Gene therapy simplified

Blood. 2016 Nov 3;128(18):2194-2195. doi: 10.1182/blood-2016-09-736983.

Abstract

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 (SB100×) 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). This system may overcome some of the difficulties associated with cell collection and manufacturing and provide technical advances for the field of gene therapy.

Publication types

  • Comment

MeSH terms

  • Genetic Therapy*
  • Humans