5 research outputs found
Gene therapy for Parkinson's disease: recent achievements and remaining challenges
Gene therapy is the use of nucleic acids as drugs. Thus, ways had to be developed to deliver this new generation of drugs to target tissues. Various viral and non-viral vectors have been engineered to carry potentially therapeutic nucleic acids into diseased organs or target cells. The brain offers a particular challenge for gene delivery to its constituent cells: it is encased by the skull, separated from the general circulation by the blood brain barrier, and made up of mostly non-dividing cells. The skull limits direct injection of vectors into the brain, the blood brain barrier inhibits the easy entry of vectors injected into the bloodstream, and post mitotic target cells restrict what type of vector can be used to deliver genes to the brain. We will discuss the main challenges faced by gene delivery to the brain, i.e. immune responses to the delivery vectors and therapeutic transgenes and length of duration of the therapy specifically as applied to Parkinson's disease. We will also discuss therapeutic strategies, which could be implemented to treat Parkinson's disease, and the models in which they have been tested
Gene therapy for Parkinson's disease: recent achievements and remaining challenges
Gene therapy is the use of nucleic acids as
drugs. Thus, ways had to be developed to deliver this
new generation of drugs to target tissues. Various viral
and non-viral vectors have been engineered to carry
potentially therapeutic nucleic acids into diseased organs
or target cells. The brain offers a particular challenge for
gene delivery to its constituent cells: it is encased by the
skull, separated from the general circulation by the blood
brain barrier, and made up of mostly non-dividing cells.
The skull limits direct injection of vectors into the brain,
the blood brain barrier inhibits the easy entry of vectors
injected into the bloodstream, and post mitotic target
cells restrict what type of vector can be used to deliver
genes to the brain. We will discuss the main challenges
faced by gene delivery to the brain, i.e. immune
responses to the delivery vectors and therapeutic
transgenes and length of duration of the therapy
specifically as applied to Parkinson's disease. We will
also discuss therapeutic strategies, which could be
implemented to treat Parkinson's disease, and the models
in which they have been tested