Essay: Adeno associated virus (AAV) vectors

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In cardiac clinical trials and animal models experiments, functional foreign genes were transferred into cardiomyocytes of patients and in animal hearts respectively using adeno associated virus (AAV) vectors. The delivery routes included were intracoronary injection, myocardia injection, sarcoplasmic reticulum calcium ATPase (SERCA2a) pump and molecular cardiac surgery with recirculating delivery (MCARD). Adeno associated virus (AAV) serotypes 1-9 were applied in experiments studies. The AAV6 and AAV7 serotypes shown significant progress in animal models while AAV1 denoted improvement in cardiac conditions in clinical trials.  Low scores for other serotypes were due to lack of suitable receptors in gene transfer process.

Progress in genetic engineering and gene therapy technique were firstly evolved in 1960’s after the breakthrough in biology which enabled scientists to use enzymes to manipulate DNA sequences. Unlike conventional breeding where the desired traits of organisms were selected within or close species, genetic engineering employs biotechnology to change genetic information of organisms regardless of their species (Brooker et al, 2009).

The artificial manipulation of genomes is made for specific reasons and it rely on a simple principle that DNA is a universal language of life which can be communicated among organisms. In practical, genes can be crossed between any organisms, for example jelly fish and cat, cabbage and scorpion as well as goat and spider.

In modern world, genetic engineering found many of its applications in agriculture and in medications. In agriculture, genetic engineering is interchangeably referred to genetic modification (GM) or genetic improvement (GI). According to (FAO, 2004) biotechnology is an essential tool of genetic engineering which uses biological procedures, living things and their byproducts to alter systems and ultimately makes new product for specific use.

It helps farmers to improve quality of their animals and crops to increase volumes of livestock and crops respectively. Moreover (GE) increases the productivity by strengthening crop resistance to cope with damage associated with weeds and diseases (FAO, 2004).

In medical care, genetic engineering reflects to therapeutic techniques such as stem cells, gene therapy and cloning which strive for treating genetic disorders instigated from mistakes in DNA sequences. In this scenario, (GE) is anticipated to replace mutant gene for a functional gene to cure inherited disorders by coding specific functional proteins needed for synthesis of hormones and enzymes. Moreover, some (GE) techniques are intended to grow certain cells to replace the defective ones.

In that view, basic understanding of deoxyribonucleic acid, DNA, chromosomes and gene as illustrated in Fig. 4 is essential. The DNA and a gene are interchangeably used to express a hereditary strand that controls a blueprint of our life (Kelemen, et al. 2015; Heartl and Jones, 2006).


2.1 Fundamentals of gene therapy

The rationale of gene therapy is to transfer genetic material to cure genetic disorders. When a mutant gene is identified, collective procedures in gene therapy are followed where copies of functional gene are introduced into non-specific areas in DNA to substitute malfunctioned gene (Fig. 5), sometime it is intended to repair

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