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Viral vector platforms within the gene therapy landscape
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 …
transitions. It has seen great strides in combating human disease, has given hope to patients …
Lentiviral vectors: basic to translational
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 …
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
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 …
passage across blood and central nervous system (CNS) compartments. Despite its critical …
T cell receptor gene therapy for cancer
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 …
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
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 …
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
Transcription through immunoglobulin switch (S) regions is essential for class switch
recombination (CSR), but no molecular function of the transcripts has been described …
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
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 …
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 …
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 …
We elucidate the cellular and molecular kinetics of the stepwise differentiation of human
embryonic stem cells (hESCs) to primitive and definitive erythromyelopoiesis from human …
embryonic stem cells (hESCs) to primitive and definitive erythromyelopoiesis from human …
Kruppel-like factor 4 is essential for inflammatory monocyte differentiation in vivo
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 …
important roles in differentiation, survival, and trafficking of blood and immune cell types. We …