HISTORICAL BACKGROUND OF GENETHERAPY: Gene therapy was first conceptualized in 1972, with the authors urging caution before commencing gene therapy studies in humans (Friedman and Roblin, 1972). The first FDA-approved gene therapy experiment in the United States occurred in September, 1990, when Ashanti Desilva was treated for Adenosine deaminase-severe combined immunodeficiency (ADA-SCID) using a retroviral vector (Sheridan, 2011).
Since then, over 1,700 clinical trials have been conducted using a number of techniques for gene therapy .At times, progress has seemed slow but the evolution of the field should be put in the context usually required to achieve significant therapeutic advances.
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In July 2012, there was a breakthrough in gene therapy in which alipogene tiparvovec marketed under trade name Glybera was clinically approved by European medicine Agency to be used in Europe and United State for treatment or compensation for lipoprotein lipase deficiency (LPLD) which can cause severe pancreatitis with ensuing diabetes mellitus.
The recommendation was endorsed by the European commission in November 2012 (Gallagher, 2012; Richards, 2012) and commercial roll out is expected in late 2013 according to Press Released (Uniqure) on 02-11-2012. (wikipelia.free encyclopedia). Currently, a team of researchers led by Michel Sadelein in New York were able to cure incurable leukaemia of David Aponte’s and other two patients within just eight days of starting a novel gene therapy.
The cured trio, who were all previously diagnosed with usually fatal relapses of acute lymphoblastic leukaemia have now been in remission for between 5 months and 2 years.The leader said that “the stunning findings were that in all five patients, tumours were undetectable after treatment”. This will serve as a hope to leukaemic patients.
Although early clinical failures in gene caused a dismissal of gene therapy but many clinical successes in 2006-2011 have given a new optimism in the promise of gene therapy. These includes successful treatment of patients with retinal disease, leber’s congenital amaurosis (Maguire, et al., 2008); Simonelli et al., 2009) X-linked severe combined immunodeficiency disease (SCID) (Fischer et al.,2010) ADA-SCID (Adenosine deaminase severe combined immunodeficiency) adrenoleukodystrophy, (Cartier and Aubourg, 2009) chronic myelogenous leukaemia (cml) (Ledford, 2011) and Parkinson’s disease (Lewitt et al., 2011) many heamatological disorders such as thalassaemia, sickle cells, haemophilia leukaemia and other form of cancers and enzyme disorders can be treated using gene therapy if full investigated and only if the mutated gene can be identified and also normal gene correctly coded in vitro using biotechnology knowledge/genetic engineering. These recent clinical successes have led to a renewed interest in gene therapy, with severed articles in scientific and popular publications calling for continued investment in the field (Kolata, 2009).
The human Genome programme in United states of America together with similar programmes in Europe have invested a lot in multi disciplinary research centers that are attempting to determine the makeup of all human genes, and it is hoped that in some years to come, the scientist shall be able to identify and treat all diseases to which humans are susceptible. This will revolutionize modern medicine and hopefully improve the quality of life of all men, women and children with the extinction of terrible effects of some genetic diseases.
MAJOR BLOOD DISORDERS AND THEIR IMPLICATED GENES
Some blood disorder with the genetic abnormities and their mutated onco-genes or tumor suppressor genes have been identified, these include;
Since the oncogenes that cause these blood disorders have been identified, it is believed that correct coding of these proto-oncogenes invitro using gene sequencing with subsequent integration into the bone marrow cells of the patients will eliminated such disorders and provide a total cure.
Gene therapy remains an avenue of hope for many haematological disorders that cannot be effectively treated with drugs, recombinant therapeutic proteins or transplantation. In addition, gene therapy has the potential to transform the lives of those patients who are dependent on lifelong parenteral therapy with recombinant proteins or blood and its products.