Gene therapy comes of age
BACKGROUND Nearly five decades ago, visionary scientists hypothesized that genetic
modification by exogenous DNA might be an effective treatment for inherited human …
modification by exogenous DNA might be an effective treatment for inherited human …
Hematopoietic stem cell gene therapy: progress and lessons learned
The use of allogeneic hematopoietic stem cells (HSCs) to treat genetic blood cell diseases
has become a clinical standard but is limited by the availability of suitable matched donors …
has become a clinical standard but is limited by the availability of suitable matched donors …
Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease
Gene therapy (GT) provides a potentially curative treatment option for patients with sickle
cell disease (SCD); however, the occurrence of myeloid malignancies in GT clinical trials …
cell disease (SCD); however, the occurrence of myeloid malignancies in GT clinical trials …
Generation of mouse induced pluripotent stem cells without viral vectors
K Okita, M Nakagawa, H Hyenjong, T Ichisaka… - Science, 2008 - science.org
Induced pluripotent stem (iPS) cells have been generated from mouse and human somatic
cells by introducing Oct3/4 and Sox2 with either Klf4 and c-Myc or Nanog and Lin28 using …
cells by introducing Oct3/4 and Sox2 with either Klf4 and c-Myc or Nanog and Lin28 using …
Incorporation of pseudouridine into mRNA enhances translation by diminishing PKR activation
BR Anderson, H Muramatsu, SR Nallagatla… - Nucleic acids …, 2010 - academic.oup.com
Previous studies have shown that the translation level of in vitro transcribed messenger RNA
(mRNA) is enhanced when its uridines are replaced with pseudouridines; however, the …
(mRNA) is enhanced when its uridines are replaced with pseudouridines; however, the …
Generation of pluripotent stem cells from adult mouse liver and stomach cells
T Aoi, K Yae, M Nakagawa, T Ichisaka, K Okita… - Science, 2008 - science.org
Induced pluripotent stem (iPS) cells have been generated from mouse and human
fibroblasts by the retroviral transduction of four transcription factors. However, the cell origins …
fibroblasts by the retroviral transduction of four transcription factors. However, the cell origins …
Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery
Achieving the full potential of zinc-finger nucleases (ZFNs) for genome engineering in
human cells requires their efficient delivery to the relevant cell types. Here we exploited the …
human cells requires their efficient delivery to the relevant cell types. Here we exploited the …
Multiple injections of electroporated autologous T cells expressing a chimeric antigen receptor mediate regression of human disseminated tumor
Redirecting T lymphocyte antigen specificity by gene transfer can provide large numbers of
tumor-reactive T lymphocytes for adoptive immunotherapy. However, safety concerns …
tumor-reactive T lymphocytes for adoptive immunotherapy. However, safety concerns …
Gene delivery by lentivirus vectors
AS Cockrell, T Kafri - Molecular biotechnology, 2007 - Springer
The capacity to efficiently transduce nondividing cells, shuttle large genetic payloads, and
maintain stable long-term transgene expression are attributes that have brought lentiviral …
maintain stable long-term transgene expression are attributes that have brought lentiviral …
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 …