Gene therapy simplified
J Ren, DF Stroncek - Blood, The Journal of the American …, 2016 - ashpublications.org
J Ren, DF Stroncek
Blood, The Journal of the American Society of Hematology, 2016•ashpublications.orgIn this issue of Blood, Richter et al report their work on an in vivo gene transduction system
using the adenovirus-based vector and hyperactive SB transposase (SB1003) system. Their
results show that this system is effective and safe and performs without needing ex vivo
expansion and transduction of hematopoietic stem cells (HSCs). 1 This system may
overcome some of the difficulties associated with cell collection and manufacturing and
provide technical advances for the field of gene therapy.
using the adenovirus-based vector and hyperactive SB transposase (SB1003) system. Their
results show that this system is effective and safe and performs without needing ex vivo
expansion and transduction of hematopoietic stem cells (HSCs). 1 This system may
overcome some of the difficulties associated with cell collection and manufacturing and
provide technical advances for the field of gene therapy.
In this issue of Blood, Richter et al report their work on an in vivo gene transduction system using the adenovirus-based vector and hyperactive SB transposase (SB1003) system. Their results show that this system is effective and safe and performs without needing ex vivo expansion and transduction of hematopoietic stem cells (HSCs). 1 This system may overcome some of the difficulties associated with cell collection and manufacturing and provide technical advances for the field of gene therapy.
ashpublications.org