3. Genetic medicines and genome editing - possible cure for monogenic and polygenic hereditary disorders

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Rabia Bibi Zainab Tariq Tanveer Hussain


Modern therapeutics and drugs use DNA/RNA transfer techniques for the modification of gene expression levels, correction and compensation of mutant genes. Molecular approaches like genetic medicines and genome editing technology can lead to comprehensive and wide-range of medical and scientific acceptance. Due to the feasibility of these techniques, these can help in the treatment of monogenic disorders, neurodegenerative diseases, multifactorial inheritance disorders or polygenic hereditary disorders, including liver, bone, nervous and skeletal syndromes. Not only this, circulating enzyme, hormonal, and coagulation factors deficiencies can also be investigated through genome editing technology. However, successful application of these strategies on clinical human trials can only be implemented by overcoming regulatory, economic and socio-political concerns. The review would focus on the practice of genetic medicines and gene therapy for the treatment of heredity disorders.

Keywords: DNA transfer; Genetic medicines; Genome Editing; Human trials; Monogenic disorders


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