Genetic Construct
OwnerMichael McDonaldinventor
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Abstract
The invention relates to the use of genetic constructs, expression cassettes and recombinant vectors comprising such constructs and cassettes for gene therapy and methods for treating neurodegenerative disorders, such as Parkinson's disease (PD). The constructs comprise a promoter operably linked to a first coding sequence, which encodes tyrosine hydroxylase (TH), and a second coding sequence, which encodes GTP cyclohydrolase 1 (GCH1). The second coding sequence is 3′ to the first coding sequence, and the first and second coding sequences are part of a single operon, wherein the genetic construct does not encode aromatic amino acid decarboxylase (AADC). The construct is delivered to the cerebrospinal fluid (CSF) of the subject.
Claims22
1-26. (canceled)
A method of treating, preventing, or ameliorating a neurodegenerative disorder in a subject, wherein the method comprises administering, to a subject in need of such treatment, a genetic construct comprising a promoter operably linked to a first coding sequence, which encodes tyrosine hydroxylase (TH), and a second coding sequence, which encodes GTP cyclohydrolase 1 (GCH1), wherein the second coding sequence is 3′ to the first coding sequence, and the first and second coding sequences are part of a single operon, wherein the genetic construct does not encode aromatic amino acid decarboxylase (AADC), and wherein the construct is delivered to the cerebrospinal fluid (CSF) of the subject.
Dependencies are inferred from the claim text.