Argininosuccinic Aciduria News and Research

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Gene defect associated with argininosuccinic aciduria corrected using CRISPR-Cas9 technique

Argininosuccinate lyase deficiency (ASLD), also known as argininosuccinic aciduria, is a disease that has been enriched in the Finnish genetic heritage.

4 Apr 2024

mRNA therapy shows promise for curing children's rare liver disease

By exploiting the technology used in Covid-19 vaccines, a team led by UCL, King's College London and Moderna scientists has created an effective therapy for a rare disease, in a study in mice, demonstrating the technology's potential therapeutic use in people.

10 Jan 2024

Argininosuccinic aciduria (ASA) and gene therapy: an interview with Dr Julien Baruteau, UCL Institute for Women's Health, London

This rare genetic condition can theoretically affect patients at any age but symptoms are more often observed in childhood and even uncommonly during the first days of life.

2 Sep 2013

Action Medical Research funds three new Research Training Fellowships

Action Medical Research, the UK-wide charity funding vital research to help sick and disabled babies and children, has today announced it is funding three new Research Training Fellowships worth more than £470,000.

9 Jul 2013