Gene insertion into genomic safe harbors for human gene therapy
EP Papapetrou, A Schambach - Molecular Therapy, 2016 - cell.com
Genomic safe harbors (GSHs) are sites in the genome able to accommodate the integration
of new genetic material in a manner that ensures that the newly inserted genetic elements:(i) …
of new genetic material in a manner that ensures that the newly inserted genetic elements:(i) …
Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity
Virus-based vectors are widely used in hematopoietic stem cell (HSC) gene therapy, and
have the ability to integrate permanently into genomic DNA, thus driving long-term …
have the ability to integrate permanently into genomic DNA, thus driving long-term …
Vascular niche promotes hematopoietic multipotent progenitor formation from pluripotent stem cells
Pluripotent stem cells (PSCs) represent an alternative hematopoietic stem cell (HSC) source
for treating hematopoietic disease. The limited engraftment of human PSC–derived (hPSC …
for treating hematopoietic disease. The limited engraftment of human PSC–derived (hPSC …
An inducible caspase-9 suicide gene to improve the safety of therapy using human induced pluripotent stem cells
Human induced pluripotent stem cells (hiPSC) hold promise for regenerative therapies,
though there are several safety concerns including the risk of oncogenic transformation or …
though there are several safety concerns including the risk of oncogenic transformation or …
A safeguard system for induced pluripotent stem cell-derived rejuvenated T cell therapy
M Ando, T Nishimura, S Yamazaki, T Yamaguchi… - Stem cell reports, 2015 - cell.com
The discovery of induced pluripotent stem cells (iPSCs) has created promising new avenues
for therapies in regenerative medicine. However, the tumorigenic potential of …
for therapies in regenerative medicine. However, the tumorigenic potential of …
Path to the clinic: assessment of iPSC-based cell therapies in vivo in a nonhuman primate model
Induced pluripotent stem cell (iPSC)-based cell therapies have great potential for
regenerative medicine but are also potentially associated with tumorigenic risks. Current …
regenerative medicine but are also potentially associated with tumorigenic risks. Current …
Preventing pluripotent cell teratoma in regenerative medicine applied to hematology disorders
A Bedel, F Beliveau, I Lamrissi-Garcia… - Stem cells …, 2017 - academic.oup.com
Iatrogenic tumorigenesis is a major limitation for the use of human induced pluripotent stem
cells (hiPSCs) in hematology. The teratoma risk comes from the persistence of hiPSCs in …
cells (hiPSCs) in hematology. The teratoma risk comes from the persistence of hiPSCs in …
Preclinical studies for induced pluripotent stem cell-based therapeutics
J Harding, O Mirochnitchenko - Journal of Biological Chemistry, 2014 - ASBMB
Induced pluripotent stem cells (iPSCs) and their differentiated derivatives can potentially be
applied to cell-based therapy for human diseases. The properties of iPSCs are being …
applied to cell-based therapy for human diseases. The properties of iPSCs are being …
Development of an inducible caspase-9 safety switch for pluripotent stem cell–based therapies
Induced pluripotent stem cell (iPSC) therapies offer a promising path for patient-specific
regenerative medicine. However, tumor formation from residual undifferentiated iPSC or …
regenerative medicine. However, tumor formation from residual undifferentiated iPSC or …
Protecting against wayward human induced pluripotent stem cells with a suicide gene
F Cheng, Q Ke, F Chen, B Cai, Y Gao, C Ye, D Wang… - Biomaterials, 2012 - Elsevier
The generation of human induced pluripotent stem cells (hiPSCs) opens a prospect for
regenerative medicine. However, transplantation of somatic cells derived from hiPSCs still …
regenerative medicine. However, transplantation of somatic cells derived from hiPSCs still …