Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency

被引：65

作者：

Chinen, Javier

Davis, Joie

De Ravin, Suk See

Hay, Beverly N.

Hsu, Amy P.

Linton, Gilda F.

Naumann, Nora

Nomicos, Effie Y. H.

Silvin, Christopher

Ulrick, Jean

Whiting-Theobald, Narda L.

Malech, Harry L.

Puck, Jennifer M.

机构：

[1] NIAID, Host Def Lab, NIH, Dept Hlth & Human Serv, Bethesda, MD 20892 USA

[2] NHGRI, Genet & Mol Biol Branch, NIAID, NIH,DHHS, Bethesda, MD 20892 USA

来源：

BLOOD | 2007年 / 110卷 / 01期

关键词：

BONE-MARROW-TRANSPLANTATION; CHRONIC GRANULOMATOUS-DISEASE; STEM-CELL TRANSPLANTATION; T-CELL; CD34(+) CELLS; TRANSDUCTION; SCID-X1; SURVIVAL; MODEL; IL2RG;

D O I：

10.1182/blood-2006-11-058933

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Retroviral gene therapy can restore immunity to infants with X-linked severe combined immunodeficiency (XSCID) caused by mutations in the IL2RG gene encoding the common gamma chain (gamma c) of receptors for interleukins 2 (IL-2), -4, -7, -9, -15, and -21. We investigated the safety and efficacy of gene therapy as salvage treatment for older XSCID children with inadequate immune reconstitution despite prior bone marrow transplant from a parent. Subjects received retrovirus-transduced autologous peripherally mobilized CD34(+) hematopoletic cells. T-cell function significantly improved in the youngest subject (age 10 years), and multilineage retroviral marking occurred in all 3 children.

引用

页码：67 / 73

页数：7

共 32 条

[1] Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies:: report of the European experience 1968-99 [J].

Antoine, C ;

Müller, S ;

Cant, A ;

Cavazzana-Calvo, M ;

Veys, P ;

Vossen, J ;

Fasth, A ;

Heilmann, C ;

Wulffraat, N ;

Seger, R ;

Blanche, S ;

Friedrich, W ;

Abinun, M ;

Davies, G ;

Bredius, R ;

Schulz, A ;

Landais, P ;

Fischer, A .

LANCET, 2003, 361 (9357) :553-560

[2]

Buckley R, 2007, PRIMARY IMMUNODEFICI, P669

[3] Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency [J].

Buckley, RH ;

Schiff, SE ;

Schiff, RI ;

Markert, ML ;

Williams, LW ;

Roberts, JL ;

Myers, LA ;

Ward, FE .

NEW ENGLAND JOURNAL OF MEDICINE, 1999, 340 (07) :508-516

[4] Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease [J].

Cavazzana-Calvo, M ;

Hacein-Bey, S ;

Basile, CD ;

Gross, F ;

Yvon, E ;

Nusbaum, P ;

Selz, F ;

Hue, C ;

Certain, S ;

Casanova, JL ;

Bousso, P ;

Le Deist, F ;

Fischer, A .

SCIENCE, 2000, 288 (5466) :669-672

[5]

Cavazzana-Calvo Marina, 2005, Annu Rev Med, V56, P585, DOI 10.1146/annurev.med.56.090203.104142

[6]

ClementDeBoers A, 1996, J PEDIATR-US, V129, P544

[7] Dental manifestations in severe combined immunodeficiency following bone marrow transplantation [J].

Cole, BOI ;

Welbury, RR ;

Bond, E ;

Abinun, M .

BONE MARROW TRANSPLANTATION, 2000, 25 (09) :1007-1009

[8] Changes in thymic function with age and during the treatment of HIV infection [J].

Douek, DC ;

McFarland, RD ;

Keiser, PH ;

Gage, EA ;

Massey, JM ;

Haynes, BF ;

Polis, MA ;

Haase, AT ;

Feinberg, MB ;

Sullivan, JL ;

Jamieson, BD ;

Zack, JA ;

Picker, LJ ;

Koup, RA .

NATURE, 1998, 396 (6712) :690-695

[9] Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector [J].

Gaspar, HB ;

Parsley, KL ;

Howe, S ;

King, D ;

Gilmour, KC ;

Sinclair, J ;

Brouns, G ;

Schmidt, M ;

Von Kalle, C ;

Barington, T ;

Jakobsen, MA ;

Christensen, HO ;

Al Ghonaium, A ;

White, HN ;

Smith, JL ;

Levinsky, RJ ;

Ali, RR ;

Kinnon, C ;

Thrasher, AJ .

LANCET, 2004, 364 (9452) :2181-2187

[10] The Effect of the B2 Mutation on Hematopoietic Reconstitution in the French X-SCID Gene Therapy Trial [J].

Gray, John T. ;

Hacein-Bey-Abina, Salima ;

Cavazzana-Calvo, Marina ;

Fischer, Alain .

MOLECULAR THERAPY, 2006, 13 :S254-S255

← 1 2 3 4 →