[HTML][HTML] Viral expression cassette elements to enhance transgene target specificity and expression in gene therapy

SK Powell, R Rivera-Soto, SJ Gray - Discovery medicine, 2015 - ncbi.nlm.nih.gov
Over the last five years, the number of clinical trials involving AAV (adeno-associated virus)
and lentiviral vectors continue to increase by about 150 trials each year. For continued …

Synthetic biology advances and applications in the biotechnology industry: a perspective

L Katz, YY Chen, R Gonzalez… - Journal of Industrial …, 2018 - academic.oup.com
Synthetic biology is a logical extension of what has been called recombinant DNA (rDNA)
technology or genetic engineering since the 1970s. As rDNA technology has been the driver …

[HTML][HTML] Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa

SM Lwin, F Syed, WL Di, T Kadiyirire, L Liu, A Guy… - JCI insight, 2019 - ncbi.nlm.nih.gov
BACKGROUND Recessive dystrophic epidermolysis bullosa (RDEB) is a severe form of skin
fragility disorder due to mutations in COL7A1 encoding basement membrane type VII …

A minimal ubiquitous chromatin opening element (UCOE) effectively prevents silencing of juxtaposed heterologous promoters by epigenetic remodeling in multipotent …

U Müller-Kuller, M Ackermann, S Kolodziej… - Nucleic acids …, 2015 - academic.oup.com
Epigenetic silencing of transgene expression represents a major obstacle for the efficient
genetic modification of multipotent and pluripotent stem cells. We and others have …

Chromatin function modifying elements in an industrial antibody production platform-comparison of UCOE, MAR, STAR and cHS4 elements

F Saunders, B Sweeney, MN Antoniou, P Stephens… - PloS one, 2015 - journals.plos.org
The isolation of stably transfected cell lines suitable for the manufacture of biotherapeutic
protein products can be an arduous process relying on the identification of a high …

Detailed comparison of retroviral vectors and promoter configurations for stable and high transgene expression in human induced pluripotent stem cells

D Hoffmann, JW Schott, FK Geis, L Lange, FJ Müller… - Gene Therapy, 2017 - nature.com
Correction of patient-specific induced pluripotent stem cells (iPSC) upon gene delivery
through retroviral vectors offers new treatment perspectives for monogenetic diseases. Gene …

Vectorology and factor delivery in induced pluripotent stem cell reprogramming

K Hu - Stem cells and development, 2014 - liebertpub.com
Induced pluripotent stem cell (iPSC) reprogramming requires sustained expression of
multiple reprogramming factors for a limited period of time (10–30 days). Conventional iPSC …

[HTML][HTML] Ubiquitous chromatin-opening elements (UCOEs): applications in biomanufacturing and gene therapy

JJ Neville, J Orlando, K Mann, B McCloskey… - Biotechnology …, 2017 - Elsevier
Abstract Ubiquitous Chromatin-opening Elements (UCOEs) are defined by their ability to
consistently confer stable, site of integration-independent transgene expression that is …

Neural lineage tracing in the mammalian brain

J Ma, Z Shen, YC Yu, SH Shi - Current opinion in neurobiology, 2018 - Elsevier
Highlights•Cell lineage underlies the developmental steps taken to acquire cell
identity.•Genetic labeling allows lineage tracing with cell type and temporal …

Cellular behavior as a dynamic field for exploring bone bioengineering: a closer look at cell–biomaterial interface

S Gemini-Piperni, ER Takamori, SC Sartoretto… - Archives of Biochemistry …, 2014 - Elsevier
Bone is a highly dynamic and specialized tissue, capable of regenerating itself
spontaneously when afflicted by minor injuries. Nevertheless, when major lesions occur, it …