To restore Dystrophin gene, exon 44 was inserted in front of the exon 45 via TALEN mediated homologous recombination. Hygromycin resistance cassette was removed by Cre recombinase, hence a single loxP sequence remain in intron 44.
Disease specific iPS cell line derived from a patient : Muscular dystrophy, Duchenne type. Correction of the dystrophin gene. HPS0383, HPS0384, HPS0385 and HPS0387 were derived from the same patient.
Disease specific iPS cell line derived from a patient : Muscular dystrophy, Duchenne type. Correction of the dystrophin gene. HPS0383, HPS0384, HPS0385 and HPS0387 were derived from the same patient. Order Form (C-0042, C-0057, C-0007).
1) The BIOLOGICAL RESOURCE must be used for academic research purpose conducted by RECIPIENT who belongs to a not-for profit academic organization (i.e. a university or another institution of higher education or any non-profit scientific or educational organization, including government agencies) . 2) The RECIPIENT agrees to obtain the prior written approval of the distribution from the APPROVER. 3) Contact Us : <a href="mailto:cellips.brc@riken.jp?subject=Disease specific iPS cell line&">RIKEN Cell Bank</a>
HPS0386
全長型DYSTROPHIN遺伝子を回復するために、TALENを用いてエクソン45の直 前にエクソン44をノックイン。ハイグロマイシン選択カセットはCreで除去済み。 イントロン44にIoxP配列が一つ残存。
疾患特異的iPS細胞株。デュシェンヌ型筋ジストロフィー患者由来。Dystrophin遺伝子を修復。HPS0383、HPS0384、HPS0385、HPS0387と同一人由来。エピソーマルベクターにより6因子（Oct3/4, Sox2, Klf4, L-Myc, Lin28, p53 shRNA）を導入して樹立。<a href='http://cell.brc.riken.jp/ja/manual/vitrification' target="_blank">ガラス化法（Vitrification法）</a>保存。
1) 非営利の団体に所属する利用者による学術研究での使用に限る。 2) 利用者は、事前に承諾者から承諾を得ること。 3) 先ずは、<a href="mailto:cellips.brc@riken.jp?subject=Disease specific iPS cell line 問い合わせ&">理研細胞バンク</a>にお問い合わせください 。
<a href="http://www.cira.kyoto-u.ac.jp/" target="_blank">http://www.cira.kyoto-u.ac.jp/</a>
疾患特異的iPS細胞株。デュシェンヌ型筋ジストロフィー患者由来。Dystrophin遺伝子を修復。HPS0383、HPS0384、HPS0385、HPS0387と同一人由来。エピソーマルベクターにより6因子（Oct3/4, Sox2, Klf4, L-Myc, Lin28, p53 shRNA）を導入して樹立。<a href='https://cell.brc.riken.jp/ja/manual/vitrification' target="_blank">ガラス化法（Vitrification法）</a>保存。
CiRA00111-TKI-I15