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Protocol
Molecular Beacons for Protein—DNA Interaction Studies
Real-time monitoring of DNA—protein interactions involving molecular beacon (MB) and molecular beacon aptamer (MBA) was discussed in this chapter. MBs are single-stranded oligonucleotide probes with a hairpin ...
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Protocol
Lentiviral Vector Delivery of siRNA and shRNA Encoding Genes into Cultured and Primary Hematopoietic Cells
Lentiviral vectors are able to transduce non-dividing cells and maintain sustained long-term expression of the transgenes. Many cell types including brain, liver, muscle, and hematopoietic stem cells have been...
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Protocol
Design of Trans-Splicing Adeno-Associated Viral Vectors for Duchenne Muscular Dystrophy Gene Therapy
The development of trans-splicing vectors opens the door for delivering a large therapeutic gene with adeno-associated viral vectors (AAV). One potential application is to deliver the 6 kb mini-dystrophin gene fo...
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Protocol
Directed Evolution of Adeno-Associated Virus (AAV) as Vector for Muscle Gene Therapy
Adeno-associated virus (AAV) is emerging as a vector of choice for muscle gene therapy because of its effective and stable transduction in striated muscles. AAV naturally evolve into multiple serotypes with di...
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Protocol
Monitoring Murine Skeletal Muscle Function for Muscle Gene Therapy
The primary function of skeletal muscle is to generate force. Muscle force production is compromised in various forms of acquired and/or inherited muscle diseases. An important goal of muscle gene therapy is t...
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Protocol
Exploiting Natural Diversity of AAV for the Design of Vectors with Novel Properties
Twelve AAV serotypes have been described so far in human and nonhuman primate (NHP) populations while surprisingly high diversity of AAV sequences is detected in tissue biopsies. The analysis of these novel AA...
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Protocol
Gene Therapy in Skeletal Muscle Mediated by Adeno-Associated Virus Vectors
Adeno-associated virus (AAV) is the most promising gene delivery vehicle for muscle-directed gene therapy. AAV’s natural tropism to muscle cells, long-term persistent transgene expression, multiple serotypes, ...
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Protocol
Haplotype Inference
Haplotypes, as they specify linkage patterns between individual nucleotide variants, confer critical information for understanding the genetics of human diseases. However, haplotype information is not directly...
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Protocol
Using ΦC31 Integrase to Mediate Insertion of DNA in Xenopus Embryos
The two most common methods used to generate transgenic Xenopus embryos, restriction enzyme-mediated insertion, and I-SceI meganuclease take advantage of relatively common but spatially unpredictable double-stran...
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Protocol
Methods for TALEN-Mediated Genomic Manipulations in Drosophila
TALEN (transcription activator-like effector nuclease) is a powerful tool for gene disruption and other genomic modifications. In the past 3 years or so, it has attracted eyes from every corner of the biologic...