Abstract
Gene therapy is the therapeutic delivery of a gene or nucleic acid into a patient’s cells to cure or alleviate the symptoms of a disease that was caused by genetic malfunction, either gain- of loss-of function. Throughout the years, gene therapy has been faced with fluctuations of development before reaching its current stage. In the early stage, gene therapy was concerned to possess several problems such as toxicity, mutagenesis, and adverse immune responses which would harm the patients, instead of benefiting them. Fortunately, gene therapy has currently reached the phase where its administration can be performed in a safe, controllable manner with a good tolerability and excellent therapeutic effect. This review will recite the development of gene therapy research, highlight the vector-related safety issues, and discuss the latest updates in recent clinical trials with promising results in correcting gene defects in the cell, reducing the symptoms of the disease, as well as improving the patient’s quality of life.
Highlights
The potency of gene therapy to cure genetic diseases has been exclaimed throughout its developmental history, intertwined with optimisms of the researchers, financial supports from the stakeholders, as well as hope and wishes from the patients and their families
A personalized approach to characterize potential recipients for gene therapy is critical, because when it comes to clinical trials, one “unexpected result” will not just affect the error bars but may halt the entire research community
Concluding remarks This review does not cover extensively the successful clinical trials for sickle cell anemia, thalassemia major, lymphoma (Kumar, Markusic, Biswas, High, & Herzog, 2016), nor the potential future for genome-editing therapy (Gabriel, von Kalle, & Schmidt, 2015; Supit, 2017) which will broaden the scope of gene therapy for the benefits of humanity
Summary
The potency of gene therapy to cure genetic diseases has been exclaimed throughout its developmental history, intertwined with optimisms of the researchers, financial supports from the stakeholders, as well as hope and wishes from the patients and their families. A personalized approach to characterize potential recipients for gene therapy is critical, because when it comes to clinical trials, one “unexpected result” will not just affect the error bars but may halt the entire research community Despite this unfortunate case, the field kept progressing, and as the potential adverse effects are being addressed and tackled, we are anticipating more encouraging reports from ongoing clinical trials and basic biomedical research. Children with spinal muscular atrophy (SMN) type 1 usually need mechanical ventilator and rarely reach beyond 2 years of age (Bharucha-Goebel & Kaufmann, 2017) Both groups can benefit from a single injection dose of gene therapy with almost 100% success rate. Once the delivery has been optimized, a secure and firm scientific basis that this gene will be delivered righteously will be established to cure diseases from their roots. ***
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