The past, present, and future of non-viral CAR T cells

A Moretti, M Ponzo, CA Nicolette… - Frontiers in …, 2022 - frontiersin.org
Adoptive transfer of chimeric antigen receptor (CAR) T lymphocytes is a powerful technology
that has revolutionized the way we conceive immunotherapy. The impressive clinical results …

Non-viral gene therapy: Gains and challenges of non-invasive administration methods

M Foldvari, DW Chen, N Nafissi, D Calderon… - Journal of Controlled …, 2016 - Elsevier
Gene therapy is becoming an influential part of the rapidly increasing armamentarium of
biopharmaceuticals for improving health and combating diseases. Currently, three gene …

CARAMBA: a first-in-human clinical trial with SLAMF7 CAR-T cells prepared by virus-free Sleeping Beauty gene transfer to treat multiple myeloma

S Prommersberger, M Reiser, J Beckmann, S Danhof… - Gene therapy, 2021 - nature.com
Clinical development of chimeric antigen receptor (CAR)-T-cell therapy has been enabled
by advances in synthetic biology, genetic engineering, clinical-grade manufacturing, and …

Life-threatening viral disease in a novel form of autosomal recessive IFNAR2 deficiency in the Arctic

CJA Duncan, MK Skouboe, S Howarth… - Journal of Experimental …, 2022 - rupress.org
Type I interferons (IFN-I) play a critical role in human antiviral immunity, as demonstrated by
the exceptionally rare deleterious variants of IFNAR1 or IFNAR2. We investigated five …

Enhanced CAR T-cell engineering using non-viral Sleeping Beauty transposition from minicircle vectors

R Monjezi, C Miskey, T Gogishvili, M Schleef… - Leukemia, 2017 - nature.com
Immunotherapy with T cell modified with gamma-retroviral or lentiviral (LV) vectors to
express a chimeric antigen receptor (CAR) has shown remarkable efficacy in clinical trials …

miRNA sponges: soaking up miRNAs for regulation of gene expression

RO Bak, JG Mikkelsen - Wiley interdisciplinary reviews: RNA, 2014 - Wiley Online Library
MicroRNAs (miRNAs) are small regulatory RNAs that act in an entangled web of interactions
with target mRNAs to shape the cellular protein landscape by post‐transcriptional control of …

Gene therapy with the sleeping beauty transposon system

P Kebriaei, Z Izsvák, SA Narayanavari, H Singh… - Trends in Genetics, 2017 - cell.com
The widespread clinical implementation of gene therapy requires the ability to stably
integrate genetic information through gene transfer vectors in a safe, effective, and …

Non-viral vectors for chimeric antigen receptor immunotherapy

US Tretbar, JG Rurik, EH Rustad, D Sürün… - Nature Reviews …, 2024 - nature.com
Chimeric antigen receptor (CAR) T cell therapy has demonstrated remarkable efficacy in
haematological malignancies. However, although viral vector-based methods are effective …

Retroviral vectors and transposons for stable gene therapy: advances, current challenges and perspectives

JE Vargas, L Chicaybam, RT Stein, A Tanuri… - Journal of translational …, 2016 - Springer
Gene therapy protocols require robust and long-term gene expression. For two decades,
retrovirus family vectors have offered several attractive properties as stable gene-delivery …

Going non-viral: the Sleeping Beauty transposon system breaks on through to the clinical side

M Hudecek, Z Izsvák, S Johnen, M Renner… - Critical reviews in …, 2017 - Taylor & Francis
Molecular medicine has entered a high-tech age that provides curative treatments of
complex genetic diseases through genetically engineered cellular medicinal products. Their …