From Liver Transplantation to Gene Therapy: A Therapeutic Revolution in Argininosuccinic Aciduria
A special issue of Cells (ISSN 2073-4409).
Deadline for manuscript submissions: 31 March 2026 | Viewed by 76
Special Issue Editor
Special Issue Information
Dear Colleagues,
This Special Issue aims to focus on the therapeutic revolution in the field of argininosuccinic aciduria (ASA). ASA is the second most common urea cycle disorder, primarily affecting the liver and presenting with systemic manifestations, which highlight the complexity of this disease.
Recent years have seen remarkable progress; clinical studies have demonstrated neurological benefits following liver transplantation, while innovative gene-based approaches—including AAV- and lentiviral-mediated therapies, mRNA replacement, and gene editing—are offering new hope for curative, less invasive, and potentially long-term solutions.
The aim of this Special Issue is to showcase the latest discoveries regarding ASA, ranging from advances in understanding its underlying mechanisms, within and beyond the liver, to the development of transformative therapeutic strategies. We invite contributions ranging from original research articles to comprehensive reviews that highlight these exciting developments and their potential to reshape the future of ASA treatment.
We look forward to receiving your contributions.
Dr. Sonam Gurung
Guest Editor
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Keywords
- urea cycle disorder
- argininosuccinic aciduria
- gene therapies
- mRNA therapies
- liver transplantation
- arginosuccinate lyase (ASL)
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