Gene therapy is the process of transferring genetic material to treat a disease
or, at the very least, to enhance a patient's clinical condition. Gene therapy can be
implemented by transforming viruses into genetic carriers that deliver the desired gene
to the target cells. Depending on the kind of genome, these vectors can be classified as
either DNA-based or RNA-based viral vectors. Gene therapy holds promise for treating
illnesses that traditional medicine cannot address. To apply it, a patient's cells must
receive one or more nucleic acids or a faulty gene. Clinical gene therapy has advanced
significantly during the past ten years. One can list several noteworthy achievements,
including the development of medicines for diabetes, Alzheimer's, Parkinson's, cystic
fibrosis, and several types of cancer. Long-term studies on the therapy of Alzheimer's
disease have led to the development of various drugs, such as monoclonal antibodies to
Aβ aggregation, cholinesterase inhibitors, and inhibitors of tau aggregation.
Researchers continued to work on the disease even though the drugs did not stop its
advancement, and as a result, gene therapy, a recently developed, state-of-the-art
technique for delivering genes to specific locations where they can express the desired
functionalities, is being introduced. For AD, gene therapy is a promising diseasemodifying treatment. “Protein-coding and noncoding RNAs make up between 70 and
90 percent of the human genome. These RNAs are the primary determinants of
biological variation in cells and populations. Among the different kinds of nucleic
acids, RNA is more adaptable than DNA because of its single-stranded structure, direct
protein-encoding capability, and high degree of modification for specific regulatory
and therapeutic uses. Even with its bright future in biomedicine, RNA-based therapy
still has several obstacles to overcome. Notably, delivering RNA effectively and
precisely while reducing immunological reactions is one of the biggest technical
challenges. Viral vectors, Virus-Like Particles (VLPs), lipid nanoparticles (LNPs), and
Extracellular Vesicles (EVs) are some of the methods that have been created for
delivering RNA.
Keywords: Biopolymers, Encoded molecules, Germline, Immune response, Innovative therapy, Lipid nanoparticles, RNA delivery systems, Somatic cells, Transcriptions, Viral vectors.
