6 Result(s)
within
Protocol
Lu**g Li
English
Animal Genetics and Genomics
Gene Therapy
Human Genetics
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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
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...
