Direct Delivery of Treatment to the Brain via Gene Therapy

Rylae-Ann Poulin did not crawl or make noises like other children her age when she was a year old. She was unable to even raise her head due to an uncommon hereditary illness. Her parents alternated in holding her during the night to help her breathe easier and get a good night’s sleep. However, months later, medical professionals in Taiwan used genetic medicine to treat the young patient’s brain directly. The 4-year-old can now run, walk, swim, read, and mount horses.

With her family, Rylae-Ann resides in Bangkok, Thailand. She was granted entry into a clinical trial for a novel gene treatment technique. According to experts, the technique has a lot of potential for treating many brain disorders. In Europe and the UK, the technique was accepted for treating AADC deficiency. The illness impairs the nerve system’s cell-to-cell communication networks. PTC Therapeutics, a US pharmaceutical company, intends to apply for the treatment’s approval in the US this year.

The National Institutes of Health (NIH) claims there are roughly 30 trials underway in the United States right now to explore gene therapy to the brain. Numerous disorders are the focus of the research. At Ohio State University, Dr. Krystof Bankiewicz is in charge of one such investigation on AADC insufficiency. Others experiment with therapies for neurological conditions like Parkinson’s, Alzheimer’s, and Huntington’s. There are still challenges, particularly with illnesses resulting from many genes. However, researchers claim that evidence for brain gene therapy is mounting. “We have a lot of exciting times ahead of us,” brain surgeon Bankiewicz stated. “There have been some advancements.”


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