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Cell and gene therapy for kidney disease
Jennifer L Peek1,2, Matthew H Wilson3,4,5,6
1Medical Scientist Training Program, Vanderbilt University School of Medicine, Nashville, TN, USA.
Nature Reviews. Nephrology
|March 27, 2023
Summary
Novel cell and gene therapies offer promising new avenues for treating kidney disease, particularly monogenic forms. These advanced treatments could overcome limitations of current interventions like dialysis and transplantation.
Area of Science:
- Nephrology
- Genetics
- Regenerative Medicine
Background:
- Kidney disease is a major global health burden with limited treatment options.
- Current therapies like dialysis and transplantation have significant drawbacks.
- A substantial portion of kidney disease cases stem from monogenic disorders, suggesting genetic medicine as a potential solution.
Purpose of the Study:
- To review the potential of cell and gene therapy for kidney disease.
- To highlight recent advances and emerging technologies in renal genetic and cell therapies.
- To discuss critical considerations for implementing these therapies in nephrology.
Main Methods:
- Review of recent genetic studies and technological advancements in cell and gene therapy.
- Analysis of the applicability of cell and gene therapy to monogenic and systemic kidney diseases.
- Discussion of challenges and future directions for renal genetic and cell therapies.
Main Results:
- Cell and gene therapy show significant promise for treating kidney diseases.
- Advances in genetic studies and technologies are paving the way for novel renal therapies.
- Despite progress, no approved cell or gene therapies currently target the kidney.
Conclusions:
- Cell and gene therapy represent a potential paradigm shift in managing kidney disease.
- Further research and development are crucial to translate these therapies into clinical practice for kidney disorders.
- Addressing key considerations will be vital for the successful application of renal genetic and cell therapies.
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