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 …
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
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 …
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 …
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 …
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 …
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
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 …
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 …
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 …
However, further improvements in transduction efficiency are desired. To develop a novel …
Generation of integration-free and region-specific neural progenitors from primate fibroblasts
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 …
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 …
diseases. The target cells, and to a degree the extra and intra-cellular barriers, are disease …