Gene Therapy Development for Acquired Diseases
Acquired disorders is one that begins at some point during one's lifetime, as opposed to disease that was already present at birth. Acquired disorders are much more common than genetic disorders such as inflammatory, neoplastic, or infiltrative diseases, etc. There are numerous acquired diseases in which viral or non-viral vector-mediated gene therapy has shown in proof-of-principle experiments a therapeutic benefit. As a CRO specializing in the field of gene therapy development, Creative Biolabs has established one-stop service platform to offer worldwide clients with preclinical or clinical requirements. Of note, various robust acquired disease models and assay systems have been used in-house and our service will definitely help move the gene therapy for acquired disease into the clinic more quickly.
With profound genetic knowledge and professional technical staff, our best gene therapy development services for acquired disorders include but not limited to:
Figure 1. Genetically modified adenovirus acting as a suicide gene. (Amer 2014)
Over the past decades, the gene technological advances coupled with promising clinical data have made cancer one of the major disease targets for gene therapy. Multiple prospective gene therapy strategies have been developed to treat different cancers, including the insertion of tumor suppressor gene to compensate for its loss or deregulation, the expression of a gene to induce apoptosis or enhance tumor sensitivity to anticancer drug, enhancing the immunogenicity of the tumor, and blocking the expression of an oncogene via antisense approach. Various innovative genetic approaches have been developed in Creative Biolabs to construct the vector or transgene and progress their safety and antitumor efficiency. Several robust animal models and assay systems are able to provide for our clients to meet your individual requirements.
Figure 2. Cardiovascular gene therapy using adenoviral vectors. (Bradshaw 2013)
Cardiovascular diseases remain a large health problem in the world. Currently, the pharmacologic drugs and device therapies have multiple limitations, but the development of gene knowledge and delivery systems has given researchers an opportunity to use gene therapy strategies to treat cardiovascular diseases. Early studies suggest that the transfer of myocardial gene could produce vascular endothelial growth factor (VEGF) or fibroblast growth factor (FGF) to improve angiogenesis. In addition, myocardial gene transfer also reduces arrhythmia vulnerability and increases myocardial contractility. There exist several delivery vectors for use in the clinic, including viral and non-viral based vectors.
Diseases of the nervous system are often caused by inherited genetic mutations or genetic changes induced by disease-related events. Standard medical and surgical practice is not effective to cure these diseases and gene therapy as a powerful approach shows huge potential to treat some of the most common neurological diseases. The most common central nervous system (CNS) gene therapy vectors, AAV, HSV and adenoviral vectors have been used to deliver therapeutic genes directly into the CNS with better safety. With years of experience, Creative Biolabs can provide the development service covering the full process, including the disease mechanisms research, gene therapy targets, therapeutic gene selection, gene vector design, and methods of delivery, particularly the preclinical validation.
Although current antiretroviral drug therapy can suppress the replication of human immunodeficiency virus (HIV), it requires lifelong administration as the HIV provirus integrated within infected cells cannot be eliminated and virus replication resumes following antiviral discontinuation. Moreover, there are a lot of limitations, such as cumulative toxicities, incomplete immune restoration, elevated cost, drug resistance, etc. Recently, advances in the field of gene-targeting strategies and gene therapies have revolutionized the HIV therapeutics. Most anti-HIV gene therapies have tried to block the infection of target cells, and allow viral clearance from carriers or prevention of infection in newly exposed individuals.
We are proficient in every process of gene therapy development for various acquired disorders and choosing Creative Biolabs can greatly accelerate your research to clinical application. We are aiming to provide one-stop, full spectrum service to deliver the most efficient and economic outcomes. If you are interested in our service, please feel free to contact us for more information.
References
- Amer, M. (2014). Gene therapy for cancer: present status and future perspective. Molecular and Cellular Therapies, 2(1), p.27.
- Bradshaw, A. and Baker, A. (2013). Gene therapy for cardiovascular disease: Perspectives and potential. Vascular Pharmacology, 58(3), pp.174-181.
- Weinberg, M., Samulski, R. and McCown, T. (2013). Adeno-associated virus (AAV) gene therapy for neurological disease. Neuropharmacology, 69, pp.82-88.
