Ontology highlight
ABSTRACT:
SUBMITTER: Eckardt S
PROVIDER: S-EPMC3026741 | biostudies-literature | 2011 Feb
REPOSITORIES: biostudies-literature
Eckardt Sigrid S Leu N Adrian NA Yanchik Ashley A Hatada Seigo S Kyba Michael M McLaughlin K John KJ
The Journal of clinical investigation 20110201 2
To be of therapeutic use, autologous stem cells derived from patients with inherited genetic disorders require genetic modification via gene repair or insertion. Here, we present proof of principle that, for diseases associated with dominant alleles (gain-of-function or haploinsufficient loss-of-function), disease allele–free ES cells can be derived from afflicted individuals without genome manipulation. This approach capitalizes on the derivation of uniparental cells, such as parthenogenetic (P ...[more]