pediatric ATMP application.
Identifiants
pubmed: 35456627
pii: pharmaceutics14040793
doi: 10.3390/pharmaceutics14040793
pmc: PMC9031205
pii:
doi:
Types de publication
Journal Article
Review
Langues
eng
Subventions
Organisme : European Commission
ID : 777554
Organisme : Research and Innovation Foundation of Cyprus
ID : EXCELLENCE/1216/0092
Organisme : Research and Innovation Foundation of Cyprus
ID : EXCELLENCE/0421/0086
Organisme : EEA and Norway Grants
ID : New infrastructure for diagnosis and treatment of patients
Références
Stem Cell Res Ther. 2021 Feb 26;12(1):152
pubmed: 33637125
Endocrinology. 2013 Feb;154(2):911-9
pubmed: 23288908
J Drug Target. 2016;24(1):58-67
pubmed: 26133964
Biomaterials. 2021 Jan;268:120580
pubmed: 33321292
Cells. 2019 Dec 05;8(12):
pubmed: 31817480
Mol Ther. 2013 Dec;21(12):2136-47
pubmed: 23817205
Biol Blood Marrow Transplant. 2014 Mar;20(3):375-81
pubmed: 24321746
J Tissue Eng. 2020 Jun 7;11:2041731420926918
pubmed: 32551034
Front Genome Ed. 2021 Mar 24;3:618346
pubmed: 34713249
J Biomed Mater Res. 2000 Mar 5;49(3):328-37
pubmed: 10602065
Haemophilia. 2021 Feb;27 Suppl 3:103-113
pubmed: 32484283
J Intern Med. 2016 Sep;280(3):246-51
pubmed: 27114283
Proc Natl Acad Sci U S A. 1995 Sep 26;92(20):9259-63
pubmed: 7568113
Hum Gene Ther Clin Dev. 2019 Sep;30(3):102-113
pubmed: 30968714
Acta Biomater. 2021 Apr 15;125:172-182
pubmed: 33639311
Mol Ther Methods Clin Dev. 2016 Mar 30;5:16020
pubmed: 27069953
Mol Ther Nucleic Acids. 2021 Jul 21;26:222-236
pubmed: 34513306
J Vis Exp. 2011 Jan 27;(47):
pubmed: 21307829
Proc Natl Acad Sci U S A. 1998 Sep 29;95(20):11939-44
pubmed: 9751769
Tissue Eng. 2004 Jan-Feb;10(1-2):231-9
pubmed: 15009948
Biosci Rep. 2015 Apr 28;35(2):
pubmed: 25797907
Nat Rev Drug Discov. 2021 Feb;20(2):101-124
pubmed: 33277608
Ann N Y Acad Sci. 2005;1054:308-16
pubmed: 16339679
N Engl J Med. 2009 Jan 29;360(5):447-58
pubmed: 19179314
Sci Transl Med. 2019 Apr 17;11(488):
pubmed: 30996081
Pharm Res. 2018 Feb 23;35(4):71
pubmed: 29476301
Cytotherapy. 2017 Feb;19(2):311-326
pubmed: 28088294
Hum Mol Genet. 2008 Apr 15;17(R1):R76-83
pubmed: 18632701
Mol Ther. 2013 Jan;21(1):175-84
pubmed: 22371846
Mol Ther. 2013 Feb;21(2):324-37
pubmed: 23299800
Am J Respir Cell Mol Biol. 2005 Aug;33(2):145-52
pubmed: 15891110
Transfus Apher Sci. 2021 Feb;60(1):103005
pubmed: 33223472
PLoS One. 2016 Jan 28;11(1):e0147170
pubmed: 26821255
Am J Physiol Lung Cell Mol Physiol. 2012 May 1;302(9):L829-37
pubmed: 22328358
Curr Transplant Rep. 2018;5(3):244-250
pubmed: 30221121
Mol Ther Oncolytics. 2021 Dec 31;24:385-399
pubmed: 35118195
PLoS One. 2017 Oct 26;12(10):e0186637
pubmed: 29073149
Nat Med. 2020 Oct;26(10):1569-1575
pubmed: 33020647
Cells. 2021 May 26;10(6):
pubmed: 34073206
Sci Adv. 2016 May 06;2(5):e1600349
pubmed: 27386551
Adv Drug Deliv Rev. 2021 Oct;177:113950
pubmed: 34454979
Dev Cell. 2020 Aug 10;54(3):333-347.e7
pubmed: 32800293
Cell Prolif. 2020 Jan;53(1):e12712
pubmed: 31730279
Hum Gene Ther Methods. 2014 Jun;25(3):197-205
pubmed: 24660751
J Clin Pharmacol. 2018 Oct;58 Suppl 10:S48-S57
pubmed: 30248197
Ophthalmic Res. 2017;58(4):217-226
pubmed: 28858866
Cells. 2021 Dec 03;10(12):
pubmed: 34943920
Bone Marrow Transplant. 2011 Feb;46(2):200-7
pubmed: 20400983
CRISPR J. 2019 Oct;2(5):299-303
pubmed: 31599678
Pediatr Crit Care Med. 2015 Jun;16(5):428-39
pubmed: 25647235
Lancet. 2015 Feb 7;385(9967):517-528
pubmed: 25319501
Stem Cells. 2015 Jan;33(1):122-32
pubmed: 25186828
Paediatr Respir Rev. 2017 Sep;24:17-18
pubmed: 28697967
Stem Cells. 2019 Sep;37(9):1176-1188
pubmed: 31116895
PLoS One. 2015 Oct 16;10(10):e0140879
pubmed: 26473479
JAMA. 2016 Nov 1;316(17):1808-1817
pubmed: 27802546
Cells. 2021 Dec 10;10(12):
pubmed: 34944002
EBioMedicine. 2015 Mar 28;2(5):467-75
pubmed: 26137590
Nature. 2018 Dec;564(7735):S6-S8
pubmed: 30542187
Cytotherapy. 2017 Dec;19(12):1383-1391
pubmed: 28935190
Cell Transplant. 2015;24(3):339-47
pubmed: 25622293
Hum Gene Ther. 2020 Jul;31(13-14):709-718
pubmed: 32578444
Yale J Biol Med. 2017 Dec 19;90(4):683-693
pubmed: 29259533
Regen Med. 2021 Jul;16(7):655-667
pubmed: 34278825
Bone Marrow Transplant. 2004 Mar;33(6):597-604
pubmed: 14716336
FASEB J. 2019 Mar;33(3):3954-3967
pubmed: 30517034
Haematologica. 2019 Nov;104(11):e497-e501
pubmed: 31004018
Hum Gene Ther. 2020 Jan;31(1-2):20-46
pubmed: 31802714
Biotechnol Bioeng. 2014 Jan;111(1):69-83
pubmed: 23893544
Leuk Lymphoma. 2022 Jun;63(6):1418-1427
pubmed: 35105265
Lancet. 2022 Jan 22;399(10322):372-383
pubmed: 35065785
Cytotherapy. 2021 Mar;23(3):261-274
pubmed: 33483292
Immunother Adv. 2021 Mar 09;1(1):ltab004
pubmed: 34056638
Br Med Bull. 2012;101:147-61
pubmed: 22279079
N Engl J Med. 2018 Apr 19;378(16):1479-1493
pubmed: 29669226
Curr Gene Ther. 2017;16(5):338-348
pubmed: 28103787
J Pediatr. 2014 May;164(5):966-972.e6
pubmed: 24508444
Pediatr Int. 2019 Oct;61(10):945-950
pubmed: 31487104
Nat Commun. 2018 Jun 26;9(1):2481
pubmed: 29946143
Nature. 2017 Nov 16;551(7680):327-332
pubmed: 29144448
Front Cell Dev Biol. 2021 Mar 19;9:654885
pubmed: 33869217
Materials (Basel). 2020 Oct 13;13(20):
pubmed: 33066064
ACS Biomater Sci Eng. 2020 Oct 12;6(10):5866-5873
pubmed: 33320575
Mol Ther Methods Clin Dev. 2018 Oct 11;11:121-130
pubmed: 30456217
Cytotherapy. 2018 Nov;20(11):1401-1413
pubmed: 30366616
Cells. 2021 Jun 14;10(6):
pubmed: 34198536
Pediatr Clin North Am. 2017 Oct;64(5):1017-1037
pubmed: 28941533
J Biol Chem. 1998 Feb 27;273(9):5093-100
pubmed: 9478961
Pharmaceutics. 2021 Mar 06;13(3):
pubmed: 33800934
Adv Drug Deliv Rev. 2014 Jun;73:162-9
pubmed: 24583376
Transplant Cell Ther. 2022 Feb;28(2):70.e1-70.e5
pubmed: 34838786
Hum Gene Ther. 2021 Oct;32(19-20):997-1003
pubmed: 33843251
Stem Cell Res Ther. 2021 Jun 16;12(1):350
pubmed: 34134774
Curr Pediatr Rev. 2018;14(4):227-238
pubmed: 30205800
EMBO Mol Med. 2017 Jun;9(6):737-740
pubmed: 28396566
Eur J Cancer. 2020 Mar;127:52-66
pubmed: 31986450
EMBO J. 2001 Dec 3;20(23):6877-88
pubmed: 11726523
J Mol Med (Berl). 2021 May;99(5):593-617
pubmed: 33594520
Hum Gene Ther. 2022 Mar;33(5-6):318-329
pubmed: 35152726
J Hematother Stem Cell Res. 2001 Aug;10(4):535-44
pubmed: 11522236
BMC Public Health. 2012 Jun 18;12:439
pubmed: 22709383
J Pharm Policy Pract. 2021 Mar 19;14(1):30
pubmed: 33741076
Mol Diagn Ther. 2019 Apr;23(2):201-222
pubmed: 30945166
Lancet Respir Med. 2019 Feb;7(2):105-106
pubmed: 30473410
Orphanet J Rare Dis. 2017 Apr 3;12(1):64
pubmed: 28372595
Polymers (Basel). 2019 Apr 25;11(4):
pubmed: 31027272
N Engl J Med. 2017 Aug 10;377(6):562-572
pubmed: 28792873
Leukemia. 2020 Apr;34(4):985-1005
pubmed: 32055000
Eur J Immunol. 2021 Sep;51(9):2151-2163
pubmed: 34196410
Gene Ther. 2020 Dec;27(12):537-544
pubmed: 31024072
Stem Cell Rev Rep. 2020 Dec;16(6):1292-1304
pubmed: 33011925
N Engl J Med. 2017 Mar 2;376(9):848-855
pubmed: 28249145
Nat Med. 2019 Feb;25(2):234-241
pubmed: 30664781
Adv Drug Deliv Rev. 2021 Feb;169:51-62
pubmed: 33181188
N Engl J Med. 2019 Apr 18;380(16):1525-1534
pubmed: 30995372
Cells. 2021 Oct 21;10(11):
pubmed: 34831046
Cell. 2022 Jan 20;185(2):250-265.e16
pubmed: 35021064
J Immunol. 2004 Jul 15;173(2):1380-9
pubmed: 15240734
Bone Marrow Transplant. 2009 Mar;43(6):447-54
pubmed: 18955980
Front Oncol. 2021 Feb 02;10:607362
pubmed: 33604290
Lancet Respir Med. 2019 Feb;7(2):115-128
pubmed: 30361119
Biochem Pharmacol. 2020 Apr;174:113692
pubmed: 31705854
Int J Mol Sci. 2021 Sep 15;22(18):
pubmed: 34576129
Mol Ther. 2007 Sep;15(9):1677-85
pubmed: 17565352
Biochim Biophys Acta Mol Basis Dis. 2021 Oct 1;1867(10):166206
pubmed: 34197912
J Vis Exp. 2021 Mar 31;(169):
pubmed: 33871447
Br J Clin Pharmacol. 2021 Jun;87(6):2450-2458
pubmed: 33600022
Bone. 2019 Jun;123:265-273
pubmed: 30946971
Blood. 2021 Dec 16;138(24):2469-2484
pubmed: 34525183
Mol Ther. 2021 Feb 3;29(2):489-504
pubmed: 33221437
PeerJ. 2022 Feb 15;10:e12872
pubmed: 35186473
J Mater Chem B. 2020 Aug 21;8(31):6548-6561
pubmed: 32452510
Int J Mol Sci. 2019 Aug 05;20(15):
pubmed: 31387282
Lancet. 2008 May 10;371(9624):1579-86
pubmed: 18468541
Lancet Respir Med. 2015 Jan;3(1):24-32
pubmed: 25529339
Mol Ther Methods Clin Dev. 2018 Feb 15;9:142-152
pubmed: 29766024
Eur J Clin Pharmacol. 2006 Nov;62(11):947-52
pubmed: 17021892
Exp Ther Med. 2021 Sep;22(3):974
pubmed: 34335916
J Biol Chem. 2009 Feb 20;284(8):4889-96
pubmed: 19098001
Curr Stem Cell Res Ther. 2008 Dec;3(4):229-36
pubmed: 19075753
World J Gastroenterol. 2015 Apr 28;21(16):4779-87
pubmed: 25944991
J Immunother Cancer. 2021 Dec;9(12):
pubmed: 34916256
J Clin Med. 2020 Mar 03;9(3):
pubmed: 32138309
BMC Pediatr. 2020 Mar 16;20(1):123
pubmed: 32178652
Pediatr Transplant. 2012 Sep;16(6):589-98
pubmed: 22738184
Pharmaceutics. 2021 May 11;13(5):
pubmed: 34064872
N Engl J Med. 2005 May 19;352(20):2069-81
pubmed: 15901860
Mol Ther. 2016 Jun;24(6):1017-1018
pubmed: 27324445
Exp Hematol. 2021 Mar;95:31-45
pubmed: 33484750
J Control Release. 2021 May 10;333:129-138
pubmed: 33775685
Front Pharmacol. 2021 Oct 08;12:755052
pubmed: 34690785
Int J Mol Sci. 2021 Nov 18;22(22):
pubmed: 34830312
EMBO Mol Med. 2019 Mar;11(3):
pubmed: 30770338
J Mark Access Health Policy. 2019 Apr 19;7(1):1600939
pubmed: 31069029
Stem Cells Transl Med. 2020 Feb;9(2):158-168
pubmed: 31746123
Stem Cell Res Ther. 2021 Mar 31;12(1):219
pubmed: 33789750
Mol Ther. 2019 Apr 10;27(4):705-707
pubmed: 30837116
Blood. 2015 Apr 23;125(17):2630-40
pubmed: 25762176
Nature. 2010 Sep 16;467(7313):318-22
pubmed: 20844535
Sci Rep. 2020 Nov 11;10(1):19532
pubmed: 33177564
Hum Gene Ther. 2009 Mar;20(3):201-15
pubmed: 19301473
Stem Cells Dev. 2018 Apr 15;27(8):515-523
pubmed: 29482456
Mol Ther. 2020 Feb 5;28(2):352-356
pubmed: 31951833
Front Vet Sci. 2020 Mar 11;7:80
pubmed: 32219101
Front Immunol. 2020 Apr 09;11:588
pubmed: 32328065
Mol Ther. 2018 Apr 4;26(4):1137-1153
pubmed: 29503198
Cell Transplant. 2018 Jan;27(1):130-139
pubmed: 29562772
Stem Cell Res Ther. 2021 Jul 2;12(1):374
pubmed: 34215336
Hum Reprod Update. 2021 Feb 19;27(2):280-304
pubmed: 33279994
Cancer Biol Med. 2021 Aug 14;:
pubmed: 34390235
Biol Blood Marrow Transplant. 2010 Sep;16(9):1293-301
pubmed: 20350611
Mol Ther. 2009 Dec;17(12):2000-9
pubmed: 19755962
Blood. 2021 Nov 4;138(18):1677-1690
pubmed: 33895800
Ann Transl Med. 2021 Nov;9(21):1615
pubmed: 34926659
Sci Adv. 2021 Jan 13;7(3):
pubmed: 33523869
Biomolecules. 2021 Apr 20;11(4):
pubmed: 33924076
Mol Ther. 2015 Mar;23(3):414-22
pubmed: 25515709
Daru. 2020 Dec;28(2):799-805
pubmed: 32617864
Exp Hematol. 2003 Feb;31(2):176-84
pubmed: 12591283
Blood. 2005 Feb 15;105(4):1815-22
pubmed: 15494428
Plast Reconstr Surg Glob Open. 2021 Jan 26;9(1):e3380
pubmed: 33564596
Trends Mol Med. 2021 Aug;27(8):728-730
pubmed: 34176774
Nat Commun. 2019 Jan 3;10(1):45
pubmed: 30604748
N Engl J Med. 2021 Nov 18;385(21):1929-1940
pubmed: 34788506
Cytotherapy. 2018 Jun;20(6):769-778
pubmed: 29730080
Hum Gene Ther. 2019 Sep;30(9):1067-1078
pubmed: 31288584
J Pediatr. 2019 Jul;210:209-213.e2
pubmed: 30992220
Pharmaceutics. 2020 Dec 30;13(1):
pubmed: 33396805
J Clin Diagn Res. 2015 Jan;9(1):GE01-6
pubmed: 25738007
Sci Rep. 2019 Aug 12;9(1):11592
pubmed: 31406195
Methods Cell Biol. 2022;167:149-161
pubmed: 35152992
Circulation. 2011 Oct 18;124(16):1747-54
pubmed: 21931080
J Exp Clin Cancer Res. 2022 Jan 3;41(1):2
pubmed: 34980210
N Engl J Med. 2018 Apr 26;378(17):1604-1610
pubmed: 29694819
Blood. 2007 Sep 15;110(6):1770-8
pubmed: 17507662
Nanotechnol Sci Appl. 2010 Sep 20;3:53-63
pubmed: 24198471
J Pediatr Hematol Oncol. 2021 Apr 1;43(3):e419-e425
pubmed: 32118816
Cells. 2019 Feb 18;8(2):
pubmed: 30781676
Cell Stem Cell. 2016 Jul 7;19(1):127-38
pubmed: 27374787
Stem Cells Transl Med. 2021 Aug;10(8):1129-1137
pubmed: 33876883
Ann Plast Surg. 2014 Jul;73(1):25-9
pubmed: 24918735
World J Stem Cells. 2022 Jan 26;14(1):54-75
pubmed: 35126828
Nat Biotechnol. 2010 Mar;28(3):271-4
pubmed: 20190738
Lancet Respir Med. 2019 Feb;7(2):154-162
pubmed: 30455077
Bone Marrow Transplant. 1995 Oct;16(4):557-64
pubmed: 8528172
Nanomedicine (Lond). 2016 Apr;11(8):941-57
pubmed: 26979802
Transfus Apher Sci. 2016 Dec;55(3):338-343
pubmed: 27765663
Stem Cell Reports. 2014 Jun 06;3(1):60-72
pubmed: 25068122
Exp Neurol. 2008 Sep;213(1):18-27
pubmed: 18639872
Paediatr Respir Rev. 2016 Sep;20:33-41
pubmed: 27425012
Orthop J Sports Med. 2019 Apr 25;7(4):2325967119841077
pubmed: 31041335
Value Health. 2019 Jun;22(6):621-626
pubmed: 31198178
Hum Gene Ther. 2020 Jul;31(13-14):743-755
pubmed: 32414297
J Neurosci Methods. 2005 Apr 30;143(2):151-8
pubmed: 15814147
Br J Pharmacol. 2021 Jan;178(2):262-279
pubmed: 33140850
Nat Med. 2018 Oct;24(10):1513-1518
pubmed: 30297903
AAPS J. 2021 Jun 2;23(4):78
pubmed: 34076797
Cytotherapy. 2018 Jan;20(1):62-73
pubmed: 29107738
Mol Ther. 2005 Oct;12(4):763-71
pubmed: 16084128
F1000Res. 2019 Dec 20;8:
pubmed: 32025285
J Manag Care Spec Pharm. 2019 Jul;25(7):793-799
pubmed: 30784347
Regen Ther. 2021 Sep 13;18:355-362
pubmed: 34584912
Front Pharmacol. 2014 Dec 19;5:270
pubmed: 25566071
Nat Genet. 1995 Mar;9(3):243-8
pubmed: 7773286
Open Med. 2007 Jul 10;1(2):e74-88
pubmed: 20101298
Front Oncol. 2021 Aug 06;11:720501
pubmed: 34422667
Cell Mol Biol (Noisy-le-grand). 2005 Sep 02;51(1):3-22
pubmed: 16171561
Blood. 2012 Mar 22;119(12):2935-42
pubmed: 22160619
Blood. 2021 Feb 4;137(5):624-636
pubmed: 32902645
Ann N Y Acad Sci. 2010 Aug;1202:45-51
pubmed: 20712771
Orphanet J Rare Dis. 2007 Sep 04;2:35
pubmed: 17784964
Oncol Rep. 2019 Dec;42(6):2183-2195
pubmed: 31578576
Mol Ther. 2019 Jan 2;27(1):137-150
pubmed: 30424953
Haematologica. 2016 Aug;101(8):985-94
pubmed: 27175026
Am J Respir Crit Care Med. 2009 Dec 1;180(11):1122-30
pubmed: 19713447
Hematol Oncol Clin North Am. 2017 Oct;31(5):771-785
pubmed: 28895846
Exp Hematol. 2018 Jun;62:45-55
pubmed: 29605545
EBioMedicine. 2020 Sep;59:102975
pubmed: 32853984
Curr Opin Virol. 2021 Jun;48:49-56
pubmed: 33892224
Gene Ther. 2010 Aug;17(8):939-48
pubmed: 20428209
Cytotherapy. 2021 Feb;23(2):165-175
pubmed: 33011075
Eur J Hum Genet. 2006 Mar;14(3):266-72
pubmed: 16333317
Cureus. 2021 Jul 7;13(7):e16226
pubmed: 34367826
Nat Med. 2021 Apr;27(4):563
pubmed: 33859432
Prenat Diagn. 2016 Dec;36(13):1242-1249
pubmed: 27862048
Blood Adv. 2021 Feb 23;5(4):1122-1135
pubmed: 33620431
Immune Netw. 2014 Apr;14(2):81-8
pubmed: 24851097
Biol Blood Marrow Transplant. 2014 Feb;20(2):229-35
pubmed: 24216185
Stem Cells. 2015 May;33(5):1532-42
pubmed: 25588820
Am J Physiol Lung Cell Mol Physiol. 2009 Dec;297(6):L1160-9
pubmed: 19734318
Mol Ther Methods Clin Dev. 2016 Jun 08;3:16036
pubmed: 27331076
Transfus Apher Sci. 2015 Aug;53(1):17-22
pubmed: 26116046
Transplant Proc. 2007 Mar;39(2):573-6
pubmed: 17362785
Stem Cell Res Ther. 2021 May 29;12(1):310
pubmed: 34051862
Blood. 2006 Nov 1;108(9):2979-88
pubmed: 16835374
Int J Stem Cells. 2021 Feb 28;14(1):112-118
pubmed: 33377456
Eur J Health Econ. 2020 Apr;21(3):311-320
pubmed: 31919703
Bone Marrow Transplant. 2013 Aug;48(8):1040-5
pubmed: 23396407
Front Immunol. 2021 Nov 11;12:738204
pubmed: 34858399
Hum Gene Ther. 2018 Mar;29(3):312-326
pubmed: 28922945
Nat Biotechnol. 2012 Jun 07;30(6):479
pubmed: 22678374
Transplant Cell Ther. 2021 Aug;27(8):658.e1-658.e10
pubmed: 33964513
Cells. 2019 Dec 17;8(12):
pubmed: 31861245
Pulm Circ. 2012 Apr-Jun;2(2):170-81
pubmed: 22837858
Thorax. 2012 Jun;67(6):475-6
pubmed: 22504891
Front Genome Ed. 2020 Dec 10;2:613252
pubmed: 34713236
Pediatr Transplant. 2003;7 Suppl 3:65-70
pubmed: 12603696
Blood. 2009 May 7;113(19):4763-70
pubmed: 19258591
J Pediatr. 2017 Jun;185:49-54.e2
pubmed: 28341525
N Engl J Med. 2019 Mar 7;380(10):897-899
pubmed: 30649993
Front Immunol. 2019 Jul 31;10:1645
pubmed: 31417542
Hear Res. 2020 Sep 1;394:107931
pubmed: 32173115
Nat Med. 2018 Sep;24(9):1317-1323
pubmed: 30013199
Nat Med. 2006 Dec;12(12):1397-402
pubmed: 17115047
Mol Ther. 2007 Oct;15(10):1782-8
pubmed: 17637720
Pharmaceutics. 2021 Dec 17;13(12):
pubmed: 34959459
Bioengineering (Basel). 2020 Jul 20;7(3):
pubmed: 32698363
Biol Blood Marrow Transplant. 2011 Apr;17(4):534-41
pubmed: 20457269
Nat Commun. 2021 Jul 13;12(1):4291
pubmed: 34257302
Stem Cells Transl Med. 2019 Nov;8(11):1135-1148
pubmed: 31313507
Int J Biochem Cell Biol. 2018 Dec;105:35-40
pubmed: 30266525
Mol Ther. 2017 Aug 2;25(8):1843-1853
pubmed: 28462816