Disease-Specific Induced Pluripotent Stem Cells

In Hyun Park; Natasha Arora; Hongguang Huo; Nimet Maherali; Tim Ahfeldt; Akiko Shimamura; M. William Lensch; Chad Cowan; Konrad Hochedlinger; George Q. Daley

doi:10.1016/j.cell.2008.07.041

Disease-Specific Induced Pluripotent Stem Cells

In Hyun Park
, Natasha Arora
, Hongguang Huo
, Nimet Maherali
, Tim Ahfeldt
, Akiko Shimamura
, M. William Lensch
, Chad Cowan
, Konrad Hochedlinger
, George Q. Daley

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

1961 Scopus citations

Abstract

Tissue culture of immortal cell strains from diseased patients is an invaluable resource for medical research but is largely limited to tumor cell lines or transformed derivatives of native tissues. Here we describe the generation of induced pluripotent stem (iPS) cells from patients with a variety of genetic diseases with either Mendelian or complex inheritance; these diseases include adenosine deaminase deficiency-related severe combined immunodeficiency (ADA-SCID), Shwachman-Bodian-Diamond syndrome (SBDS), Gaucher disease (GD) type III, Duchenne (DMD) and Becker muscular dystrophy (BMD), Parkinson disease (PD), Huntington disease (HD), juvenile-onset, type 1 diabetes mellitus (JDM), Down syndrome (DS)/trisomy 21, and the carrier state of Lesch-Nyhan syndrome. Such disease-specific stem cells offer an unprecedented opportunity to recapitulate both normal and pathologic human tissue formation in vitro, thereby enabling disease investigation and drug development.

Original language	English
Pages (from-to)	877-886
Number of pages	10
Journal	Cell
Volume	134
Issue number	5
DOIs	https://doi.org/10.1016/j.cell.2008.07.041
State	Published - 5 Sep 2008
Externally published	Yes

Keywords

HUMDISEASE
STEMCELL

Access to Document

10.1016/j.cell.2008.07.041

Cite this

@article{7a52a6c384f6472caf5f9e9d80a5068e,

title = "Disease-Specific Induced Pluripotent Stem Cells",

abstract = "Tissue culture of immortal cell strains from diseased patients is an invaluable resource for medical research but is largely limited to tumor cell lines or transformed derivatives of native tissues. Here we describe the generation of induced pluripotent stem (iPS) cells from patients with a variety of genetic diseases with either Mendelian or complex inheritance; these diseases include adenosine deaminase deficiency-related severe combined immunodeficiency (ADA-SCID), Shwachman-Bodian-Diamond syndrome (SBDS), Gaucher disease (GD) type III, Duchenne (DMD) and Becker muscular dystrophy (BMD), Parkinson disease (PD), Huntington disease (HD), juvenile-onset, type 1 diabetes mellitus (JDM), Down syndrome (DS)/trisomy 21, and the carrier state of Lesch-Nyhan syndrome. Such disease-specific stem cells offer an unprecedented opportunity to recapitulate both normal and pathologic human tissue formation in vitro, thereby enabling disease investigation and drug development.",

keywords = "HUMDISEASE, STEMCELL",

author = "Park, \{In Hyun\} and Natasha Arora and Hongguang Huo and Nimet Maherali and Tim Ahfeldt and Akiko Shimamura and Lensch, \{M. William\} and Chad Cowan and Konrad Hochedlinger and Daley, \{George Q.\}",

note = "Funding Information: G.Q.D. was supported by grants from the National Institutes of Health, the NIH Director's Pioneer Award of the NIH Roadmap for Medical Research, and private funds contributed to the Harvard Stem Cell Institute and the Children's Hospital Stem Cell Program. The work was made possible by the support and vision of Douglas and Diana Berthiaume, Jonathan and Patti Kraft, and the John W. Henry Family Foundation. G.Q.D. is a recipient of Clinical Scientist Awards in Translational Research from the Burroughs Wellcome Fund and the Leukemia and Lymphoma Society and is an Investigator of the Howard Hughes Medical Institute. K.H. was supported by the NIH Director's Innovator Award and the Harvard Stem Cell Institute. N.M. was supported by the Natural Sciences and Engineering Council of Canada and a Sir James Lougheed Award. ",

year = "2008",

month = sep,

day = "5",

doi = "10.1016/j.cell.2008.07.041",

language = "English",

volume = "134",

pages = "877--886",

journal = "Cell",

issn = "0092-8674",

publisher = "Elsevier B.V.",

number = "5",

}

TY - JOUR

T1 - Disease-Specific Induced Pluripotent Stem Cells

AU - Park, In Hyun

AU - Arora, Natasha

AU - Huo, Hongguang

AU - Maherali, Nimet

AU - Ahfeldt, Tim

AU - Shimamura, Akiko

AU - Lensch, M. William

AU - Cowan, Chad

AU - Hochedlinger, Konrad

AU - Daley, George Q.

N1 - Funding Information: G.Q.D. was supported by grants from the National Institutes of Health, the NIH Director's Pioneer Award of the NIH Roadmap for Medical Research, and private funds contributed to the Harvard Stem Cell Institute and the Children's Hospital Stem Cell Program. The work was made possible by the support and vision of Douglas and Diana Berthiaume, Jonathan and Patti Kraft, and the John W. Henry Family Foundation. G.Q.D. is a recipient of Clinical Scientist Awards in Translational Research from the Burroughs Wellcome Fund and the Leukemia and Lymphoma Society and is an Investigator of the Howard Hughes Medical Institute. K.H. was supported by the NIH Director's Innovator Award and the Harvard Stem Cell Institute. N.M. was supported by the Natural Sciences and Engineering Council of Canada and a Sir James Lougheed Award.

PY - 2008/9/5

Y1 - 2008/9/5

N2 - Tissue culture of immortal cell strains from diseased patients is an invaluable resource for medical research but is largely limited to tumor cell lines or transformed derivatives of native tissues. Here we describe the generation of induced pluripotent stem (iPS) cells from patients with a variety of genetic diseases with either Mendelian or complex inheritance; these diseases include adenosine deaminase deficiency-related severe combined immunodeficiency (ADA-SCID), Shwachman-Bodian-Diamond syndrome (SBDS), Gaucher disease (GD) type III, Duchenne (DMD) and Becker muscular dystrophy (BMD), Parkinson disease (PD), Huntington disease (HD), juvenile-onset, type 1 diabetes mellitus (JDM), Down syndrome (DS)/trisomy 21, and the carrier state of Lesch-Nyhan syndrome. Such disease-specific stem cells offer an unprecedented opportunity to recapitulate both normal and pathologic human tissue formation in vitro, thereby enabling disease investigation and drug development.

AB - Tissue culture of immortal cell strains from diseased patients is an invaluable resource for medical research but is largely limited to tumor cell lines or transformed derivatives of native tissues. Here we describe the generation of induced pluripotent stem (iPS) cells from patients with a variety of genetic diseases with either Mendelian or complex inheritance; these diseases include adenosine deaminase deficiency-related severe combined immunodeficiency (ADA-SCID), Shwachman-Bodian-Diamond syndrome (SBDS), Gaucher disease (GD) type III, Duchenne (DMD) and Becker muscular dystrophy (BMD), Parkinson disease (PD), Huntington disease (HD), juvenile-onset, type 1 diabetes mellitus (JDM), Down syndrome (DS)/trisomy 21, and the carrier state of Lesch-Nyhan syndrome. Such disease-specific stem cells offer an unprecedented opportunity to recapitulate both normal and pathologic human tissue formation in vitro, thereby enabling disease investigation and drug development.

KW - HUMDISEASE

KW - STEMCELL

UR - https://www.scopus.com/pages/publications/50549089957

U2 - 10.1016/j.cell.2008.07.041

DO - 10.1016/j.cell.2008.07.041

M3 - Article

C2 - 18691744

AN - SCOPUS:50549089957

SN - 0092-8674

VL - 134

SP - 877

EP - 886

JO - Cell

JF - Cell

IS - 5

ER -

Disease-Specific Induced Pluripotent Stem Cells

Abstract

Keywords

Access to Document

Fingerprint

Cite this