Gene editing technology for improving life quality: A dream coming true?

Area of Science:

• Genetics
• Molecular Biology
• Biotechnology

Background:

• Monogenic diseases stem from single-gene mutations, making gene correction a key therapeutic strategy.
• Advances in genome editing have accelerated the development of tools for genetic aberration correction.
• CRISPR/Cas9, TALENs, and ZFNs are powerful genome editing technologies with in vitro and in vivo applications.

Purpose of the Study:

• To provide an updated overview of major gene editing tools.
• To discuss the history, mechanisms, delivery methods, and applications of these technologies.
• To highlight ongoing clinical trials and their outcomes.

Main Methods:

• Review of published in vitro and in vivo studies on gene editing tools.
• Analysis of clinical trial data employing CRISPR/Cas9, TALENs, ZFNs, and meganucleases.
• Compilation of information on the history, nature, delivery, and application of gene editing technologies.

Main Results:

• Gene editing tools demonstrate efficacy in correcting genetic aberrations.
• Clinical trials utilizing these technologies show encouraging outcomes.
• Significant challenges persist regarding the safety and targeted delivery of gene editing tools.

Conclusions:

• Genome editing technologies hold significant potential for treating monogenic diseases.
• Further research is needed to address safety and delivery challenges for clinical translation.
• The field is rapidly advancing, with increasing clinical trials indicating therapeutic promise.