The Potential Benefits & Advantages of AAV Gene Therapy
Potential Advantages of AAV Gene Therapy
Use of Human Genes
AAV gene therapy utilizes human genes as drugs - not small molecules as used by most big pharma (pills). The use of these genes provides us with countless, yet-to-be discovered treatments for disease.
Enhanced Efficacy
The mechanisms of action of these gene drugs would be expected to be more physiologically integrated with our human metabolism. Because of this we would expect such an approach to often give higher efficacy than pill based treatment.
Safety
Through the incorporation of disease monitoring transcriptional promoters, treatment is targeted to where it’s needed - lowering the chance of adverse reactions.
A Long-Term Solution
As AAV-based gene therapy lasts multiple years, perhaps many years, the ever-present concern of patient non-compliance stands to be virtually eliminated.
The Science Behind HGT Treatments
Houston Gene Therapeutics CEO, Dr. Paul Hermonat, explains the science behind AAV gene therapy, with the help of this illustration:
“This illustration depicts AAV’s “parts” (here AAV2 as an example), somewhat simplified, as we know it today:
The ITRs are not genes but are actually structural components needed for replication of the AAV viral DNA.
The Rep gene encodes Rep78, Rep68, Rep52, and Rep40 proteins needed for viral transcriptional regulation, viral DNA replication, and viral DNA packaging. Cap gene encodes the “capsid”, the protective coat proteins (VP1, VP2, and VP3) of the virus, which protect the viral DNA. AAP helps the assembly of the virus. X encodes a protein which enhances viral DNA replication.
Transcriptional start sites include the p5, p19, p40, and p81 promoters.
All internal genes and promoters can be replaced by different ones. Additionally, various cellular promoters can be inserted and, in large part, they can function as the original cellular promoters, a big advantage for AAV-based gene therapy.”
