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丁香实验推荐阅读
Gene Modification in Embryonic Stem Cells by Single-Stranded DNA Oligonucleotides

Oligonucleotide-mediated gene targeting is an attractive alternative to current procedures to subtly modify the genome of mouse embryonic stem (ES) cells. However, oligonucleotide-directed substitution, insertion or deletion of a single or a few nucleotides was hampered by DNA m ...

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Chromosome Engineering in ES Cells

Chromosomal rearrangements, such as deletions, duplications, inversions and translocations, occur frequently in humans and can be disease-associated or phenotypically neutral. To understand the genetic consequences of such genomic changes, these mutations need to be mode ...

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Gene-Trap Vectors and Mutagenesis

Gene trapping can be used to introduce insertional mutations into the genome of mouse embryonic stem cells (ESCs). The method has been adapted for high-throughput use, in an effort to inactivate all genes in the mouse genome. Gene trapping is performed with vectors that simultaneously inacti ...

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Overview on Mouse Mutagenesis

In this chapter we give an overview of mutagenesis methods in the mouse as they evolved over the last two decades, an outlook of ongoing and future developments and advice for choosing a mutagenesis strategy. Where appropriate, reference is given to relevant chapters of this book, key original art ...

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Delivery of DNA to Lung Airway Epithelium

Delivering exogenous DNA or genes directly to the lung airways offers a unique and appealing opportunity for specifically targeting gene expression to airway and alveolar epithelium. A large body of literature and experience supports the feasibility of this approach. However, airw ...

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Delivery of Adenoviral DNA to Mouse Liver

The liver represents a major target organ for gene delivery owing to its high biosynthetic capacity and access to the bloodstream. Adenoviral vectors are highly efficient gene-transfer vehicles, making them among the most promising systems for in vivo gene transfer to the liver. Following ...

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DNA Delivery to Cells in Culture: Generation of Adenoviral Libraries for High-Throughput Functional Screening

In functional genomics, the use of expression libraries of DNA variants in combination with potent screening techniques is a powerful tool for gene discovery. They allow study of gene and protein function, generation of peptide variants with novel properties, as well as identification of f ...

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Adenovirus-Mediated Gene Delivery: An Overview

Adenoviruses, which were first isolated in the 1950s, have been developed as gene-delivery vehicles, or vectors, since the early 1980s (1). The adenoviruses constitute the Adenoviridae family, which is divided into two genera: the Aviadenovirus genus infects only birds, whereas the Mast ...

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Delivery of DNA to Pulmonary Endothelium Using Adenoviral Vectors

Delivery of genes to the pulmonary vascular endothelium is a rational approach for the investigation and potential therapy of pulmonary vascular diseases. Furthermore, in view of the exposure of this vascular bed to the entire cardiac output, this technique could be used as an efficient bas ...

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Alternative Splicing as a Therapeutic Target for Human Diseases

The majority of eukaryotic genes undergo alternative splicing, an evolutionarily conserved phenomenon, to generate functionally diverse protein isoforms from a single transcript. The fact that defective pre-mRNA splicing can generate non-functional and often toxic prote ...

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Development and Application of a Dual-Purpose Nanoparticle Platform for Delivery and Imaging of siRNA in Tumors

The vision of using a single therapeutic agent with sufficient generality to allow application to a wide variety of diseases, yet specific enough to permit intervention at single molecular stages of the pathology, is rapidly becoming a reality through the emergence of RNA interference. RNA ...

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Targeted Gene Silencing into Solid Tumors with Electrically Mediated siRNA Delivery

Short interfering RNAs (siRNAs) represent new potential therapeutic tools, owing to their capacity to induce strong, sequence-specific gene silencing in cells. However, their clinical development requires new, safe, and efficient in vivo siRNA delivery methods. In this study, we rep ...

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Liposomal siRNA for Ovarian Cancer

Discovery of RNA interference (RNAi) has been one of the most important findings in the last ten years. In recent years, small interfering RNA (siRNA)-mediated gene silencing is beginning to show substantial promise as a new treatment modality in preclinical studies because of its robust gene ...

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Therapeutic Applications of RNAi for Silencing Virus Replication

RNA interference (RNAi) is an evolutionarily conserved gene-silencing mechanism in which small 19–23-nucleotide double-stranded RNA molecules, or small interfering RNAs (siRNAs), target cognate RNA for destruction with exquisite potency and selectivity. The RNAi machine ...

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RNAi in the Malaria Vector, Anopheles gambiae

Malaria is a disease that kills more than a million people each year in tropical and subtropical countries. The disease is caused by Plasmodium parasites and is transmitted to humans exclusively by mosquitoes of the genus Anopheles. The lack of functional approaches has hampered study of the bi ...

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RNAi Using a Chitosan/siRNA Nanoparticle System: In Vitro and In Vivo Applications

Delivery is a key issue in development of clinically relevant RNAi therapeutics. Polymeric nanoparticles formed by self-assembly of polycations with siRNA can be used for extracellular delivery, cellular uptake and intracellular trafficking as a strategy to improve the therape ...

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Lentiviral and Adeno-Associated Vector-Based Therapy for Motor Neuron Disease Through RNAi

RNAi holds promise for neurodegenerative disorders caused by gain-of-function mutations. We and others have demonstrated proof-of-principle for viral-mediated RNAi in a mouse model of motor neuron disease. Lentivirus and adeno-associated virus have been used to knockdown leve ...

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Studying Autoimmunity by In Vivo RNA Interference

The occurrence of autoimmunity is strongly associated with multiple gene variants that predispose individuals to disease. The identification of the gene polymorphisms that modulate disease susceptibility is key to our understanding of disease etiology and pathogenesis. Whi ...

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Transcriptional Gene Silencing Using Small RNAs

RNA interference is a potent gene silencing pathway initiated by short molecules of double-stranded RNA. Small interfering RNAs (siRNAs) with full sequence complementarity to mRNAs induce cleavage of their target transcripts in the cytoplasm. Recent evidence has shown, however, th ...

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Sybil: Methods and Software for Multiple Genome Comparison and Visualization

With the successful completion of genome sequencing projects for a variety of model organisms, the selection of candidate organisms for future sequencing efforts has been guided increasingly by a desire to enable comparative genomics. This trend has both depended on and encouraged the d ...

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