That research is boosted by the ... for therapies to be tested for the first time in humans through “investigator-initiated trials.” · These small-scale clinical trials generally undergo less oversight than the traditional approval pathway for a green light. Similar models exist in other countries and are seen as important to accelerating breakthroughs and administering life-saving treatments especially for rare diseases, where there ... That research is boosted by the country’s so-called dual-track regulatory system, experts say, which allows for therapies to be tested for the first time in humans through “investigator-initiated trials.” · These small-scale clinical trials generally undergo less oversight than the traditional approval pathway for a green light. Similar models exist in other countries and are seen as important to accelerating breakthroughs and administering life-saving treatments especially for rare diseases, where there is little commercial incentive for research. Even as results from related research were published in the Nature journal, the outcome was never made public by the doctors or their institutions. It was against this backdrop the Shanghai couple who shared their story with Science undertook a journey to get treatment for their young daughter. The girl had been diagnosed with a rare gene defect that produces a neurodevelopmental condition known as Snijders Blok-Campeau syndrome, Science reported. The disease can cause intellectual deficits as well as severe physical symptoms. While the girl showed symptoms that were milder than others, her parents decided to explore specialized gene therapy, according to Science. Qiu was known for research working with a cutting-edge technique known as base editing to make custom treatments for children with rare diseases. Under a treatment plan he developed for the family, the girl would be the first person in the world to receive a gene-editing therapy directed at the brain, according to Science.

That research is boosted by the ... for therapies to be tested for the first time in humans through “investigator-initiated trials.” · These small-scale clinical trials generally undergo less oversight than the traditional approval pathway for a green light. Similar models exist in other countries and are seen as important to accelerating breakthroughs and administering life-saving treatments especially for rare diseases, where there ... That research is boosted by the country’s so-called dual-track regulatory system, experts say, which allows for therapies to be tested for the first time in humans through “investigator-initiated trials.” · These small-scale clinical trials generally undergo less oversight than the traditional approval pathway for a green light. Similar models exist in other countries and are seen as important to accelerating breakthroughs and administering life-saving treatments especially for rare diseases, where there is little commercial incentive for research. Even as results from related research were published in the Nature journal, the outcome was never made public by the doctors or their institutions. It was against this backdrop the Shanghai couple who shared their story with Science undertook a journey to get treatment for their young daughter. The girl had been diagnosed with a rare gene defect that produces a neurodevelopmental condition known as Snijders Blok-Campeau syndrome, Science reported. The disease can cause intellectual deficits as well as severe physical symptoms. While the girl showed symptoms that were milder than others, her parents decided to explore specialized gene therapy, according to Science. Qiu was known for research working with a cutting-edge technique known as base editing to make custom treatments for children with rare diseases. Under a treatment plan he developed for the family, the girl would be the first person in the world to receive a gene-editing therapy directed at the brain, according to Science.