Current prospects for mRNA gene delivery

A Yamamoto, M Kormann, J Rosenecker… - European Journal of …, 2009 - Elsevier
Replication-deficient viruses have been used most successfully in the field of gene therapy
because of their high transfection efficiency. However, the risk of insertional mutagenesis …

An insight into non-integrative gene delivery approaches to generate transgene-free induced pluripotent stem cells

KK Haridhasapavalan, MP Borgohain, C Dey, B Saha… - Gene, 2019 - Elsevier
Over a decade ago, a landmark study that reported derivation of induced Pluripotent Stem
Cells (iPSCs) by reprogramming fibroblasts has transformed stem cell research attracting …

Efficient induction of transgene-free human pluripotent stem cells using a vector based on Sendai virus, an RNA virus that does not integrate into the host genome

N Fusaki, H Ban, A Nishiyama, K Saeki… - Proceedings of the …, 2009 - jstage.jst.go.jp
Induced pluripotent stem cells (iPSC) have been generated from somatic cells by
introducing reprogramming factors. Integration of foreign genes into the host genome is a …

Direct in vivo reprogramming with Sendai virus vectors improves cardiac function after myocardial infarction

K Miyamoto, M Akiyama, F Tamura, M Isomi… - Cell stem cell, 2018 - cell.com
Direct cardiac reprogramming holds great promise for regenerative medicine. We previously
generated directly reprogrammed induced cardiomyocyte-like cells (iCMs) by …

Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis

EWFW Alton, JM Beekman, AC Boyd, J Brand… - Thorax, 2017 - thorax.bmj.com
We have recently shown that non-viral gene therapy can stabilise the decline of lung
function in patients with cystic fibrosis (CF). However, the effect was modest, and more …

Induced pluripotent stem cells: reprogramming platforms and applications in cell replacement therapy

A Al Abbar, SC Ngai, N Nograles, SY Alhaji… - BioResearch open …, 2020 - liebertpub.com
The generation of induced pluripotent stem cells (iPSCs) from differentiated mature cells is
one of the most promising technologies in the field of regenerative medicine. The ability to …

Cystic fibrosis gene therapy in the UK and elsewhere

U Griesenbach, KM Pytel, EWFW Alton - Human gene therapy, 2015 - liebertpub.com
The cystic fibrosis transmembrane conductance regulator (CFTR) gene was identified in
1989. This opened the door for the development of cystic fibrosis (CF) gene therapy, which …

Toward gene therapy for cystic fibrosis using a lentivirus pseudotyped with Sendai virus envelopes

K Mitomo, U Griesenbach, M Inoue, L Somerton… - Molecular therapy, 2010 - cell.com
Gene therapy for cystic fibrosis (CF) is making encouraging progress into clinical trials.
However, further improvements in transduction efficiency are desired. To develop a novel …

Generation of integration-free and region-specific neural progenitors from primate fibroblasts

J Lu, H Liu, CTL Huang, H Chen, Z Du, Y Liu… - Cell reports, 2013 - cell.com
Postnatal and adult human and monkey fibroblasts were infected with Sendai virus
containing the Yamanaka factors for 24 hr, then they were cultured in a chemically defined …

Gene transfer to the lung: lessons learned from more than 2 decades of CF gene therapy

U Griesenbach, EWFW Alton… - Advanced drug delivery …, 2009 - Elsevier
Gene therapy is currently being developed for a wide range of acute and chronic lung
diseases. The target cells, and to a degree the extra and intra-cellular barriers, are disease …