Molecular Therapy — Nucleic Acids · 2020 · 23 citations · 71 references
Life SciencesBiotechnologyMolecular BiologyGene ExpressionStem Cell BiotechnologySynthetic BiologyRna EngineeringRna InterferenceSmall Interfering Rna BiologySmall Interfering Rna DeliveryOsteogenic DifferentiationOsteogenic MarkersRna Interference TherapeuticsSmall Interfering Ribonucleic Acids (Sirnas)Small Interfering Rna (Sirna) ChemistrySmall Interfering Rna DesignSmall Interfering Rna FunctionSynthetic Small Interfering Ribonucleic Acid Design
ectopic bone formation. Collectively, we demonstrate that the engineered FAMSi system provides a fast-track platform for assembling multiplexed siRNAs in a single vector, and thus it may be a valuable tool to study gene functions or to develop novel siRNA-based therapeutics.
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Multiplex Genome Engineering Using CRISPR/Cas Systems
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