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Promising gene therapy delivers treatment directly to brain

Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends computer class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. A treatment Rylae-Ann received recently became the first brain-delivered gene therapy on the market after its approval in Europe and the U.K. for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin walks to physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. (AP Photo/Sakchai Lalit) Richard Poulin, works with his daughter, Rylae-Ann Poulin during a physical and cognitive therapy session at their home in Bangkok, Thailand, Saturday, Jan. 14, 2023. (AP Photo/Sakchai Lalit) Richard Poulin, right, watches his daughter, Rylae-Ann Poulin, climb a rope ladder at a playground in Bangkok, Thailand, Saturday, Jan. 14, 2023. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin learns to ride a horse in Bangkok, Thailand, Saturday, Jan. 14, 2023.(AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. Rylae-Ann was among the first to benefit from a new way of delivering gene therapy _ directly into the brain _ that experts believe holds great promise for treating a host of brain disorders. (AP Photo/Sakchai Lalit) Richard Poulin, left and Judy Wei, right, work with their daughter, Rylae-Ann Poulin, during a physical and cognitive therapy session at their home in Bangkok, Thailand, Saturday, Jan. 14, 2023. The family hopes other families dealing with devastating genetic diseases like Rylae-Ann's will someday get to see the transformations they’ve seen. (AP Photo/Sakchai Lalit) Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. Rylae-Ann’s disease was caused by mutations in a gene needed for an enzyme that helps make neurotransmitters like dopamine and serotonin, the body’s chemical messengers. A one-time treatment delivered a working version of the gene. (AP Photo/Sakchai Lalit) This photo provided by Judy Wei shows her daughter, Rylae-Ann Poulin, the first time she was admitted to an ICU, at a Singapore hospital in September 2018. When Rylae-Ann Poulin was a year old, she didn’t crawl or babble like other kids her age. A rare genetic disorder kept her from even lifting her head. Her parents took turns holding her upright at night just so she could breathe comfortably and sleep. (Judy Wei via AP) Rylae-Ann Poulin smiles as she learns to ride a horse in Bangkok, Thailand, Saturday, Jan. 14, 2023. Rylae-Ann was among the first to benefit from a new way of delivering gene therapy _ directly into the brain _ that experts believe holds great promise for treating a host of brain disorders. (AP Photo/Sakchai Lalit)
Promising gene therapy delivers treatment directly to brain