Viral vector platforms within the gene therapy landscape

JT Bulcha, Y Wang, H Ma, PWL Tai… - Signal transduction and …, 2021 - nature.com
Throughout its 40-year history, the field of gene therapy has been marked by many
transitions. It has seen great strides in combating human disease, has given hope to patients …

Lentiviral vectors: basic to translational

T Sakuma, MA Barry, Y Ikeda - Biochemical Journal, 2012 - portlandpress.com
More than two decades have passed since genetically modified HIV was used for gene
delivery. Through continuous improvements these early marker gene-carrying HIVs have …

Modeling blood-brain barrier formation and cerebral cavernous malformations in human PSC-derived organoids

L Dao, Z You, L Lu, T Xu, AK Sarkar, H Zhu, M Liu… - Cell Stem Cell, 2024 - cell.com
The human blood-brain barrier (hBBB) is a highly specialized structure that regulates
passage across blood and central nervous system (CNS) compartments. Despite its critical …

T cell receptor gene therapy for cancer

TM Schmitt, GB Ragnarsson, PD Greenberg - Human gene therapy, 2009 - liebertpub.com
T cell-based adoptive immunotherapy has been shown to be a promising treatment for
various types of cancer. However, adoptive T cell therapy currently requires the custom …

CD34+ hematopoietic stem-progenitor cell microRNA expression and function: a circuit diagram of differentiation control

RW Georgantas III, R Hildreth, S Morisot, J Alder… - Proceedings of the …, 2007 - pnas.org
MicroRNAs (miRNAs) are a recently identified class of epigenetic elements consisting of
small noncoding RNAs that bind to the 3′ untranslated region of mRNAs and down …

Non-coding RNA generated following lariat debranching mediates targeting of AID to DNA

S Zheng, BQ Vuong, B Vaidyanathan, JY Lin… - Cell, 2015 - cell.com
Transcription through immunoglobulin switch (S) regions is essential for class switch
recombination (CSR), but no molecular function of the transcripts has been described …

Gene therapy progress and prospects: development of improved lentiviral and retroviral vectors–design, biosafety, and production

PL Sinn, SL Sauter, PB McCray - Gene therapy, 2005 - nature.com
Replication defective vectors derived from simple retroviruses or the more complex
genomes of lentiviruses continue to offer the advantages of long-term expression, cell and …

Exploring precision treatments in immune‐mediated inflammatory diseases: harnessing the infinite potential of nucleic acid delivery

L Xu, Z Shao, X Fang, Z **n, S Zhao, H Zhang… - …, 2024 - Wiley Online Library
Immune‐mediated inflammatory diseases (IMIDs) impose an immeasurable burden on
individuals and society. While the conventional use of immunosuppressants and disease …

Hematopoietic differentiation of human embryonic stem cells progresses through sequential hematoendothelial, primitive, and definitive stages resembling human …

ET Zambidis, B Peault, TS Park, F Bunz, CI Civin - Blood, 2005 - ashpublications.org
We elucidate the cellular and molecular kinetics of the stepwise differentiation of human
embryonic stem cells (hESCs) to primitive and definitive erythromyelopoiesis from human …

Kruppel-like factor 4 is essential for inflammatory monocyte differentiation in vivo

JK Alder, RW Georgantas, RL Hildreth… - The Journal of …, 2008 - journals.aai.org
Several members of the Kruppel-like factor (KLF) family of transcription factors play
important roles in differentiation, survival, and trafficking of blood and immune cell types. We …