[논문]Fetal stem cell and gene therapy

Witt, Russell; MacKenzie, Tippi C.; Peranteau, William H.

doi:10.1016/j.siny.2017.05.003

Fetal stem cell and gene therapy

Seminars in fetal & neonatal medicine, v.22 no.6, 2017년, pp.410 - 414

Witt, Russell (Division of Pediatric Surgery, University of California, San Francisco, San Francisco, CA, USA) , MacKenzie, Tippi C. (Division of Pediatric Surgery, University of California, San Francisco, San Francisco, CA, USA) , Peranteau, William H. (Division of Pediatric General, Thoracic and Fetal Surgery, University of Pennsylvania Perelman School of Medicine, The Children's Hospital of Philadelphia, Philadelphia, PA, USA)

Abstract ▼ AI-Helper

Abstract Advances in our understanding of stem cells, gene editing, prenatal imaging and fetal interventions have opened up new opportunities for the treatment of congenital diseases either through in-utero stem cell transplantation or in-utero gene therapy. Improvements in ultrasound-guided access to the fetal vasculature have also enhanced the safety and efficacy of cell delivery. The fetal environment offers accessible stem cell niches, localized cell populations with large proliferative potential, and an immune system that is able to acquire donor-specific tolerance. In-utero therapy seeks to take advantage of these factors and has the potential to cure diseases prior to the onset of symptoms, a strategy that offers substantial social and economic benefits. In this article, we examine previous studies in animal models as well as clinical attempts at in-utero therapy. We also discuss the barriers to successful in-utero therapy and future strategies for overcoming these obstacles.

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참고문헌 (50)

Minerva Ginecol De Santis 63 387 2011 In-utero stem cell transplantation: clinical use and therapeutic potential

상세보기
Science Owen 102 400 1945 10.1126/science.102.2651.400 Immunogenetic consequences of vascular anastomoses between bovine twins

상세보기
Nature Billingham 172 603 1953 10.1038/172603a0 Actively acquired tolerance of foreign cells

상세보기
Ann N. Y Acad Sci Simonsen 59 448 1955 10.1111/j.1749-6632.1955.tb45959.x The acquired immunity concept in kidney homotransplantation

상세보기
Proc Natl Acad Sci U. S. A Fleischman 76 5736 1979 10.1073/pnas.76.11.5736 Prevention of genetic anemias in mice by microinjection of normal hematopoietic stem cells into the fetal placenta

상세보기
J Exp Med Fleischman 159 731 1984 10.1084/jem.159.3.731 Development of adult bone marrow stem cells in H-2-compatible and -incompatible mouse fetuses

상세보기
Blood Blazar 86 4353 1995 10.1182/blood.V86.11.4353.bloodjournal86114353 In utero transfer of adult bone marrow cells into recipients with severe combined immunodeficiency disorder yields lymphoid progeny with T- and B-cell functional capabilities

상세보기
Biol Blood Marrow Transpl Boelig 22 991 2016 10.1016/j.bbmt.2016.01.017 The intravenous route of injection optimizes engraftment and survival in the murine model of in utero hematopoietic cell transplantation

상세보기
Blood Peranteau 108 4268 2006 10.1182/blood-2006-04-018986 CD26 inhibition enhances allogeneic donor-cell homing and engraftment after in utero hematopoietic-cell transplantation

상세보기
Blood Kim 128 2457 2016 10.1182/blood-2016-06-723981 Enhanced in utero allogeneic engraftment in mice after mobilizing fetal HSCs by alpha4beta1/7 inhibition

상세보기
Blood Derderian 124 973 2014 10.1182/blood-2014-02-550327 In utero depletion of fetal hematopoietic stem cells improves engraftment after neonatal transplantation in mice

상세보기
Blood Peranteau 109 1331 2007 10.1182/blood-2006-04-018606 Evidence for an immune barrier after in utero hematopoietic-cell transplantation

상세보기
Blood Peranteau 100 2225 2002 10.1182/blood-2002-01-0166 High-level allogeneic chimerism achieved by prenatal tolerance induction and postnatal nonmyeloablative bone marrow transplantation

상세보기
Blood Nijagal 121 4595 2013 10.1182/blood-2012-10-463174 Direct and indirect antigen presentation lead to deletion of donor-specific T cells after in utero hematopoietic cell transplantation in mice

상세보기
J Clin Invest Merianos 119 2590 2009 Maternal alloantibodies induce a postnatal immune response that limits engraftment following in utero hematopoietic cell transplantation in mice

상세보기
J Clin Invest Nijagal 121 582 2011 10.1172/JCI44907 Maternal T cells limit engraftment after in utero hematopoietic cell transplantation in mice

상세보기
Blood Durkin 112 5245 2008 10.1182/blood-2007-12-128116 Early chimerism threshold predicts sustained engraftment and NK-cell tolerance in prenatal allogeneic chimeras

상세보기
Front Pharmacol Peranteau 5 251 2014 10.3389/fphar.2014.00251 In utero hematopoietic cell transplantation: induction of donor specific immune tolerance and postnatal transplants

상세보기
Blood Peranteau 126 1245 2015 10.1182/blood-2015-03-636803 Correction of murine hemoglobinopathies by prenatal tolerance induction and postnatal nonmyeloablative allogeneic BM transplants

상세보기
Exp Hematol Ashizuka 34 359 2006 10.1016/j.exphem.2005.11.011 Busulfan-conditioned bone marrow transplantation results in high-level allogeneic chimerism in mice made tolerant by in utero hematopoietic cell transplantation

상세보기
Biol Blood Marrow Transpl Peranteau 15 293 2009 10.1016/j.bbmt.2008.11.034 Haploidentical in utero hematopoietic cell transplantation improves phenotype and can induce tolerance for postnatal same-donor transplants in the canine leukocyte adhesion deficiency model

상세보기
Science Flake 233 776 1986 10.1126/science.2874611 Transplantation of fetal hematopoietic stem cells in utero: the creation of hematopoietic chimeras

상세보기
Experientia Pearce 45 307 1989 10.1007/BF01951819 Induction of hemopoietic chimerism in the caprine fetus by intraperitoneal injection of fetal liver cells

상세보기
J Med Primatol Shields 34 201 2005 10.1111/j.1600-0684.2005.00110.x The use of CD 34(+) mobilized peripheral blood as a donor cell source does not improve chimerism after in utero hematopoietic stem cell transplantation in non-human primates

상세보기
Transplantation Lee 79 1084 2005 10.1097/01.TP.0000161247.61727.67 In utero bone marrow transplantation induces kidney allograft tolerance across a full major histocompatibility complex barrier in swine

상세보기
Ann N. Y Acad Sci Storb 770 149 1995 10.1111/j.1749-6632.1995.tb31052.x Prevention of graft-versus-host disease. Studies in a canine model

상세보기
Am J Obstet Gynecol Blakemore 190 960 2004 10.1016/j.ajog.2004.01.014 In utero hematopoietic stem cell transplantation with haploidentical donor adult bone marrow in a canine model

상세보기
Blood Vrecenak 124 1987 2014 10.1182/blood-2013-11-537571 Stable long-term mixed chimerism achieved in a canine model of allogeneic in utero hematopoietic cell transplantation

상세보기
Lancet Touraine 1 1382 1989 10.1016/S0140-6736(89)92819-5 In-utero transplantation of stem cells in bare lymphocyte syndrome

상세보기
N Engl J Med Flake 335 1806 1996 10.1056/NEJM199612123352404 Treatment of X-linked severe combined immunodeficiency by in utero transplantation of paternal bone marrow

상세보기
Lancet Wengler 348 1484 1996 10.1016/S0140-6736(96)09392-0 In-utero transplantation of parental CD34 haematopoietic progenitor cells in a patient with X-linked severe combined immunodeficiency (SCIDXI)

상세보기
Blood Flake 94 2179 1999 10.1182/blood.V94.7.2179.419k43_2179_2191 In utero hematopoietic stem cell transplantation: ontogenic opportunities and biologic barriers

상세보기
Bone Marrow Transpl Bambach 19 399 1997 10.1038/sj.bmt.1700665 Engraftment following in utero bone marrow transplantation for globoid cell leukodystrophy

상세보기
Hum Gene Ther Clin Dev Calcedo 24 108 2013 10.1089/humc.2012.249 Self-reactive CFTR T cells in humans: implications for gene therapy

상세보기
Nat Med Mingozzi 13 419 2007 10.1038/nm1549 CD8(+) T-cell responses to adeno-associated virus capsid in humans

상세보기
Clin Vaccine Immunol Calcedo 18 1586 2011 10.1128/CVI.05107-11 Adeno-associated virus antibody profiles in newborns, children, and adolescents

상세보기
Mol Ther Sabatino 15 1677 2007 10.1038/sj.mt.6300219 Persistent expression of hF.IX after tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice

상세보기
PLoS One Davey 12 e0171132 2017 10.1371/journal.pone.0171132 Induction of immune tolerance to foreign protein via adeno-associated viral vector gene transfer in mid-gestation fetal sheep

상세보기
Gene Ther Endo 17 61 2010 10.1038/gt.2009.115 The developmental stage determines the distribution and duration of gene expression after early intra-amniotic gene transfer using lentiviral vectors

상세보기
Hum Gene Ther Methods Joyeux 25 197 2014 10.1089/hgtb.2013.143 In utero lung gene transfer using adeno-associated viral and lentiviral vectors in mice

상세보기
Gene Ther Rahim 19 936 2012 10.1038/gt.2011.157 In utero administration of Ad5 and AAV pseudotypes to the fetal brain leads to efficient, widespread and long-term gene expression

상세보기
Gene Ther Koppanati 16 1130 2009 10.1038/gt.2009.71 Systemic delivery of AAV8 in utero results in gene expression in diaphragm and limb muscle: treatment implications for muscle disorders

상세보기
Mol Ther Methods Clin Dev Stitelman 1 14040 2014 10.1038/mtm.2014.40 Developmental stage determines efficiency of gene transfer to muscle satellite cells by in utero delivery of adeno-associated virus vector serotype 2/9

상세보기
Gene Ther Roybal 19 1085 2012 10.1038/gt.2011.186 Early gestational gene transfer with targeted ATP7B expression in the liver improves phenotype in a murine model of Wilson's disease

상세보기
Mol Ther Maeder 24 430 2016 10.1038/mt.2016.10 Genome-editing technologies for gene and cell therapy

상세보기
Nat Biotechnol Yang 34 334 2016 10.1038/nbt.3469 A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

상세보기
Science Tabebordbar 351 407 2016 10.1126/science.aad5177 In vivo gene editing in dystrophic mouse muscle and muscle stem cells

상세보기
Elife Lin 3 e04766 2014 10.7554/eLife.04766 Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery

상세보기
Mol Ther Bennett 25 1076 2017 10.1016/j.ymthe.2017.03.008 Taking stock of retinal gene therapy: looking back and moving forward

상세보기
Semin Hematol George 53 46 2016 10.1053/j.seminhematol.2015.10.002 Gene therapy for hemophilia: past, present and future

상세보기

표제어: PCR

동의어: Packet Collision Rate

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용어 설명: PCR은 세균 특이성이 있는 primer를 이용하여 적은 수의 세균이 있을지라도 쉽게 검출할 수 있는 유용한 방법이며, 이를 이용하여 구강 내 치면세균막이나 타액에서 직접 세균을 검출할 수 있게 되었다[8].

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Fetal stem cell and gene therapy

Abstract ▼ AI-Helper

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