[ExI] New ‘Universal Switch’ Lets Scientists Fine-Tune Gene Therapy

[John Grigg]

A very exciting development...

"It’s not that gene therapy was designed to be a rule-breaker, but until
now, scientists haven’t been able to effectively control its strength. Once
unleashed into the body, the treatment replaces a defective gene with a
healthy one, allowing the body to produce functional proteins. But
sometimes the level of those proteins are too low—essentially nixing any
therapeutic effects—and other times too high, poisoning the cells they’re
supposed to treat.

Last week, scientists finally gained control
<https://www.nature.com/articles/s41586-021-03770-2?utm_source=other&utm_medium=other&utm_content=null&utm_campaign=JRCN_1_DD01_CN_NatureRJ_article_paid_XMOL>
of
the technology with a genius strategy. A team from the Children’s Hospital
of Philadelphia (CHOP) tapped into a natural process during gene
expression—when genes make proteins—and hijacked it to make a “dimmer” that
controls the strength of potentially any gene therapy.

Dubbed Xon, the on switch is easy: just pop a simple drug, one that’s
already in later-stage clinical trials. The drug then works with the team’s
custom-designed switch to turn on the therapy throughout the body—the
larger the dose, the stronger the gene expression. As the body metabolizes
the drug, the dimmer gradually turns off the treatment. Need more protein?
Take another pill.

In mice, the team fine-tuned the expression of proteins that help treat
anemia in chronic kidney disease, upped the level of a protein that
protects against a type of dementia, and controlled the strength of CRISPR
for editing genes in the liver.

“We’re taking the field of gene therapy to an entirely new level where
fine-tuned dosing is required for safety, utility, and success,” said
<https://www.chop.edu/news/children-s-hospital-philadelphia-researchers-develop-dimmer-switch-help-control-gene-therapy>
senior
author Dr. Beverly L. Davidson."

https://singularityhub.com/2021/08/03/new-universal-switch-lets-scientists-fine-tune-gene-therapy/
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