Trial Offers Hope to Children with Hypoplastic Left Heart Syndrome.
Molecular therapy the journal of the American Society of Gene Therapy(2024)
摘要
Mesenchymal stromal cells (MSCs) have been the subject of intense study for decades, and their potency as a biological therapeutic agent has been demonstrated in a variety of animal models of disease and in numerous human phase 1/2 clinical trials. Nevertheless, few phase 3 clinical trials have unequivocally confirmed their ability to mediate their expected medical benefit. It is likely the lack of standards regarding MSCs' critical quality attributes and of harmonized practices regarding tissue sources, isolation, and culture methods have all contributed to these conflicting outcomes.
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