Gene therapy in polycystic kidney disease: A promising future

Area of Science:

• Nephrology
• Genetics
• Biotechnology

Background:

• Polycystic kidney disease (PKD) is a genetic disorder characterized by kidney cyst formation, leading to progressive renal dysfunction.
• It encompasses autosomal dominant (ADPKD) and autosomal recessive (ARPKD) forms, with ADPKD being more prevalent.
• Current treatments manage symptoms and slow progression but do not offer a cure.

Purpose of the Study:

• To explore the potential of gene editing technologies for treating polycystic kidney disease.
• To review novel therapeutic strategies targeting genetic defects in PKD.
• To assess the progress and challenges of gene therapy in PKD research.

Main Methods:

• Review of recent advancements in gene editing technologies, including CRISPR-Cas9.
• Analysis of therapeutic approaches such as antisense oligonucleotides and gene knockdown.
• Examination of studies involving Pkd1 gene enhancement and 3-UTR modification.

Main Results:

• Gene editing strategies, including CRISPR-Cas9, have shown promise in animal models and early clinical trials for PKD.
• Specific therapies like miR-17 antisense oligonucleotides and Pkd1 enhancement are under investigation.
• Promising results were observed for ARPKD with c-myc targeting and P2rx7 gene knockdown.

Conclusions:

• Gene therapy presents a significant and hopeful avenue for treating polycystic kidney disease.
• Challenges remain, including technological limitations, ethical considerations, and cost.
• Interdisciplinary collaboration is crucial for advancing effective PKD treatments.