Abstract: Compositions and methods for treating subjects with preexisting neuronal damage are disclosed. The compositions and methods use adeno-associated virus (AAV)-based gene delivery systems for delivering glial cell line-derived neutrotrophic factor (GDNF) to subjects with neurodegenerative conditions such as Parkinson's disease.

Abstract: The present invention provides an expression control system with which any gene of interest can be transferred into mammalian cells to thereby change the nature thereof and the gene can be excised at any time by administering a low molecular weight compound to the cells.

Abstract: Methods for treating Parkinson's disease (PD) are provided. Recombinant adeno-associated virus (rAAV) virions are used to deliver genes encoding dopamine-synthesizing enzymes to the central nervous system of a primate. Once delivered, the genes are expressed, which then results in dopamine synthesis and amelioration in the clinical signs and symptoms of PD. The methods of the present invention can be used to deliver the three central dopamine synthesizing enzymes: tyrosine hydroxylase, aromatic L-amino acid decarboxylase, and guanosine triphosphate cyclohydrolase I thereby enhancing dopamine biosynthesis and providing for enhanced therapeutic efficacy.

Abstract: The use of recombinant adeno-associated virus (rAAV) virions for delivery of genes encoding angiogenic factors to muscle tissue is disclosed. The invention describes such methods of delivery and also describes methods for treating an ischemic condition such as myocardial ischemia. The methods include direct delivery of rAAV virions to a muscle via intramuscular injection and indirect delivery to a muscle via an injection into a blood vessel supplying blood to the muscle. The invention provides for sustained expression of the rAAV virion-delivered angiogenic factor gene or genes, which subsequently leads to a quantifiable therapeutic effect, namely an increase in new blood vessel formation resulting in an increase in blood flow to the ischemic muscle tissue.

Abstract: Selective amplification of cells is enabled by introducing into cells a gene encoding a fusion protein comprising (a) a ligand-binding domain, (b) a domain that associates when the ligand binds to the domain of (a), and (c) a domain that imparts proliferation activity to the cells upon the association and stimulating the cells with the ligand.

Abstract: Selective amplification of cells is enabled by introducing into cells a gene encoding a fusion protein comprising (a) a ligand-binding domain, (b) a domain that associates when the ligand binds to the domain of (a), and (c) a domain that imparts proliferation activity to the cells upon the association and stimulating the cells with the ligand.