Gene therapy: advances, challenges and perspectives.

Area of Science:

• Genetics and Bioengineering
• Molecular Biology
• Medical Genetics

Background:

• Site-specific genome modification is a long-standing medical goal.
• Gene therapy aims to correct genetic defects or introduce therapeutic modifications.
• Advances in genetics and bioengineering facilitate the development of gene therapy vectors.

Purpose of the Study:

• To review the advancements in gene therapy techniques.
• To discuss the optimization of delivery vectors for gene therapy.
• To highlight recent biotechnological breakthroughs relevant to gene therapy.

Main Methods:

• Review of scientific literature on gene therapy.
• Analysis of vector technologies, including plasmids, nanostructures, and viruses.
• Discussion of in vivo studies and clinical trial outcomes.
• Exploration of emerging technologies like induced pluripotent stem cells and CRISPR/Cas9.

Main Results:

• Viral vectors are effective for cell invasion and genetic material delivery.
• In vivo studies in somatic cells show satisfactory results.
• Clinical trials for gene therapy are approved and ongoing globally.
• Emerging technologies show significant potential for therapeutic applications.

Conclusions:

• Gene therapy holds significant promise for treating genetic diseases.
• Vector optimization and safety remain critical areas of research.
• Recent biotechnological advances are expanding the scope and efficacy of gene therapy.