genevirusnonviral vectorgene deliveryGene therapy promises to be the next revolution in medicine, in which drugs made of genetic material can be used to treat disease. Although success has been limited thus far, new technological advances are providing hope for the future.doi:10.1038/npg.els....
The solution, ideally, would be to specifically correct the mutant copy using CRISPR. With this gene-editing tool, scientists can direct a pair of molecular "scissors" to any part of the genome (DNA). Unfortunately, first-generation CRISPR technologies—while good at cutting genes—do not have...
For years, researchers have attempted to harness the full potential of gene therapy, a technique that inserts genes into a patient's cells to treat aggressive diseases such as cancer. But getting engineered DNA molecules into cells is not an easy task. J. Mark Meacham, assistant professor of ...
“From then on, numerous studies on HIV-1/AIDS gene therapy by CRISPR/Cas9 technology have been reported, suggesting its tremendous potential to treat HIV-1/AIDS.” They noted that CRISPR technology offers more convenient and efficient target site design, easier vector construction, ...
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The safe, effective, and tissue-specific delivery of CRISPR-Cas tools in vivo determines whether CRISPR-based gene therapy can be used for this tissue. Therapeutic in vivo delivery systems for CRISPR-Cas have recently been discussed [118,119]. CRISPR-Cas tools can be delivered in the form of...
doi:10.1038/nbt0194-42Summers, Nevin M.Cooney, Charles L.Nature BiotechnologySummers, N., and Cooney, C., 1994, "Gene therapy: biotech's n + 1 technology", Bio/Technology, Vol.12, No.1, pp.42-45.
Gene transfer into hepatocytes and human liver tissue by baculovirus vectors. Gene therapy of liver diseases requires the development of efficient vectors for gene transfer in vivo. Retroviral and adenoviral vectors have been shown t... Sandig,Hofmann,Steinert,... - 《Human Gene Therapy》 被引量...
Researchers demonstrate that cholesterol-enriched exosomes can deliver siRNA directly into cancer cells, bypassing normal cellular barriers and significantly enhancing gene silencing. This offers a more effective method for gene therapy applications.
31引文 斯高帕斯(Scopus) 摘要 Rapid propagations in materials technology together with biology have initiated great hopes in the possibility of treating many diseases by gene therapy technology. Viral and non-viral gene carriers are currently applied for gene delivery. Non-viral technology is safe an...