Functional nucleic acid molecules directed to targets for nervous system disorders
OwnerLaura GRASSO, Carlotta BON +3inventors
InventorsLaura GRASSO, Carlotta BON, Stefano GUSTINCICH, Stefano ESPINOZA, Salvatore PULCRANO
Life of this patent
Estimating the term…
Abstract
The invention relates to therapeutic agents which enhance protein translation of an endogenous mRNA sequence, particularly for use in a method of treating a disease or disorder of the nervous system. In particular, the therapeutic agent is a functional nucleic acid molecule comprising at least one target determinant sequence and at least one regulatory sequence.
Claims22
-23. (canceled)
A functional nucleic acid molecule comprising:
- at least one target binding sequence comprising a sequence reverse complementary to a target mRNA sequence selected from the group consisting of:
- a Nurr1, GDNF, cRET or GBA mRNA sequence;
- and
- at least one regulatory sequence comprising a SINE B2 element, a functionally active fragment of a SINE B2 element, an internal ribosome entry site (IRES) sequence or a functionally active fragment of an internal ribosome entry site (IRES) sequence.
A method of treating a disease or disorder of the nervous system comprising administering a therapeutic agent that increases translation of an endogenous mRNA sequence selected from the group consisting of:
- an endogenous Nurr1, GDNF, cRET or GBA mRNA sequence, to a subject with a disease or disorder of the nervous system, to a subject with a disease or disorder of the nervous system.
A method of producing a non-human animal model of Parkinson's disease, comprising:
- administering a functional nucleic acid molecule comprising:
- at least one target binding sequence comprising a sequence reverse complementary to a SNCA mRNA sequence;
- and at least one regulatory sequence comprising a SINE B2 element, a functionally active fragment of a SINE B2 element, an IRES sequence or a functionally active fragment of an IRES sequence, to a non-human animal.
Dependencies are inferred from the claim text.