Oxford Biomedica (uk) Limited

Oxford Biomedia (uk) Limited

Nicholas D Mazarakis

Susan Mary Kingsman

Alan John Kingsman

Jonathan Rohll

Stuart Naylor

Mimoun Azzouz

Enca Martin-Rendon

Patrick Aebischer

Provided are retroviral vector genomes and vector systems comprising the genomes. In particular, a retroviral vector genome comprising two or more NOIs, operably linked by one or more Internal Ribosome Entry Site(s); a lentiviral vector genome comprising two or more NOIs suitable for treating a neurodegenerative disorder; and a lentiviral vector genome which encodes tyrosine hydroxylase, GTP-cyclohydrolase I and, optionally, Aromatic Amino Acid Dopa Decarboxylase are provided.

Vector system

Disclosed and claimed are methods for treating or preventing neurodegenerative diseases, conditions or maladies or symptoms or physiology associated therewith, such as treating or preventing Parkinson's disease or symptoms or physiology associated therewith such as motor deficits or nigrostriatal degeneration; or, for inducing nigrostriatal regeneration. Advantageously, the methods involve administering a lentiviral vector that expresses GDNF, such as human GDNF, or a variant, homolog, analog or derivative thereof.

Lentiviral-mediated growth factor gene therapy for neurodegenerative diseases

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The Oxford Science Park, United Kingdom

Nicholas D Mazarakis