Through gene therapy, genetic cells can be altered for treatment of diseases by replacing, editing, silencing, or augmenting defective genes responsible for disease development.
Gene therapy is a less-invasive procedure- compared to surgeries; capable of slowing down progressions or targeting and removing diseases, such as Retinitis Pigmentosa, with one treatment.
Determine disease or condition in need of treatment- and further identify malfunctioning or mutated gene contributing to disease.
Determine the need to edit, replace, silence, or augment genes contributing to disease occurrence.
Utilize electroporation or modified viruses to efficiently deliver genetic material into cells for repair.
Test safety and efficacy of gene therapy, as well as, set optimal dosage and delivery method-utilizing standard treatments for comparison.
Compile necessary data on safety, efficacy, and manufacturing to meet Federal Drug Administration (FDA) standards.
Educate clients and patients on implementation process.
Monitor the performance of gene therapy to ensure ongoing safety and effectiveness.
As with any treatment, gene therapy may pose risks and challenges- such as inflammation, antibody-rejection, unintended changes in genomes, and more.
*Information is courtesy of Artificial Intelligence.
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