T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years

R. Michael Blaese; Kenneth W. Culver; A. Dusty Miller; Charles S. Carter; Thomas Fleisher; Mario Clerici; Gene Shearer; Lauren Chang; Yawen Chiang; Paul Tolstoshev; Jay J. Greenblatt; Steven A. Rosenberg; Harvey Klein; Melvin Berger; Craig A. Mullen; W. Jay Ramsey; Linda Muul; Richard A. Morgan; W. French Anderson

doi:10.1126/science.270.5235.475

T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years

R. Michael Blaese, Kenneth W. Culver, A. Dusty Miller, Charles S. Carter, Thomas Fleisher, Mario Clerici, Gene Shearer, Lauren Chang, Yawen Chiang, Paul Tolstoshev, Jay J. Greenblatt, Steven A. Rosenberg, Harvey Klein, Melvin Berger, Craig A. Mullen, W. Jay Ramsey, Linda Muul, Richard A. Morgan, W. French Anderson

Research output: Contribution to journal › Article › peer-review

1094 Scopus citations

Abstract

In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase (ADA) gene into the T cells of two children with severe combined immunodeficiency (ADA- SCID). The number of blood T cells normalized as did many cellular and humoral immune responses. Gene treatment ended after 2 years, but integrated vector and ADA gene expression in T cells persisted. Although many components remain to be perfected, it is concluded here that gene therapy can be a safe and effective addition to treatment for some patients with this severe immunodeficiency disease.

Original language	English (US)
Pages (from-to)	475-480
Number of pages	6
Journal	Science
Volume	270
Issue number	5235
DOIs	https://doi.org/10.1126/science.270.5235.475
State	Published - 1995
Externally published	Yes

ASJC Scopus subject areas

General

Access to Document

10.1126/science.270.5235.475

Cite this

Blaese, R. M., Culver, K. W., Miller, A. D., Carter, C. S., Fleisher, T., Clerici, M., Shearer, G., Chang, L., Chiang, Y., Tolstoshev, P., Greenblatt, J. J., Rosenberg, S. A., Klein, H., Berger, M., Mullen, C. A., Ramsey, W. J., Muul, L., Morgan, R. A., & Anderson, W. F. (1995). T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years. Science, 270(5235), 475-480. https://doi.org/10.1126/science.270.5235.475

Blaese, RM, Culver, KW, Miller, AD, Carter, CS, Fleisher, T, Clerici, M, Shearer, G, Chang, L, Chiang, Y, Tolstoshev, P, Greenblatt, JJ, Rosenberg, SA, Klein, H, Berger, M, Mullen, CA, Ramsey, WJ, Muul, L, Morgan, RA & Anderson, WF 1995, 'T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years', Science, vol. 270, no. 5235, pp. 475-480. https://doi.org/10.1126/science.270.5235.475

@article{0da3977970cd43dc9180d7ff23d25985,

title = "T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years",

abstract = "In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase (ADA) gene into the T cells of two children with severe combined immunodeficiency (ADA- SCID). The number of blood T cells normalized as did many cellular and humoral immune responses. Gene treatment ended after 2 years, but integrated vector and ADA gene expression in T cells persisted. Although many components remain to be perfected, it is concluded here that gene therapy can be a safe and effective addition to treatment for some patients with this severe immunodeficiency disease.",

author = "Blaese, {R. Michael} and Culver, {Kenneth W.} and Miller, {A. Dusty} and Carter, {Charles S.} and Thomas Fleisher and Mario Clerici and Gene Shearer and Lauren Chang and Yawen Chiang and Paul Tolstoshev and Greenblatt, {Jay J.} and Rosenberg, {Steven A.} and Harvey Klein and Melvin Berger and Mullen, {Craig A.} and Ramsey, {W. Jay} and Linda Muul and Morgan, {Richard A.} and Anderson, {W. French}",

year = "1995",

doi = "10.1126/science.270.5235.475",

language = "English (US)",

volume = "270",

pages = "475--480",

journal = "Science",

issn = "0036-8075",

publisher = "American Association for the Advancement of Science",

number = "5235",

}

TY - JOUR

T1 - T lymphocyte-directed gene therapy for ADA-SCID

T2 - Initial trial results after 4 years

AU - Blaese, R. Michael

AU - Culver, Kenneth W.

AU - Miller, A. Dusty

AU - Carter, Charles S.

AU - Fleisher, Thomas

AU - Clerici, Mario

AU - Shearer, Gene

AU - Chang, Lauren

AU - Chiang, Yawen

AU - Tolstoshev, Paul

AU - Greenblatt, Jay J.

AU - Rosenberg, Steven A.

AU - Klein, Harvey

AU - Berger, Melvin

AU - Mullen, Craig A.

AU - Ramsey, W. Jay

AU - Muul, Linda

AU - Morgan, Richard A.

AU - Anderson, W. French

PY - 1995

Y1 - 1995

N2 - In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase (ADA) gene into the T cells of two children with severe combined immunodeficiency (ADA- SCID). The number of blood T cells normalized as did many cellular and humoral immune responses. Gene treatment ended after 2 years, but integrated vector and ADA gene expression in T cells persisted. Although many components remain to be perfected, it is concluded here that gene therapy can be a safe and effective addition to treatment for some patients with this severe immunodeficiency disease.

AB - In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase (ADA) gene into the T cells of two children with severe combined immunodeficiency (ADA- SCID). The number of blood T cells normalized as did many cellular and humoral immune responses. Gene treatment ended after 2 years, but integrated vector and ADA gene expression in T cells persisted. Although many components remain to be perfected, it is concluded here that gene therapy can be a safe and effective addition to treatment for some patients with this severe immunodeficiency disease.

UR - http://www.scopus.com/inward/record.url?scp=0028807727&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=0028807727&partnerID=8YFLogxK

U2 - 10.1126/science.270.5235.475

DO - 10.1126/science.270.5235.475

M3 - Article

C2 - 7570001

AN - SCOPUS:0028807727

SN - 0036-8075

VL - 270

SP - 475

EP - 480

JO - Science

JF - Science

IS - 5235

ER -

T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years

Abstract

ASJC Scopus subject areas

Access to Document

Other files and links

Fingerprint

Cite this