'Molecular Prosthetics' May Give New Hope for Incurable Diseases

Small molecules may replace proteins to cure diseases the way prosthetics replace missing body parts.

Scientists believe they may be able to use molecules to replace deficient proteins, which are the source of many diseases. The treatment would be similar to how artificial limbs replace physical functions.
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Scientists may be on their way to treating some diseases the same way they treat patients with missing limbs, according to new research describing the emerging field of "molecular prosthetics."

Just as a peg leg or an artificial hand replaces physical functions, scientists believe certain molecules could replace protein functions that are either inhibited or absent in humans. Many diseases – from epilepsy to cystic fibrosis to migraines – are caused by missing or deficient proteins.

[READ: Protein Linked to Cancer Growth May Also Lead to Cure]

In research presented Monday at the American Chemical Society's annual meeting, researcher Martin Burke described how scientists may one day be able to use small molecules to create new drugs that would replace these protein functions and treat the diseases.

"Artificial limbs replace the function of an arm or leg that's missing due to injury," said Burke, an associate professor at the University of Illinois at Urbana-Champaign, in a statement. "Some diseases occur because proteins in the body are missing or not working properly. Molecular prosthetics envisions treating those diseases with medicines that replace the functions of the missing proteins."

But in order to make this new branch of medicine a reality, scientists must first overcome two hurdles: speeding the process of synthesizing the molecules and understanding how molecules can operate like proteins.

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Burke and his team previously developed a platform to make synthesize molecules in a matter of days. Normally, that process can take months or even years, Burke said.

"With advances in genomics, a person will soon know all of their genes and which one(s) are missing or defective," Burke said. "With that knowledge and a quick way to make small molecules that function like proteins, our ultimate goal is to help enable the development of treatments that can be given to people before symptoms even arise."

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