Exploratory Therapy for the Skeletal/Dental Phenotype in PHOSPHO1 Deficiency

PHOSPHO1 缺乏症骨骼/牙齿表型的探索性治疗

• 批准号: 10427969
• 负责人: JOSE LUIS MILLAN
• 金额: $ 30.93万
• 依托单位: SANFORD BURNHAM PREBYS MEDICAL DISCOVERY INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-03-11 至 2024-02-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10427969
• 关键词: Ablation; Alkaline Phosphatase; Biological; Childhood; Clinical Data; Defect; Dental; Disease; Embryo; Fracture; Genes; Human; Hypophosphatasia; Injection Site Reaction; Injections; Laboratories; Lead; Minerals; Mus; Mutation; Osteomalacia; Patients; Phenotype; Phosphoric Monoester Hydrolases; Rare Diseases; Therapeutic; Tissues; Viral Vector; Work; adeno-associated viral vector; biomineralization; bone; comparative efficacy; early onset; in vivo; mineralization; novel therapeutic intervention; pre-clinical; scoliosis; skeletal; symptomatology; therapeutic enzyme

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