Establishment of HIV-1 resistance in CD4(+) T cells by genome editing using zinc-finger nucleases

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In the past decade, the field of gene therapy is progressing swiftly even though at the beginning did not show any prospects. Nowadays, there are more than 2000 different gene therapies that have entered clinical trials. Gene therapy is a method in which scientists use nucleic acid polymers to treat or prevent certain diseases by altering the patient’s genetic material. The two approaches that are commonly used include transfer of a working gene to replace the demaged one or disrupting the genes that were not working properly. In both cases, the working gene as well as genes that edit chromosomal DNA, have to be administered to the patient, to get into the demaged cell, enter the cell and express proteins to achieve desired effect. Therapeutic genes can be incorporated into a viral vector and administered to the patient as vaccine or the target cells can be transfected ex vivo and then returned to the patient, in so called adoptive cell transfer. The role of viral vectors is to deliver and incorporate DNA to the cell genome that would hopefully result in expression of proteins, which will modify cells’ phenotype back to normal. However, certain disadvantages that are present when using viral methods are circumvented with the usage of non-viral methods such as electroporation, the injection of naked DNA and the use of dendrimer, etc. Generally, the most often used method in gene therapies is the incorporation of additional gene to the cell genome. However, with the gained knowledge of the function of nucleases scientists started to use them as a reagents in editing cromosomal DNA. For example, zinc finger nucleases have become useful reagents for gene modifications of many plants and animals. It works in two steps: • Recognition of the sequence, • cleveage of the chromosomal DNA. This method allows scientists to disable or edit a demaged allele by taking advantage of the endogenous DNA repair mechanism.

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