Exposing SMA-affected cells to aminoglycoside drugs can increase SMN

NewsGuard 100/100 Score

MDA grantee Christian Lorson at the University of Missouri was on a team that found that exposing cells carrying a mutation that causes spinal muscular atrophy (SMA) to drugs in the aminoglycoside family helps them produce more of the needed SMN protein, a lack of which leads to SMA.

One way to explain the increase, Lorson says, is that a molecular “tail” is added to the short, relatively unstable form of SMN that SMA-affected cells make, making it more like the full-length form of SMN that they lack. Aminoglycosides are known to allow cells to “read past” certain genetic stop signals and thereby produce longer protein molecules.

Although this seems a likely explanation, Lorson notes that there are other possibilities. “It may be that we are interfering with the cell’s normal pathway for protein degradation, or that some other protein, such as an SMN binding protein, is altered by the drug and can then stabilize the short SMN protein.”

In a paper published in the May 1 issue of Human Molecular Genetics, the investigators say that the aminoglycoside effect “identifies a possible alternative approach for therapeutic intervention” in SMA.

Comments

The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical.
Post a new comment
Post

While we only use edited and approved content for Azthena answers, it may on occasions provide incorrect responses. Please confirm any data provided with the related suppliers or authors. We do not provide medical advice, if you search for medical information you must always consult a medical professional before acting on any information provided.

Your questions, but not your email details will be shared with OpenAI and retained for 30 days in accordance with their privacy principles.

Please do not ask questions that use sensitive or confidential information.

Read the full Terms & Conditions.

You might also like...
Study finds microdosing LSD leads to longer sleep: Insights from a controlled trial