NUCLEIC ACID MOLECULE, EXPRESSION CASSETTE, EXPRESSION VECTOR, EUKARYOTIC HOST CELL, INDUCTION METHOD OF RNA INTERFERENCDE IN EUKARYOTIC HOST AND USE OF THE NUCLEIC ACID MOLECULE IN THERAPY OF DISEASE

Abstract: Subjects of the invention are: nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of RNA interference in eukaryotic host and use of nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide CAG-type repeats. Solution relates to the new concept of treating hereditary human neurological diseases caused by expansion of CAG-type trinucleotide repeats using RNA interference technology.
Patent Number: US2016376586(A1)
Public Date:
Inventor: KRZYZOSIAK WLODZIMIERZ [PL]; OLEJNICZAK MARTA [PL]; GALKA-MARCINIAK PAULINA [PL]; FISZER AGNIESZKA [
Applicant: INST CHEMII BIOORGANICZNEJ PAN [PL]
Application Number: US201414916039 20140902Global Dossier
Application Date:
Priority Number: PL20130405224 20130902;WO2014PL00100 20140902
International Classification: C12N15/113
Cooperative Classification: C12N15/113; C12N2310/141; C12N2310/334; C12N2310/335; C12N2310/336; C12N2310/531; C12N2310/533; C12N
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