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A Comprehensive Repository of AATD Patient-Specific Induced Pluripotent Stem Cells with Detailed Clinical Phenotyping for Translational Research
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A7127 - A Comprehensive Repository of AATD Patient-Specific Induced Pluripotent Stem Cells with Detailed Clinical Phenotyping for Translational Research
Author Block: A. A. Wilson, J. E. Kaserman, K. J. Hurley, M. Dodge, M. Higgins, E. Meninno, J. C. Jean, M. F. James, G. Mostoslavsky, D. N. Kotton; Center for Regenerative Medicine (CReM), Boston Univ Sch of Med, Boston, MA, United States.
Since their discovery in 2006, induced pluripotent stem cells (iPSCs) have rapidly proven to be a valuable translational research tool. iPSCs provide researchers with an unlimited supply of patient-derived cells with the capacity to differentiate into cell types relevant to diseases of interest, including lung and liver cells. Recent literature from our lab and others has demonstrated the capacity of the differentiated progeny of patient-iPSCs to recapitulate the salient biological features of alpha-1 antitrypsin deficiency (AATD), the monogenic cause of COPD and hepatic cirrhosis, at the cellular level. Additional published reports indicate that AATD patient primary cells including differentiated iPSCs exhibit in vitro phenotypes that mirror the in vivo disease phenotype of the cell donor, underscoring their potential application to model the patient-to-patient variability that is commonly observed clinically. A repository of AATD patient iPSCs that are linked to detailed clinical data will be an essential resource if we are to leverage the potential of this translational model system. To address this need, we have generated a large repository comprised of samples from a total of >100 patients representing a large range of AATD genotypes, including ZZ, SZ, MZ, MS, SS, and rare allele combinations with associated clinical data. From these samples, we have generated and characterized iPSCs from 28 individuals. We have further characterized a subset of these lines to define differentiation efficiency and global transcriptomic profiling of iPSC-derived lung and liver, the cell types affected in AATD. We anticipate the application of this repository and its constituent lines to advance translational AATD studies and have made them freely available to other academic researchers to accomplish this goal.
Author Block: A. A. Wilson, J. E. Kaserman, K. J. Hurley, M. Dodge, M. Higgins, E. Meninno, J. C. Jean, M. F. James, G. Mostoslavsky, D. N. Kotton; Center for Regenerative Medicine (CReM), Boston Univ Sch of Med, Boston, MA, United States.
Since their discovery in 2006, induced pluripotent stem cells (iPSCs) have rapidly proven to be a valuable translational research tool. iPSCs provide researchers with an unlimited supply of patient-derived cells with the capacity to differentiate into cell types relevant to diseases of interest, including lung and liver cells. Recent literature from our lab and others has demonstrated the capacity of the differentiated progeny of patient-iPSCs to recapitulate the salient biological features of alpha-1 antitrypsin deficiency (AATD), the monogenic cause of COPD and hepatic cirrhosis, at the cellular level. Additional published reports indicate that AATD patient primary cells including differentiated iPSCs exhibit in vitro phenotypes that mirror the in vivo disease phenotype of the cell donor, underscoring their potential application to model the patient-to-patient variability that is commonly observed clinically. A repository of AATD patient iPSCs that are linked to detailed clinical data will be an essential resource if we are to leverage the potential of this translational model system. To address this need, we have generated a large repository comprised of samples from a total of >100 patients representing a large range of AATD genotypes, including ZZ, SZ, MZ, MS, SS, and rare allele combinations with associated clinical data. From these samples, we have generated and characterized iPSCs from 28 individuals. We have further characterized a subset of these lines to define differentiation efficiency and global transcriptomic profiling of iPSC-derived lung and liver, the cell types affected in AATD. We anticipate the application of this repository and its constituent lines to advance translational AATD studies and have made them freely available to other academic researchers to accomplish this goal.
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