SELF COMPLEMENTARY AAV-MEDIATED DELIVERY OF INTERFERING RNA MOLECULES TO TREAT OR PREVENT OCULAR DISORDERS
The invention provides methods for delivering interfering RNA molecules to an eye of a patient to treat ocular disorders. In particular, the methods of the invention comprise the use of a self-complementary adeno-associated (scAAV) viral vector that can deliver an interfering RNA molecule to an eye...
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Format: | Patent |
Sprache: | eng ; spa |
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Zusammenfassung: | The invention provides methods for delivering interfering RNA molecules to an eye of a patient to treat ocular disorders. In particular, the methods of the invention comprise the use of a self-complementary adeno-associated (scAAV) viral vector that can deliver an interfering RNA molecule to an eye of a patient to inhibit expression of a gene that is associated with an ocular disorder.
La invención proporciona métodos para suministrar moléculas de ARN interferentes al ojo de un paciente para tratar trastornos oculares. En particular, los métodos de la invención comprenden el uso de un vector viral adeno-asociado auto-complementario (scAAV) que puede suministrar una molécula de ARN interferente al ojo de un paciente para inhibir la expresión de un gen que se asocia con un trastorno ocular. |
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