CRISPR/Cas9 System and its Research Progress in Gene Therapy.

Area of Science:

• Molecular Biology
• Genetics
• Biotechnology

Background:

• Genome editing involves altering an organism's genetic sequence through knockout, insertion, or site mutation.
• The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein-9 nuclease (Cas9) system is a powerful genome editing tool.
• CRISPR/Cas9 originates from microbial immune systems and precisely targets DNA sequences via complementary base pairing.

Purpose of the Study:

• To review the history, structure, and mechanism of the CRISPR/Cas9 system.
• To explore the diverse applications of CRISPR/Cas9 technology.
• To focus on the use of CRISPR/Cas9 in gene therapy and dermatological disease treatment.

Main Methods:

• Literature review of CRISPR/Cas9 history, structure, and mechanism.
• Analysis of CRISPR/Cas9 applications in biotechnology, gene therapy, and dermatology.
• Synthesis of current research on CRISPR/Cas9 for therapeutic interventions.

Main Results:

• CRISPR/Cas9 enables accurate editing of endogenous genomic DNA sequences.
• The system has broad applicability across various scientific and medical fields.
• Significant progress has been made in utilizing CRISPR/Cas9 for cancer gene therapy and treating skin conditions.

Conclusions:

• CRISPR/Cas9 is a versatile and precise genome editing tool with transformative potential.
• Its applications in gene therapy and dermatology are rapidly expanding.
• Further research promises enhanced therapeutic strategies for genetic and dermatological diseases.