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Synthetic dsRNA Dicer substrates enhance RNAi potency and efficacy
TLDR
These results provide an alternative strategy for eliciting RNAi-mediated target cleavage using low concentrations of synthetic RNA as substrates for cellular Dicer-mediated cleavage.
Axicabtagene Ciloleucel CAR T‐Cell Therapy in Refractory Large B‐Cell Lymphoma
TLDR
Patients with refractory large B‐cell lymphoma who received CAR T‐cell therapy with axi‐cel had high levels of durable response, with a safety profile that included myelosuppression, the cytokine release syndrome, and neurologic events.
Argonaute-1 directs siRNA-mediated transcriptional gene silencing in human cells
TLDR
The results establish a connection between RNAi components AGO1 and TRBP2, RNAPII transcription and Polycomb-regulated control of gene expression, implicating Polycomb silencing in the mechanism of mammalian TGS.
RNAi therapeutics: principles, prospects and challenges.
TLDR
This review discusses mechanistic aspects of RNAi, the potential problem areas and solutions and therapeutic applications, and it is anticipated that RNAi will be a major therapeutic modality within the next several years.
Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells
TLDR
A mammalian Pol III promoter system is described capable of expressing functional double-stranded siRNAs following transfection into human cells and achieving up to 4 logs of inhibition of expression from the HIV-1 DNA.
MicroRNA-192 in diabetic kidney glomeruli and its function in TGF-β-induced collagen expression via inhibition of E-box repressors
TLDR
A role for miRs in the kidney and DN is uncovered in controlling TGF-β-induced Col1a2 expression by down-regulating E-box repressors.
Strategies for silencing human disease using RNA interference
TLDR
This work has highlighted both promise and challenges in using RNAi for therapeutic applications and suggests design and delivery strategies for RNAi effector molecules must be carefully considered to address safety concerns and to ensure effective, successful treatment of human diseases.
Unlocking the potential of the human genome with RNA interference
TLDR
RNAi has revolutionized approaches to decoding gene function and has the potential to be exploited therapeutically, and clinical trials to test this possibility are already being planned.
MicroRNA-directed transcriptional gene silencing in mammalian cells
TLDR
The results suggest the existence of an epigenetic mechanism of miRNA-directed transcriptional gene silencing (TGS) in mammalian cells.
Approaches for the sequence-specific knockdown of mRNA
TLDR
The major classes of antisense agents currently used by investigators for sequence-specific mRNA knockdowns are antisense oligonucleotides, ribozymes, DNAzymes and RNA interference (RNAi).
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