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A Method of Delivering Genes
to the Central Nervous System
Inventors:
Nigel W. Fraser and John
Wolfe
Tech ID:
FN-9132
Description:
One of the limitations of gene therapy
has been the lack of methods to effectively
deliver genes to the central nervous system.
Herpes simplex virus (HSV) is a neurotropic
virus that naturally establishes latent
infections in the peripheral nervous system
and the central nervous system of humans.
Wistar researchers have developed a novel
method for using neurotropic HSV to deliver
genes of therapeutic value. Additionally,
the Wistar technology promotes long-term
expression of the therapeutic genes, which
is necessary for treatment of a genetic
defect.
The Wistar technology has several
advantages over other methods of delivering genes to the central
nervous system (CNS). First, no helper virus is needed, and second,
long-term expression of the gene is achieved using a naturally-occurring
viral promoter. This technology has been used successfully in vivo
in a mouse model. In this study, the gene encoding §-glucorinodase
was delivered to the CNS and long-term expression (over 4 months)
of the gene product was obtained.
Key Words:
Therapeutic, gene therapy, central nervous system
Applications
and Advantages: This technology can be used to treat
a variety of genetic diseases that affect the CNS, such as lysosomal
storage diseases (e.g. Tay-Sachs, Neimann-Pick, Sly's Disease).
The technology may also be useful for treating other conditions
that result from a biochemical deficiency in the CNS (e.g. ParkinsonÕs
Disease, possibly AlzheimerÕs), or in which delivery of other gene
products may be useful (e.g. delivery of cytokines).
Intellectual
Property Status: A U.S. patent application has been filed.
Licensing
Opportunity: Wistar is seeking
an exclusive license for this technology.
Contact:
Meryle
J. Melnicoff
Director, Business Development
The Wistar Institute
3601 Spruce Street
Philadelphia, PA 19104
Phone: (215) 898-0049
Fax: (215) 573-2456
melnicoff@wistar.org
Last updated: Nov.
99
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