"Tiny wound-up circular bits of DNA that carry a short "hairpin" of RNA could prove a solution to the quest for a non-toxic, long-lived gene therapy vector designed to silence cancer genes in tumor cells, said researchers from Baylor College of Medicine and The Methodist Hospital Research Institute in a report that appears in the current issue of the journal Gene Therapy.
At a minimum, this Minivector™ DNA will prove a valuable laboratory tool, said Dr. Lynn Zechiedrich, associate professor of molecular virology and microbiology at BCM and one of the report's senior authors. "In the best case scenario, it could prove a valuable therapeutic tool that can work on cells that are difficult to manipulate. This may help clinicians treat some diseases."
Minivector DNA avoids the toxicity of viruses, lasts longer than plasmids (large DNA circles containing bacterial sequences toxic to human cells) and survives and continues gene delivery longer than small interfering RNAs (siRNA), currently used to silence the genes in the laboratory research setting. Plasmids are as much as 10 times larger than Minivectors, and siRNAs, while small, are too unstable in blood to translate to patient treatment.
"Plasmids are too large and toxic," said Zechiedrich. "Smaller is better. Also, deleting toxic bacterial sequences from the tiny circles prevents bad side effects."
Lymphoma is the fifth most common cancer in the United States, and a type called anaplastic large cell lymphoma is the most common T-cell lymphoma in children and the second most common aggressive systemic T-cell lymphoma in adults. Doctors treat it with a specific regimen of a cocktail of many anti-cancer drugs."
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