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US 2026/0240889 A1Published application
Compositions and methods for prevention and treatment of genetic disease
AssigneeChildren's Medical Center Corporation
InventorDario O. Fauza
Life of this patent
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Abstract
Methods and compositions for the transamniotic delivery of mRNA encoding alpha-1 antitrypsin protein to a fetus for the prenatal treatment of alpha-1 antitrypsin deficiency.
Claims20
A method for treating alpha-1 antitrypsin deficiency (AATD) in a developing fetus, the method comprising:
- administering to the amniotic fluid surrounding a developing fetus an mRNA molecule encoding an alpha-1 antitrypsin (AAT) polypeptide, thereby treating AATD in the developing fetus.
A method for treating a disease or disorder associated with an AAT deficiency in a developing fetus, the method comprising:
- administering to the amniotic fluid surrounding a developing fetus an mRNA molecule encoding an AAT polypeptide, thereby treating the disease or disorder associated with AAT deficiency in the developing fetus.
A method for delivering one or more mRNA molecules to the bloodstream of a developing fetus, the method comprising:
- administering to the amniotic fluid surrounding the developing fetus an mRNA molecule encoding a polypeptide that has reduced expression and/or activity in the developing fetus relative to a healthy developing fetus.
Dependencies are inferred from the claim text.