Prenatal Stem Cell Transplantation and Gene Therapy

被引:16
作者
Santore, Matthew T. [1 ]
Roybal, Jessica L. [1 ]
Flake, Alan W. [1 ]
机构
[1] Univ Penn, Sch Med, Dept Surg, Childrens Hosp Philadelphia,Childrens Ctr Fetal R, Philadelphia, PA 19104 USA
关键词
Prenatal treatment; Fetal therapy; Gene therapy; Hematopoietic stem cell transplantation; Immunologic tolerance; IN-UTERO TRANSPLANTATION; BONE-MARROW-TRANSPLANTATION; SEVERE COMBINED IMMUNODEFICIENCY; VERSUS-HOST-DISEASE; GESTATIONAL INTRAAMNIOTIC INJECTION; LEVEL ALLOGENEIC CHIMERISM; TERM TRANSGENE EXPRESSION; CYSTIC-FIBROSIS PHENOTYPE; FETAL LIVER; LONG-TERM;
D O I
10.1016/j.clp.2009.03.006
中图分类号
R71 [妇产科学];
学科分类号
100211 ;
摘要
At the present time, the most likely and eminent application of stem cell therapy to the fetus is in utero hematopoietic stem cell transplantation (IUHCT), and this stem cell type will be discussed as a paradigm for all prenatal stem cell therapy. The authors feel that the most likely initial application of IUHCT will use adult HSC derived from bone marrow (BM) or peripheral blood (PB), and will focus this article on this specific approach. The article also reviews the experimental data that support the capacity of IUHCT to induce donor-specific tolerance.
引用
收藏
页码:451 / +
页数:22
相关论文
共 123 条
[1]   THE USE OF SKIN GRAFTING TO DISTINGUISH BETWEEN MONOZYGOTIC AND DIZYGOTIC TWINS IN CATTLE [J].
ANDERSON, D ;
BILLINGHAM, RE ;
LAMPKIN, GH ;
MEDAWAR, PB .
HEREDITY, 1951, 5 (03) :379-+
[2]   Busulfan-conditioned bone marrow transplantation results in high-level allogeneic chimerism in mice made tolerant by in utero hematopoietic cell transplantation [J].
Ashizuka, S ;
Peranteau, WH ;
Hayashi, S ;
Flake, AW .
EXPERIMENTAL HEMATOLOGY, 2006, 34 (03) :359-368
[3]   Vertebrate cranial placodes I. Embryonic induction [J].
Baker, CVH ;
Bronner-Fraser, M .
DEVELOPMENTAL BIOLOGY, 2001, 232 (01) :1-61
[4]   ACTIVELY ACQUIRED TOLERANCE OF FOREIGN CELLS [J].
BILLINGHAM, RE ;
BRENT, L ;
MEDAWAR, PB .
NATURE, 1953, 172 (4379) :603-606
[5]   Developing a new paradigm for thymus organogenesis [J].
Blackburn, CC ;
Manley, NR .
NATURE REVIEWS IMMUNOLOGY, 2004, 4 (04) :278-289
[6]   In utero hematopoietic stem cell transplantation with haploidentical donor adult bone marrow in a canine model [J].
Blakemore, K ;
Hattenburg, C ;
Stetten, G ;
Berg, K ;
South, S ;
Murphy, K ;
Jones, R .
AMERICAN JOURNAL OF OBSTETRICS AND GYNECOLOGY, 2004, 190 (04) :960-972
[7]   IN-UTERO TRANSFER OF ADULT BONE-MARROW CELLS INTO RECIPIENTS WITH SEVERE COMBINED IMMUNODEFICIENCY DISORDER YIELDS LYMPHOID PROGENY WITH T-CELL AND B-CELL FUNCTIONAL CAPABILITIES [J].
BLAZAR, BR ;
TAYLOR, PA ;
VALLERA, DA .
BLOOD, 1995, 86 (11) :4353-4366
[8]   ADULT BONE-MARROW-DERIVED PLURIPOTENT HEMATOPOIETIC STEM-CELLS ARE ENGRAFTABLE WHEN TRANSFERRED IN-UTERO INTO MODERATELY ANEMIC FETAL RECIPIENTS [J].
BLAZAR, BR ;
TAYLOR, PA ;
VALLERA, DA .
BLOOD, 1995, 85 (03) :833-841
[9]  
BONEKO VM, 1988, ACTA ANAT, V133, P325
[10]   Long-term transgene expression in cardiac and skeletal muscle following fetal administration of adenoviral or adeno-associated viral vectors in mice [J].
Bouchard, S ;
MacKenzie, TC ;
Radu, AP ;
Hayashi, S ;
Peranteau, WH ;
Chirmule, N ;
Flake, AW .
JOURNAL OF GENE MEDICINE, 2003, 5 (11) :941-950