GENE TRANSFER OVERVIEW
Deoxyribonucleic acid ("DNA") is organized into segments called genes, with each gene representing the region of DNA that determines the structure of a protein, as well as the timing and location of such protein's production. Occasionally, DNA for one or more genes can be defective, resulting in the absence or improper production of a functioning protein in the cell. This improper expression can alter a cell's normal function and may result in disease. One goal of gene transfer is to treat these diseases by delivering DNA containing the corrected gene into the affected cells. Also, gene transfer can increase or decrease the synthesis of gene products or introduce new genes into a cell and thus provide new or augmented functions to that cell.
There are several different ways of delivering genes into cells. Each of the methods of delivery uses carriers, called "vectors," to transport the genes into cells. Similar to the relationship between a delivery truck and its cargo, the vector (the "truck") provides a mode of transport and the therapeutic agent (the "cargo") provides the disease remedy. These carriers can be either man-made components or modified viruses. The use of viruses takes advantage of their natural ability to introduce DNA into cells. Gene transfer takes advantage of this property by replacing viral DNA with a payload consisting of a specific gene. Once the vector inserts the gene into the cell, the gene acts as a blueprint directing the cell to make the therapeutic protein.
Neurologix Gene Transfer Platform
Neurologix's technological approach is based on the targeted delivery of DNA encoding a therapeutic protein or peptide (a "gene") directly into the specific area of the brain associated with a particular neurological disorder. Neurologix's gene transfer programs employ a viral vector known as the adeno-associated virus ("AAV"), based on a non-disease causing human virus, to delivery its gene-based therapies. AAV is considered the vector of choice for human applications because of the efficiency of gene transfer coupled with its safety profile. Characteristics of AAV include:
Neurologix's technological approach is based on the targeted delivery of DNA encoding a therapeutic protein or peptide (a "gene") directly into the specific area of the brain associated with a particular neurological disorder. Neurologix's gene transfer programs employ a viral vector known as the adeno-associated virus ("AAV"), based on a non-disease causing human virus, to delivery its gene-based therapies. AAV is considered the vector of choice for human applications because of the efficiency of gene transfer coupled with its safety profile. Characteristics of AAV include:
- Safety: AAV vectors are based on a virus that has never been associated with a human disease
- Non-Pathogenic
- Non-Replicative and Non-Integrating
- Currently Used in Over 30 Human Clinical Trials
- Efficiency of Delivery: All AAV vectors are stereotactically infused into defined targeted regions; they do not integrate into the genome nor spread systemically. The vectors stably and effectively deliver genes to the local cells. Once in the cell, the Company believes that genes delivered by AAV vectors direct production of effective amounts of protein, on a continuous basis, for the life of that cell (in the case of the brain, for the life of the individual) from a single administration.
- Ability to Deliver Many Different Genes: Most gene coding parts (cDNA) fit into AAV vectors and have been successfully delivered to a wide range of cell types. The Company employs unique genes for the treatment of each disease; the GAD gene for Parkinsons's disease, the NPY gene for Epilepsy, the P11 gene for depression, and the XIAP gene for Huntington's Disease.
- A Simpler and Safer Option than Standard Surgery: We administer our AAV vector-based products in a procedure that is simpler and safer than other established neurosurgical procedures.
“The activity of the motorrelated network declined after surgery and persisted at one year. These network changes correlated with improved clinical disability ratings.”
PNAS, December 4, 2007