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Scientists Engineer Dead Cells to Treat Muscular Dystrophy

Sep 03, 2014 05:26 PM EDT

Scientists from The Scripps Research Institute (TSRI) have engineered dead cells to synthesize their own drug that can treat a form of muscular dystrophy, and ultimately could lead to many new medicines.

"We're using a cell as a reaction vessel and a disease-causing defect as a catalyst to synthesize a treatment in a diseased cell," TSRI Professor Matthew Disney explained in a news release. "Because the treatment is synthesized only in diseased cells, the compounds could provide highly specific therapeutics that only act when a disease is present. This means we can potentially treat a host of conditions in a very selective and precise manner in totally unprecedented ways."

The promising research was published recently in the journal Angewandte Chemie International Edition.

Myotonic dystrophy type 2 is a rare, relatively mild form of muscular dystrophy, which is a progressive muscle weakening disease. It's caused by a type of RNA defect known as a "tetranucleotide repeat," in which a series of four nucleotides is repeated more times than is normal.

In a process known as "click chemistry," researchers developed a pair of small molecules (modules) that can bind together the two abnormal ends of the cell, essentially like they're holding hands. When the small molecule binds tightly to the defect, it reverses the disease's effects on a molecular level.

"When these compounds assemble in the cell, they are 1,000 times more potent than the small molecule itself and 100 times more potent than our most active lead compound," explained the study's first author Suzanne Rzuczek. "This is the first time this has been validated in live cells."

RNAs, since they are modular, make ideal targets for this type of approach, according to Disney. This technique could potentially treat a host of other incurable diseases, such as ALS (Lou Gehrig's Disease), Huntington's disease, and more than 20 others, all caused by defects in RNA.

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