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Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...

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Truncated complement factor H Y402 gene therapy rescues C3 glomerulonephritis.

Lindsey A Chew¹, Daniel Grigsby², C Garren Hester³

¹Department of Ophthalmology, Duke Eye Center, Duke University Medical Center, Durham, NC 27710, USA; Department of Cell Biology, Duke University Medical Center, Durham, NC 27710, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy

|April 26, 2025

Summary

Gene therapy using adeno-associated virus (AAV)-mediated delivery of truncated complement factor H (tCFH) shows promise for treating complement-mediated diseases like C3 glomerulonephritis (C3G) and age-related macular degeneration (AMD). This approach restored complement pathway function and reversed disease in a mouse model without immune rejection.

Keywords:

C3 glomerulonephritis C3 glomerulopathy age-related macular degeneration complement factor H complement regulation gene replacement gene therapy

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Area of Science:

Ophthalmology
Nephrology
Gene Therapy
Immunology

Background:

No effective therapies currently exist for dry age-related macular degeneration (AMD) and C3 glomerulonephritis (C3G).
Previous attempts to treat C3G with human complement factor H (CFH) were hindered by immune rejection.
Challenges in AMD research include identifying therapeutic targets and effective delivery methods.

Purpose of the Study:

To investigate adeno-associated virus (AAV)-mediated delivery of truncated CFH (tCFH) as a potential therapy for C3G.
To explore the parallels in complement dysregulation between AMD and C3G for therapeutic development.
To demonstrate proof of concept for AAV-mediated tCFH gene augmentation therapy for AMD.

Main Methods:

Utilized a Cfh knockout mouse model (Cfh-/-) to study C3G.
Employed adeno-associated virus (AAV) vectors for the delivery of truncated complement factor H (tCFH).
Evaluated three different tCFH vectors for transduction efficiency and therapeutic efficacy.

Main Results:

Successfully restored inhibition of the complement alternative pathway in the Cfh-/- mouse model.
Achieved long-term reversal of C3G disease without inducing immune rejection.
Observed significant variations in therapeutic efficacy among the tested tCFH vectors.

Conclusions:

AAV-mediated tCFH replacement therapy is a promising strategy for C3G treatment.
This study provides proof of concept for AAV-mediated tCFH gene therapy in AMD.
The findings highlight the potential of targeting complement dysregulation for treating both C3G and AMD.